Ebstein AnomalyPhase 1EbsteinNCT02914171
Study of Autologous Bone Marrow Derived Mononuclear Cells for Treatment of Ebstein Anomaly
A Phase 1 study testing Autologous Bone Marrow-derived Mononuclear Cells and Insertable cardiac monitor in people with Ebstein Anomaly, run by Timothy J. Nelson at 1 site in United States.
Trial summary
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNS) collected from bone marrow (BM) and using an add-on intramyocardial delivery for individuals with Ebstein anomaly undergoing surgical intervention compared to the control group undergoing the same surgical… Read more about this study
- Who is it for?
- Ages 6 months to 30 years
- Conditions the study is for
- Ebstein Anomaly
- What is being tested?
- Autologous Bone Marrow-derived Mononuclear Cells and Insertable cardiac monitor
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2016to 18 Mar 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 6 months to 30 years
- Individuals clinically planned for elective surgical Ebstein repair
- Individuals able to undergo bone marrow aspirate according to clinical consultation with Hematology (cell treatment group only)
- Individuals able to undergo preoperative MRI or CT examination
- Individual and/or parent willing and able to give informed consent and willing to commit to completion of follow-up
Who cannot join
- Individuals requiring cavopulmonary shunt at the time of surgical Ebstein repair; planned preoperatively or required intraoperatively
- Individuals with, or reasonably expected to have, complications during surgical Ebstein repair or during post-operative recovery
- Individual who have not completed or will not be completing all pre-procedure work-up within 30 days of surgical Ebstein repair AND lack of pre-procedure work-up documented as a safety concern by a site investigator
- Individuals who have other clinical concerns as documented by a site investigator that could reasonably increase the risk of complications during or after surgical Ebstein repair
- Individuals whose cells have been determined, by the sponsor, to not be acceptable for release to the investigational site or individual whose cells have been compromised after cells released to investigational site (cell treatment group only)
- Individuals who require surgery on pulmonary, mitral, or aortic valve
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- Individuals with pulmonary atresia or atrioventricular discordance with ventriculoarterial discordance
- Individuals with history of ventricular arrhythmia or new onset ventricular arrhythmia after enrollment that requires medical management
- Individuals who have undergone previous sternotomy
- Individuals with preoperative ventricular arrhythmia requiring medical management
- Individuals with severe chronic diseases, extensive extra-cardiac syndromes, or history of any cancer
- Individuals with current IV inotrope requirements
- Individuals with bleeding disorders or history of thrombosis
- Subjects not eligible for MRI or CT examination due to either a medical contraindication, including acute or chronic renal failure
- Individuals with a currently active infection being treated with oral antibiotics
- Individuals with the following conditions within 60 days prior to Ebstein repair surgery:
- Cardiogenic shock or extracorporeal circulation
- Documented infection requiring treatment with intravenous antibiotics
- Cardiac condition requiring emergency procedure
- Current or uncontrolled seizures or neurological injury that has resulted in a persistent deficit
- Multi-system organ failure including acute or chronic renal failure
- Female subjects 10 years and older with positive pregnancy test or lack of effective birth control method during the 30 days prior to surgical Ebstein repair
- Individuals who weigh more than 90 kg
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I Safety and Feasibility Study of Intramyocardial Delivery of Autologous Bone Marrow Derived Mononuclear Cells to Right Ventricle of Patients With Ebstein Anomaly During Cardiac Surgery
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNS) collected from bone marrow (BM) and using an add-on intramyocardial delivery for individuals with Ebstein anomaly undergoing surgical intervention compared to the control group undergoing the same surgical procedure without cell delivery. This add-on procedure has the potential to foster a new strategy for individuals with congenital heart disease.This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) incidence and severity of adverse event and 2) monitor changes in cardiac structure and function.
Read the detailed description
This study is an open label Phase I trial to determine the safety and feasibility of bone marrow-derived mononuclear cells to the right ventricle of subjects with Ebstein anomaly at the time of a planned surgical Ebstein repair compared to the control group undergoing the same planned surgical intervention without the cell delivery add-on procedure. Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial. Subject/Family will decide to participate in cell delivery or control arm. Once informed consent has been obtained, subjects will undergo identical evaluation (other than Hematology consultation and BM harvest occurring only in the cell-treatment group),preoperative values will be established/confirmed and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria.However, individuals at high-risk, primarily neonates and those with preoperative advanced right heart failure will NOT be included such that the risk of this add-on procedure may not be acceptable. Following surgical Ebstein repair for the control group and following cell-based product delivery for the treatment group, all subjects will be followed for 24-months according to a pre-determined schedule that includes imaging studies along with a questionnaire, electrophysiology and laboratory studies at 1, 6, and 24 months post procedure. All subjects will be contacted for surveillance by phone at 3, 12, and 18 month post procedure.
- Conditions
- Ebstein Anomaly
- Being tested
- Autologous Bone Marrow-derived Mononuclear Cells, Insertable cardiac monitor
- Keywords
- Myopathic right ventricle
- Medical subject headings
- Ebstein Anomaly
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Bone Marrow-derived Mononuclear CellsBiological
Also called MNC
Insertable cardiac monitorDevice
Also called ICM, ILR
Following surgical Ebstein repair a Medtronic Reveal LINQ Insertable Cardiac Monitor (ICM) will be placed subcutaneously in the anterior chest to continuously monitor the subject's heart rhythm and to record cardiac information through automatic detection of arrhythmias.
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm | Experimental | Individuals with Ebstein anomaly and underlying myopathic right ventricle undergoing planned surgical intervention using an add-on procedure delivering autologous bone marrow-derived mononuclear cells into the right ventricle. Receives: Biological: Autologous Bone Marrow-derived Mononuclear Cells; Device: Insertable cardiac monitor |
| Control arm | Other | Individuals with Ebstein anomaly and underlying myopathic right ventricle undergoing planned surgical intervention without cell delivery. Receives: Device: Insertable cardiac monitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events from time of index procedure
Safety assessment of adverse events from time of index procedure will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Analysis of cardiac adverse events is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Severity of adverse events from time of index procedure
Safety assessment of severity of adverse events from time of index procedure will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Number of subjects who died
Safety assessment of all-cause mortality will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Number of subjects with sustained symptomatic cardiac arrhythmias
Safety assessment of sustained symptomatic cardiac arrhythmias will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Number of subjects with myocardial infarction
Safety assessment of myocardial infarctions will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Number of subjects with unexpected, invasive cardiovascular procedures
Safety assessment of unexpected, invasive cardiovascular procedures will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Number of serious adverse events
Safety assessment of serious adverse events will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: 24 months post index procedure
Percentage of subjects in the treatment group that have cells delivered
Feasibility of the BM-MNC cell therapy will be evaluated comparing number of subjects enrolled in the treatment group to subjects in the treatment group who are accrued and have cells delivered by percentage using the binomial distribution and exact confidence limits given.
Measured: 24 months post index procedure
Percentage of subjects in the treatment group completing the 24 month follow-up
Feasibility of the BM-MNC cell therapy will be evaluated comparing number of subjects enrolled in the treatment group to subjects in the treatment group who are accrued and have cells delivered, who complete the 24 month follow-up visit by percentage.
Measured: 24 months post index procedure
Incidence of cardiac related hospitalizations from time of Ebstein repair
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 1, 6, and 24 months post index procedure
Incidence of cardiac arrhythmias from time of index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 1, 6, and 24 months post index procedure
Change in CT derived right ventricular dimensions from time of pre-operative evaluation
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. End volume measurements recorded using mL and indexed to body surface area (BSA) using mL/m^2.A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will be used to distinguish common rates from very rare rates. Analysis is in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 6 and 24 months post index procedure
Change in echocardiography derived cardiac output from time of pre-operative evaluation
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group.Volume is measured in L/min and Volume Index is L/min/m^2.Using t-test will have 80% power to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group to distinguish common rates from very rare rates. Analysis in terms of yes/no information at 24 months. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 1, 6, and 24 months post index procedure
Change in NT-Pro-BNP derived cardiac function trend from time of pre-operative evaluation
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: During hospitalization up to 29 days, 1, 6 and 24 months post index procedure
Incidence of cardiac related hospitalizations in the treatment group from time of pre-operative evaluation compared to the control group
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 1, 6, and 24 months post index procedure
Incidence of cardiac arrhythmias in the treatment group from time of pre-operative evaluation compared to the control group
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 1, 6, and 24 months post index procedure
Change in MRI derived cardiac output in the treatment group from time of pre-operative evaluation compared to the control group
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group.Volume is measured in L/min and Volume Index is L/min/m^2.Using t-test will have 80% power to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group to distinguish common rates from very rare rates. Analysis in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 6 and 24 months post index procedure
Change in echocardiography derived right ventricular dimensions from time of pre-operative evaluation
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. End volume measurements recorded using mL and indexed to body surface area (BSA) using mL/m^2,diameter as cm.A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will be used to distinguish common rates from very rare rates. Analysis is in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 1, 6, and 24 months post index procedure
Change in NT-Pro-BNP derived cardiac function trend in the treatment group from time of pre-operative evaluation compared to the control group
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: During hospitalization up to 29 days, 1, 6, and 24 month post index procedure
Number of serious adverse events from time of enrollment
Safety assessment of the number of serious adverse events from time of enrollment will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Measured: index procedure
Percentage of subjects in the treatment group whose cells meet all release criteria
Feasibility of the BM-MNC cell therapy will be evaluated comparing number of subjects enrolled in the treatment group to subjects in the treatment group with collected bone marrow and the bone marrow cells have met all product release criteria to any products that did not meet release criteria by percentage.
Measured: 24 months post index procedure
Change in CT derived cardiac output from time of pre-operative evaluation
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group.Volume is measured in L/min and Volume Index is L/min/m^2.Using t-test will have 80% power to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group to distinguish common rates from very rare rates. Analysis in terms of yes/no information at 24 months. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 6 and 24 months post index procedure
Change in MRI derived right ventricle ejection fraction in the treatment group from time of pre-operative evaluation compared to the control group
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group measured in %.Using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 6 and 24 months post index procedure
Change in MRI derived right ventricular dimensions in the treatment group from time of pre-operative evaluation compared to the control group
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. End volume measurements recorded using mL and indexed to body surface area (BSA) using mL/m^2.A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will be used to distinguish common rates from very rare rates. Analysis is in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 6 and 24 months post index procedure
Change in CT derived right ventricle ejection fraction from time of pre-operative evaluation
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group measured in %.Using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Measured: 6 and 24 months post index procedure
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2016 (estimated)
- Study startSep 2016
- Primary completion18 Mar 2021
- Study completion18 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Ebstein AnomalyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Timothy J. Nelson
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Timothy J. Nelson, Mayo Clinic
- Investigators
- Muhammad Y Qureshi, MBBS, Principal investigator, Mayo ClinicTimothy J Nelson, MD, PhD, Study director, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02914171
- Sponsor's protocol ID
- 15-007580
- Acronym
- Ebstein
- Status last verified
- Apr 2022
- First submitted
- 22 Sep 2016
- Last updated
- 26 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
O'Leary PW, Qureshi MY, Cetta F, Nelson TJ, Holst KA, Dearani JA; Wanek Program Clinical Pipeline Group. Cone Reconstruction for Ebstein Anomaly: Ventricular Remodeling and Preliminary Impact of Stem Cell Therapy. Mayo Clin Proc. 2021 Dec;96(12):3053-3061. doi: 10.1016/j.mayocp.2021.02.015. Epub 2021 Sep 1.
PubMed 34479739
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Safety and Feasibility Study of Intramyocardial Delivery of Autologous Bone Marrow Derived Mononuclear Cells to Right Ventricle of Patients With Ebstein Anomaly During Cardiac Surgery
- ClinicalTrials.gov ID
- NCT02914171
- Acronym / sponsor ID
- Ebstein / 15-007580
- Conditions
- Ebstein Anomaly
- Keywords
- Myopathic right ventricle
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Timothy J. NelsonUniversity, hospital or other
- Study start
- Sep 2016
- Primary completion
- 18 Mar 2021 (actual)
- Study completion
- 18 Mar 2021 (actual)
- First posted
- 26 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNS) collected from bone marrow (BM) and using an add-on intramyocardial delivery for individuals with Ebstein anomaly undergoing surgical intervention compared to the control group undergoing the same surgical procedure without cell delivery. This add-on procedure has the potential to foster a new strategy for individuals with congenital heart disease.This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) incidence and severity of adverse event and 2) monitor changes in cardiac structure and function.
Detailed description
This study is an open label Phase I trial to determine the safety and feasibility of bone marrow-derived mononuclear cells to the right ventricle of subjects with Ebstein anomaly at the time of a planned surgical Ebstein repair compared to the control group undergoing the same planned surgical intervention without the cell delivery add-on procedure. Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial. Subject/Family will decide to participate in cell delivery or control arm. Once informed consent has been obtained, subjects will undergo identical evaluation (other than Hematology consultation and BM harvest occurring only in the cell-treatment group),preoperative values will be established/confirmed and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria.However, individuals at high-risk, primarily neonates and those with preoperative advanced right heart failure will NOT be included such that the risk of this add-on procedure may not be acceptable. Following surgical Ebstein repair for the control group and following cell-based product delivery for the treatment group, all subjects will be followed for 24-months according to a pre-determined schedule that includes imaging studies along with a questionnaire, electrophysiology and laboratory studies at 1, 6, and 24 months post procedure. All subjects will be contacted for surveillance by phone at 3, 12, and 18 month post procedure.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment arm | Experimental | Biological: Autologous Bone Marrow-derived Mononuclear Cells, Device: Insertable cardiac monitorIndividuals with Ebstein anomaly and underlying myopathic right ventricle undergoing planned surgical intervention using an add-on procedure delivering autologous bone marrow-derived mononuclear cells into the right ventricle. |
| Control arm | Other | Device: Insertable cardiac monitorIndividuals with Ebstein anomaly and underlying myopathic right ventricle undergoing planned surgical intervention without cell delivery. |
Who may qualify
- Ages
- 6 months to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Age 6 months to 30 years
- Individuals clinically planned for elective surgical Ebstein repair
- Individuals able to undergo bone marrow aspirate according to clinical consultation with Hematology (cell treatment group only)
- Individuals able to undergo preoperative MRI or CT examination
- Individual and/or parent willing and able to give informed consent and willing to commit to completion of follow-up
Key exclusion criteria (23 listed)
- Individuals requiring cavopulmonary shunt at the time of surgical Ebstein repair; planned preoperatively or required intraoperatively
- Individuals with, or reasonably expected to have, complications during surgical Ebstein repair or during post-operative recovery
- Individual who have not completed or will not be completing all pre-procedure work-up within 30 days of surgical Ebstein repair AND lack of pre-procedure work-up documented as a safety concern by a site investigator
- Individuals who have other clinical concerns as documented by a site investigator that could reasonably increase the risk of complications during or after surgical Ebstein repair
- Individuals whose cells have been determined, by the sponsor, to not be acceptable for release to the investigational site or individual whose cells have been compromised after cells released to investigational site (cell treatment group only)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Bone Marrow-derived Mononuclear CellsAlso: MNC | Biological | Treatment arm |
| Insertable cardiac monitorAlso: ICM, ILRFollowing surgical Ebstein repair a Medtronic Reveal LINQ Insertable Cardiac Monitor (ICM) will be placed subcutaneously in the anterior chest to continuously monitor the subject's heart rhythm and to record cardiac information through automatic detection of arrhythmias. | Device | Control arm, Treatment arm |
Primary outcome
Number of adverse events from time of index procedure
Time frame: 24 months post index procedure
Safety assessment of adverse events from time of index procedure will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Analysis of cardiac adverse events is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Severity of adverse events from time of index procedure
Time frame: 24 months post index procedure
Safety assessment of severity of adverse events from time of index procedure will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Number of subjects who died
Time frame: 24 months post index procedure
Safety assessment of all-cause mortality will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Number of subjects with sustained symptomatic cardiac arrhythmias
Time frame: 24 months post index procedure
Safety assessment of sustained symptomatic cardiac arrhythmias will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Number of subjects with myocardial infarction
Time frame: 24 months post index procedure
Safety assessment of myocardial infarctions will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Number of subjects with unexpected, invasive cardiovascular procedures
Time frame: 24 months post index procedure
Safety assessment of unexpected, invasive cardiovascular procedures will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Number of serious adverse events
Time frame: 24 months post index procedure
Safety assessment of serious adverse events will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Percentage of subjects in the treatment group that have cells delivered
Time frame: 24 months post index procedure
Feasibility of the BM-MNC cell therapy will be evaluated comparing number of subjects enrolled in the treatment group to subjects in the treatment group who are accrued and have cells delivered by percentage using the binomial distribution and exact confidence limits given.
Percentage of subjects in the treatment group completing the 24 month follow-up
Time frame: 24 months post index procedure
Feasibility of the BM-MNC cell therapy will be evaluated comparing number of subjects enrolled in the treatment group to subjects in the treatment group who are accrued and have cells delivered, who complete the 24 month follow-up visit by percentage.
Incidence of cardiac related hospitalizations from time of Ebstein repair
Time frame: 1, 6, and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Incidence of cardiac arrhythmias from time of index procedure
Time frame: 1, 6, and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in CT derived right ventricular dimensions from time of pre-operative evaluation
Time frame: 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. End volume measurements recorded using mL and indexed to body surface area (BSA) using mL/m^2.A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will be used to distinguish common rates from very rare rates. Analysis is in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in echocardiography derived cardiac output from time of pre-operative evaluation
Time frame: 1, 6, and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group.Volume is measured in L/min and Volume Index is L/min/m^2.Using t-test will have 80% power to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group to distinguish common rates from very rare rates. Analysis in terms of yes/no information at 24 months. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in NT-Pro-BNP derived cardiac function trend from time of pre-operative evaluation
Time frame: During hospitalization up to 29 days, 1, 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Incidence of cardiac related hospitalizations in the treatment group from time of pre-operative evaluation compared to the control group
Time frame: 1, 6, and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Incidence of cardiac arrhythmias in the treatment group from time of pre-operative evaluation compared to the control group
Time frame: 1, 6, and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in MRI derived cardiac output in the treatment group from time of pre-operative evaluation compared to the control group
Time frame: 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group.Volume is measured in L/min and Volume Index is L/min/m^2.Using t-test will have 80% power to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group to distinguish common rates from very rare rates. Analysis in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in echocardiography derived right ventricular dimensions from time of pre-operative evaluation
Time frame: 1, 6, and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. End volume measurements recorded using mL and indexed to body surface area (BSA) using mL/m^2,diameter as cm.A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will be used to distinguish common rates from very rare rates. Analysis is in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in NT-Pro-BNP derived cardiac function trend in the treatment group from time of pre-operative evaluation compared to the control group
Time frame: During hospitalization up to 29 days, 1, 6, and 24 month post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Number of serious adverse events from time of enrollment
Time frame: index procedure
Safety assessment of the number of serious adverse events from time of enrollment will be analyzed by counts and percents using the chi-square test and Cochran-Armitage test for trends, and the two groups compared using the Fisher exact test. A Fisher's exact test with a 0.05 two-sided significance level will have 80% power to detect a difference in a sample size of 10 in each group. Thus having reasonable power to distinguish common rates for adverse events from very rare rates. Subjects will be censored upon lost to follow up. If some subjects are lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method and comparison between the two groups using the log-rank test. A confidence interval for the difference between groups will be given.
Percentage of subjects in the treatment group whose cells meet all release criteria
Time frame: 24 months post index procedure
Feasibility of the BM-MNC cell therapy will be evaluated comparing number of subjects enrolled in the treatment group to subjects in the treatment group with collected bone marrow and the bone marrow cells have met all product release criteria to any products that did not meet release criteria by percentage.
Change in CT derived cardiac output from time of pre-operative evaluation
Time frame: 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group.Volume is measured in L/min and Volume Index is L/min/m^2.Using t-test will have 80% power to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group to distinguish common rates from very rare rates. Analysis in terms of yes/no information at 24 months. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in MRI derived right ventricle ejection fraction in the treatment group from time of pre-operative evaluation compared to the control group
Time frame: 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group measured in %.Using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in MRI derived right ventricular dimensions in the treatment group from time of pre-operative evaluation compared to the control group
Time frame: 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group. End volume measurements recorded using mL and indexed to body surface area (BSA) using mL/m^2.A sample size of 10 in each group using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will be used to distinguish common rates from very rare rates. Analysis is in terms of yes/no information at 24 months.If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Change in CT derived right ventricle ejection fraction from time of pre-operative evaluation
Time frame: 6 and 24 months post index procedure
Efficacy will be evaluated using analysis of covariance (ANCOVA) from baseline to 24 months post-index procedure comparing outcomes between the treatment group to the control group measured in %.Using t-test will have 80% power to detect an effect size of 1.325 with a 0.050 two-sided significance level to identify a difference between means from the two groups. Descriptive statistics such as mean, median, SD, min, max, and 95% confidence intervals of the mean, and frequency distributions will be calculated for all variables by group. Fisher exact test will have 80% power to detect a difference in a sample size of 10 in each group having reasonable power to distinguish common rates from very rare rates. Analysis is in terms of the yes/no information at 24 months. Subjects will be censored upon lost to follow up. If a subject is lost to follow-up then fractions at 24 months will be described using the Kaplan-Meier method.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Apr 2022.
- Derived from this study: PubMed 34479739O'Leary PW, Qureshi MY, Cetta F, Nelson TJ, Holst KA, Dearani JA; Wanek Program Clinical Pipeline Group. Cone Reconstruction for Ebstein Anomaly: Ventricular Remodeling and Preliminary Impact of Stem Cell Therapy. Mayo Clin Proc. 2021 Dec;96(12):3053-3061. doi: 10.1016/j.mayocp.2021.02.015. Epub 2021 Sep 1.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Ebstein Anomaly
RecruitingObservational study
Fetal Ebstein Anomaly and Tricuspid Valve Dysplasia Registry
Ebstein Anomaly, Tricuspid Valve Dysplasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Muhammad Y Qureshi, MBBS principal investigator
Mayo Clinic
Timothy J Nelson, MD, PhD study director
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Bone Marrow-derived Mononuclear Cells and Insertable cardiac monitor in people with Ebstein Anomaly, run by Timothy J. Nelson at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events from time of index procedure (time frame: 24 months post index procedure). The study lists 25 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02914171, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Apr 2022.