Congenital Heart DiseasePhase 1NCT02549625
Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells
A Phase 1 study testing Autologous Bone Marrow-derived Mononuclear Cells in people with Congenital Heart Disease, run by Timothy J Nelson, MD, PhD at 1 site in United States.
Trial summary
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) and intracoronary delivery for individuals with declining performance of their single right ventricle systemic pumps. Read more about this study
- Who is it for?
- Ages 2 to 30 years
- Conditions the study is for
- Congenital Heart Disease
- What is being tested?
- Autologous Bone Marrow-derived Mononuclear Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2015to Nov 2020
- 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 2 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Individuals with Fontan circulation with right ventricular dysfunction
- Ejection fraction equal to or less than 40% despite optimal outpatient medical management for at least 3 months
- Able to undergo bone marrow aspirate according to clinical consultation with hematology (INR must be maintained at or below 1.5)
- Able to undergo an MRI or CT examination
Who cannot join
- Individuals or parents of minors unwilling to consent to participation
- Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer
- Females 10 years and older with positive pregnancy test or lack of effective birth control method
- Individuals currently requiring IV inotropes
- Individuals with bleeding disorders or history of thrombosis
- Individuals not eligible for MRI and have elevated serum creatinine level (eGFR less that 45 mL/min) and therefore cannot have a CT examination
Show all 15Show fewer
- Individuals not eligible for MRI and have a prior significant reaction to intravenous contrast required for CT examination
- Individuals with the following conditions within 60 days prior to procedure:
- Cardiogenic shock or extracorporeal circulation;
- New arrhythmia that required medication for control;
- Documented infection requiring treatment with antibiotics, and/or current infection being treated with antibiotics;
- Cardiac condition requiring emergency procedure;
- Cardiovascular surgery;
- Seizures or history of significant neurological injury;
- Multi-system organ failure including acute or chronic renal failure
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells for Systemic, Single Right Ventricular Failure Due to Congenital Heart Disease
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) and intracoronary delivery for individuals with declining performance of their single right ventricle systemic pumps. This procedure has the potential to foster a new strategy for congenital heart patients. This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) related serious adverse events 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 delivered into the coronary circulation of subjects with Fontan circulation and right ventricular dysfunction. Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial. Once informed consent has been obtained; preoperative values will be established and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria. This will require a non-study cardiologist to review the clinical case and confirm the baseline cardiac function with evidence of disease progression with right ventricle dysfunction. However, individuals with decreased cardiac function requiring inotropic support and immediate listing for cardiac transplantation will NOT be included such that the risk of this research procedure may not be acceptable. Following cell-based product delivery, subjects will be followed for 6-months according to a pre-determined schedule that includes imaging studies along with questionnaires, electrophysiology and laboratory studies. Subjects will be contacted for an additional 18-months for surveillance by phone.
- Conditions
- Congenital Heart Disease
- Being tested
- Autologous Bone Marrow-derived Mononuclear Cells
- Keywords
- Single right ventricular failure
- Medical subject headings
- Heart Defects, Congenital
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
| Study group | Role | What they receive |
|---|---|---|
| Single-arm | Experimental | Intracoronary delivery of autologous bone marrow-derived mononuclear cells using catheterization procedure Receives: Biological: Autologous Bone Marrow-derived Mononuclear Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of related serious adverse events
Measured: 6 months post cell delivery
Secondary outcomes (12)
Number of subjects who died
Measured: 6 months post cell delivery
Number of subjects with sustained or symptomatic ventricular arrhythmias
Measured: 6 months post cell delivery
Number of subjects with acute decompensated heart failure
Measured: 6 months post cell delivery
Number of subjects with myocardial infarction
Measured: 6 months post cell delivery
Number of subjects with cardiac infection
Measured: 6 months post cell delivery
Number of subjects with unexpected cardiovascular procedures within 6 months following cellular transplantation
Measured: 6 months post cell delivery
Percentage of subjects that have cells delivered
Measured: 6 months post cell delivery
Percentage of subjects completing the 6-month follow-up
Measured: 6 months post cell delivery
Change in CT/MRI derived right ventricle ejection fraction
Measured: Baseline, 6 months post cell delivery
Change in diastolic ventricular volume
Measured: Baseline, 6 months post cell delivery
Change in systolic ventricular volume
Measured: Baseline, 6 months post cell delivery
Change in echocardiography derived right ventricle ejection fraction
Measured: Baseline, 6 months post cell delivery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2015 (estimated)
- Study startAug 2015
- Primary completionNov 2020
- Study completionNov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Heart DiseaseStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Timothy J Nelson, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Mayo Clinic
- Responsible party
- Sponsor-investigator, Timothy J Nelson, MD, PhD, ReGen Theranostics, Inc.
- Investigators
- Timothy J Nelson, M.D., Ph.D., Study director, Mayo ClinicMuhammad Y Qureshi, MBBS, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02549625
- Sponsor's protocol ID
- 13-006427
- Status last verified
- May 2021
- First submitted
- 10 Sep 2015
- Last updated
- 14 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells for Systemic, Single Right Ventricular Failure Due to Congenital Heart Disease
- ClinicalTrials.gov ID
- NCT02549625
- Acronym / sponsor ID
- 13-006427
- Conditions
- Congenital Heart Disease
- Keywords
- Single right ventricular failure
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Timothy J Nelson, MD, PhDUniversity, hospital or other
- Collaborators
- Mayo Clinic
- Study start
- Aug 2015
- Primary completion
- Nov 2020 (actual)
- Study completion
- Nov 2020 (actual)
- First posted
- 15 Sep 2015 (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 (MNC) collected from bone marrow (BM) and intracoronary delivery for individuals with declining performance of their single right ventricle systemic pumps. This procedure has the potential to foster a new strategy for congenital heart patients. This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) related serious adverse events 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 delivered into the coronary circulation of subjects with Fontan circulation and right ventricular dysfunction. Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial. Once informed consent has been obtained; preoperative values will be established and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria. This will require a non-study cardiologist to review the clinical case and confirm the baseline cardiac function with evidence of disease progression with right ventricle dysfunction. However, individuals with decreased cardiac function requiring inotropic support and immediate listing for cardiac transplantation will NOT be included such that the risk of this research procedure may not be acceptable. Following cell-based product delivery, subjects will be followed for 6-months according to a pre-determined schedule that includes imaging studies along with questionnaires, electrophysiology and laboratory studies. Subjects will be contacted for an additional 18-months for surveillance by phone.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single-arm | Experimental | Biological: Autologous Bone Marrow-derived Mononuclear CellsIntracoronary delivery of autologous bone marrow-derived mononuclear cells using catheterization procedure |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Individuals with Fontan circulation with right ventricular dysfunction
- Ejection fraction equal to or less than 40% despite optimal outpatient medical management for at least 3 months
- Able to undergo bone marrow aspirate according to clinical consultation with hematology (INR must be maintained at or below 1.5)
- Able to undergo an MRI or CT examination
Key exclusion criteria (15 listed)
- Individuals or parents of minors unwilling to consent to participation
- Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer
- Females 10 years and older with positive pregnancy test or lack of effective birth control method
- Individuals currently requiring IV inotropes
- Individuals with bleeding disorders or history of thrombosis
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 | Single-arm |
Primary outcome
Number of related serious adverse events
Time frame: 6 months post cell delivery
12 secondary outcomes
Number of subjects who died
Time frame: 6 months post cell delivery
Number of subjects with sustained or symptomatic ventricular arrhythmias
Time frame: 6 months post cell delivery
Number of subjects with acute decompensated heart failure
Time frame: 6 months post cell delivery
Number of subjects with myocardial infarction
Time frame: 6 months post cell delivery
Number of subjects with cardiac infection
Time frame: 6 months post cell delivery
Number of subjects with unexpected cardiovascular procedures within 6 months following cellular transplantation
Time frame: 6 months post cell delivery
Percentage of subjects that have cells delivered
Time frame: 6 months post cell delivery
Percentage of subjects completing the 6-month follow-up
Time frame: 6 months post cell delivery
Change in CT/MRI derived right ventricle ejection fraction
Time frame: Baseline, 6 months post cell delivery
Change in diastolic ventricular volume
Time frame: Baseline, 6 months post cell delivery
Change in systolic ventricular volume
Time frame: Baseline, 6 months post cell delivery
Change in echocardiography derived right ventricle ejection fraction
Time frame: Baseline, 6 months post cell delivery
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 14 May 2021.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Timothy J Nelson, M.D., Ph.D. study director
Mayo Clinic
Muhammad Y Qureshi, MBBS principal investigator
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 in people with Congenital Heart Disease, run by Timothy J Nelson, MD, PhD 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 4 inclusion and 15 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 14 May 2021. 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 related serious adverse events (time frame: 6 months post cell delivery). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 May 2021.