Congenital Heart Disease, SRV DependentPhase 1NCT04907526
Intramyocardial Injection of Autologous UCB-MNC During Fontan Surgery for SRV Dependent CHD
A Phase 1 study testing Autologous mononuclear cells in people with Congenital Heart Disease, SRV Dependent, run by Timothy J Nelson, MD, PhD at 6 sites in United States.
Trial summary
Researchers want to better understand what happens to the heart when the autologous (from one's own body) stem cells are injected directly into muscle of the right side of the heart during the Fontan (Stage III) surgery. Read more about this study
- Who is it for?
- Ages 2 to 5 years
- Conditions the study is for
- Congenital Heart Disease, SRV Dependent
- What is being tested?
- Autologous mononuclear cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 6 sitesUnited States
- Study dates
- 1 Jun 2021to 30 Aug 2025
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of a congenital hear defect with functionally single right ventricle (such as HLHS, HLHS variants, unbalanced AV septal defect with R dominance, DORV with Hypoplastic LV) undergoing Fontan surgical palliation
- At least 2 and less than or equal to 5 years of age at time of Fontan surgical palliation
- For subjects enrolling in the treatment arm, previous participation in clinical trial Umbilical Cord Blood Collection and Processing for Hypoplastic Left Heart Syndrome patients (NCT01856049) with autologous UCB-MNC product collected and available for distribution is required.
Who cannot join
- History of DMSO reaction (treatment arm only subjects).
- Parent(s) or legal guardian unwilling to have their child participate or unwilling to follow the study procedures.
- Severe chronic diseases at the discretion of the treating physician.
- Extensive extra-cardiac syndromic features.
- History of cancer.
- Any of the following complications of his/her congenital heart disease:
Show all 13Show fewer
- any condition requiring urgent, or unplanned intervention procedure within 15 days prior to Fontan surgical palliation, unless complete and full cardiac recovery is documented by site investigator.
- severe pulmonary hypertension (reported in the medical record as >70% systemic pressure)
- Other clinical concerns as documented by a site investigator that would predict (more likely to happen than not to happen) a risk of severe complications or very poor outcome, not directly related tot he stem cell product or its injection procedure, during or after Fontan surgical palliation.
- Individuals with severe heart failure that requires heart transplantation
- Individuals with refractory or worsening arrhythmia
- Individuals with an automated implantable cardioverter defibrillator (AICD) or pacemaker
- Patient with prior surgical complications during the Fontan surgical palliation that resulted in or could be reasonably expected to significantly decrease cardiac function
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
University of Alabama Medical Center
Birmingham, Alabama, 35233
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of Oklahoma Medical Center
Oklahoma City, Oklahoma, 73104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
About this study
Official title: Phase I Study of Intramyocardial Injection of Autologous Umbilical Cord Blood-Derived Mononuclear Cells During Fontan Surgical Palliation of Single Right Ventricle-Dependent Congenital Heart Disease
Researchers want to better understand what happens to the heart when the autologous (from one's own body) stem cells are injected directly into muscle of the right side of the heart during the Fontan (Stage III) surgery. They want to see if there are changes in the electrical activity, the structure, and the function of the heart following this stem cell-based therapy. Researchers will compare the results from people who receive the stem cells to the results from people who do not receive the stem cells.
Read the detailed description
This Phase I study is a multicenter, prospective, open-label, non-randomized study designed to evaluate the safety, of autologous UCB-MNC delivered into the right ventricular myocardium of subjects with severe CHD defined by single right ventricular dependent circulatory systems at the time of a planned Fontan surgical palliation. This will be achieved by comparing the data collected in the treatment arm to the equivalent data collected in the control arm. The purpose of this non-randomized open-label Phase I clinical study is to prospectively evaluate the safety, as measured by the short-term and long-term safety endpoints and change in baseline comparatives, for the autologous UCB-MNC intramyocardial injections into the single, morphologically right ventricle of subjects with severe CHD requiring Fontan surgical palliation. The treatment group will be compared to an untreated control group.
- Conditions
- Congenital Heart Disease, SRV Dependent
- Being tested
- Autologous mononuclear cells
- Keywords
- Hypoplastic Left Heart Syndrome, HLHS, Congenital Heart Disease, Umbilical Cord Blood, UCB, Cord Blood, Stem Cells, Regenerative therapy, Stage III Fontan, Fontan surgery
- Medical subject headings
- Heart Defects, Congenital, Hypoplastic Left Heart Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous mononuclear cellsBiological
Autologous mononuclear cells delivered into right ventricle at time of Stage III Fontan surgery.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | Autologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 3 million cells per kilogram of body weight. This is a one time treatment at the time of Stage III Fontan surgery. Receives: Biological: Autologous mononuclear cells |
| Control Arm | No intervention | Control cohort not receiving the cell product, which will be enrolled and followed using the same inclusion/exclusion criteria and follow-up requirements as the treatment arm. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Short-term safety
Measure of new or worsening adverse events
Measured: Within 3 months post Fontan surgery
Secondary outcomes (8)
Long term safety
Measure of new or worsening adverse events
Measured: Within 2 years post Fontan surgery
Right ventricular function
Change from baseline Transthoracic Echocardiogram right ventricular function as measured by biplane Fractional Area Change (FAC) at discharge and 3 months
Measured: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery
High sensitivity Troponin T
Change from baseline in High sensitivity Troponin T at 3 hours and 6 hours after enrollment and at hospital discharge
Measured: 3 hours post enrollment, 6 hours post enrollment, hospital discharge (up to 30 days post Fontan surgery)
NT-pro-BNP
Change from baseline in NT-pro-BNP levels compared to baseline, at hospital discharge and at 3 months post-Fontan surgery
Measured: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery
Panel Reactive Antibody
Change from baseline in PRA levels at discharge and 3 months post-Fontan surgery compared to baseline.
Measured: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery
Weight
Change from baseline in weight at 3 and 12 months post-Fontan surgery.
Measured: Baseline, 3 months post-Fontan surgery, 12 months post-Fontan surgery
Cumulative days hospitalization
Cumulative days of hospitalization per patient at 3 and 12 months post-Fontan surgery discharge
Measured: 3 months post discharge, 12 months post discharge
PROMIS Parent Proxy Scale v1.0-Global Health 7
Change from baseline in PROMIS Parent Proxy Scale v1.0-Global Health 7 at discharge, 3, 12, 18, and 24 months post-Fontan surgery
Measured: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months, 12 months, 18 months, 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2021
- Study start1 Jun 2021
- Primary completion24 May 2023
- Study completion30 Aug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Heart Disease, SRV DependentStudies 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
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Timothy J Nelson, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Mayo Clinic, University of Oklahoma, Children's Hospital of Philadelphia, Children's Hospital Los Angeles, Children's Hospitals and Clinics of Minnesota, Children's Hospital Colorado, Ochsner Health System, Children's of Alabama, Children's Hospital Medical Center, Cincinnati, The Hospital for Sick Children
- Responsible party
- Sponsor-investigator, Timothy J Nelson, MD, PhD, ReGen Theranostics, Inc.
- Investigators
- Joseph Dearani, Principal investigator, Mayo ClinicHarold M Burkhart, Principal investigator, Children's Hospital Oklahoma University Medical CenterJoseph w Rossano, Principal investigator, Children's Hospital of PhiladelphiaDavid M Overman, Principal investigator, Children's MinnesotaJohn D Cleveland, MD, Principal investigator, Children's Hospital Los AngelesJames Jaggers, MD, Principal investigator, Children's Hospital ColoradoBenjamin Peeler, MD, Principal investigator, Ochsner Health SystemWaldemer Carlo, M.D., Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04907526
- Sponsor's protocol ID
- 21-000088
- Status last verified
- Jun 2025
- First submitted
- 12 May 2021
- Last updated
- 27 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Intramyocardial Injection of Autologous Umbilical Cord Blood-Derived Mononuclear Cells During Fontan Surgical Palliation of Single Right Ventricle-Dependent Congenital Heart Disease
- ClinicalTrials.gov ID
- NCT04907526
- Acronym / sponsor ID
- 21-000088
- Conditions
- Congenital Heart Disease, SRV Dependent
- Keywords
- Hypoplastic Left Heart Syndrome, HLHS, Congenital Heart Disease, Umbilical Cord Blood, UCB, Cord Blood, Stem Cells, Regenerative therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Timothy J Nelson, MD, PhDUniversity, hospital or other
- Collaborators
- Mayo Clinic, University of Oklahoma, Children's Hospital of Philadelphia, Children's Hospital Los Angeles, Children's Hospitals and Clinics of Minnesota, Children's Hospital Colorado, Ochsner Health System, Children's of Alabama, Children's Hospital Medical Center, Cincinnati, The Hospital for Sick Children
- Study start
- 1 Jun 2021 (actual)
- Primary completion
- 24 May 2023 (actual)
- Study completion
- 30 Aug 2025 (estimated)
- First posted
- 28 May 2021 (actual)
- 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.
Researchers want to better understand what happens to the heart when the autologous (from one's own body) stem cells are injected directly into muscle of the right side of the heart during the Fontan (Stage III) surgery. They want to see if there are changes in the electrical activity, the structure, and the function of the heart following this stem cell-based therapy. Researchers will compare the results from people who receive the stem cells to the results from people who do not receive the stem cells.
Detailed description
This Phase I study is a multicenter, prospective, open-label, non-randomized study designed to evaluate the safety, of autologous UCB-MNC delivered into the right ventricular myocardium of subjects with severe CHD defined by single right ventricular dependent circulatory systems at the time of a planned Fontan surgical palliation. This will be achieved by comparing the data collected in the treatment arm to the equivalent data collected in the control arm. The purpose of this non-randomized open-label Phase I clinical study is to prospectively evaluate the safety, as measured by the short-term and long-term safety endpoints and change in baseline comparatives, for the autologous UCB-MNC intramyocardial injections into the single, morphologically right ventricle of subjects with severe CHD requiring Fontan surgical palliation. The treatment group will be compared to an untreated control group.
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 mononuclear cellsAutologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 3 million cells per kilogram of body weight. This is a one time treatment at the time of Stage III Fontan surgery. |
| Control Arm | No intervention | –Control cohort not receiving the cell product, which will be enrolled and followed using the same inclusion/exclusion criteria and follow-up requirements as the treatment arm. |
Who may qualify
- Ages
- 2 to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of a congenital hear defect with functionally single right ventricle (such as HLHS, HLHS variants, unbalanced AV septal defect with R dominance, DORV with Hypoplastic LV) undergoing Fontan surgical palliation
- At least 2 and less than or equal to 5 years of age at time of Fontan surgical palliation
- For subjects enrolling in the treatment arm, previous participation in clinical trial Umbilical Cord Blood Collection and Processing for Hypoplastic Left Heart Syndrome patients (NCT01856049) with autologous UCB-MNC product collected and available for distribution is required.
Key exclusion criteria (13 listed)
- History of DMSO reaction (treatment arm only subjects).
- Parent(s) or legal guardian unwilling to have their child participate or unwilling to follow the study procedures.
- Severe chronic diseases at the discretion of the treating physician.
- Extensive extra-cardiac syndromic features.
- History of cancer.
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 mononuclear cellsAutologous mononuclear cells delivered into right ventricle at time of Stage III Fontan surgery. | Biological | Treatment Arm |
Primary outcome
Short-term safety
Time frame: Within 3 months post Fontan surgery
Measure of new or worsening adverse events
8 secondary outcomes
Long term safety
Time frame: Within 2 years post Fontan surgery
Right ventricular function
Time frame: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery
High sensitivity Troponin T
Time frame: 3 hours post enrollment, 6 hours post enrollment, hospital discharge (up to 30 days post Fontan surgery)
NT-pro-BNP
Time frame: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery
Panel Reactive Antibody
Time frame: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery
Weight
Time frame: Baseline, 3 months post-Fontan surgery, 12 months post-Fontan surgery
Cumulative days hospitalization
Time frame: 3 months post discharge, 12 months post discharge
PROMIS Parent Proxy Scale v1.0-Global Health 7
Time frame: Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months, 12 months, 18 months, 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2025.
- Individual participant dataSharing planned: No
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.
Joseph Dearani principal investigator
Mayo Clinic
Harold M Burkhart principal investigator
Children's Hospital Oklahoma University Medical Center
Joseph w Rossano principal investigator
Children's Hospital of Philadelphia
6 sites 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 mononuclear cells in people with Congenital Heart Disease, SRV Dependent, run by Timothy J Nelson, MD, PhD at 6 sites 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 3 inclusion and 13 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: under way, not looking for participants, as of the sponsor's last update on 27 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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?
This study is still under way. Results are not available in the source record yet.
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?
Short-term safety (time frame: Within 3 months post Fontan surgery). 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 27 Jun 2025.