Stem Cell Therapy for Congenital Heart Disease

Not currently recruiting at 5 trial locations

Overseen By Lynn Padley

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: Timothy J Nelson, MD, PhD

Disqualifiers: Cancer, Severe heart failure, Arrhythmia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to observe changes in the heart when autologous mononuclear cells are injected during the Fontan surgery. Researchers are investigating whether these stem cells can enhance the heart's electrical signals, structure, and function. Participants will be divided into two groups: one receiving the stem cells and one not. Children with a congenital heart defect involving a single right ventricle who are scheduled for the Fontan surgery may be suitable candidates. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that using a person's own cells from their umbilical cord blood is generally safe. In past studies, patients with heart conditions who received these cells tolerated the treatment well and did not experience serious side effects.

These studies involved injecting the cells directly into the heart muscle, similar to the method tested in this trial. The FDA's approval of similar treatments for other conditions further suggests their safety. However, as this is an early-stage trial, the primary goal is to confirm safety before evaluating effectiveness. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for congenital heart disease, which often involve surgical interventions and medications to manage symptoms, the innovative approach of using autologous mononuclear cells offers a regenerative angle. These cells are derived directly from a patient's own umbilical cord blood and injected into the heart muscle, potentially promoting natural repair and growth. This method not only minimizes the risk of rejection, as the cells are from the patient, but also harnesses the body's innate healing processes, offering hope for more effective and lasting heart repair.

What evidence suggests that this stem cell therapy might be an effective treatment for congenital heart disease?

This trial will compare the effects of autologous mononuclear cells derived from umbilical cord blood with a control group not receiving the cell product. Research has shown that using cells from one's own umbilical cord blood might improve heart function. Specifically, previous patients experienced long-term benefits in heart health and development after similar treatments. Another study examined safety and found these cells safe to use, with no major heart-related problems. However, some research did not find short-term improvements in heart strain, indicating mixed results. Overall, the therapy appears promising, especially for long-term heart health improvements. ^{1 2 4 6 7}

Who Is on the Research Team?

Joseph W Rossano, M.D.

Principal Investigator

Children's Hospital of Philadelphia

Waldemer Carlo, M.D.

Principal Investigator

University of Alabama at Birmingham

James Tweddell, M.D.

Principal Investigator

Children's Hospital Medical Center, Cincinnati

John D Cleveland, MD

Principal Investigator

Children's Hospital Los Angeles

James Jaggers, M.D.

Principal Investigator

Children's Hospital Colorado

Benjamin Peeler, MD

Principal Investigator

Ochsner Health System

Jason Maynes, MD

Principal Investigator

The Hospital forSick Children

Joseph Dearani, M.D

Principal Investigator

Mayo Clinic

Harold M Burkhart, M.D.

Principal Investigator

Children's Hospital Oklahoma University Medical Center

David M Overman, M.D.

Principal Investigator

Children's Minnesota

Are You a Good Fit for This Trial?

This trial is for children aged 2-5 with a specific heart defect (like HLHS) who are undergoing Fontan surgery. They must have previously participated in a related clinical trial and have their own stem cells available. Kids can't join if they've had severe reactions to certain preservatives, other serious chronic diseases, cancer, recent urgent procedures, or conditions that could worsen their outcome.

Inclusion Criteria

I have a single functioning right ventricle and am undergoing Fontan surgery.

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.

I had the Fontan surgery between 2 and 5 years old.

Exclusion Criteria

I need a heart transplant due to severe heart failure.

I haven't had any emergency procedures within 15 days before my Fontan surgery, or if I did, I've fully recovered.

Your doctor has other health concerns that could make the treatment risky for you.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Intramyocardial injection of autologous UCB-MNC during Fontan surgery

1 day

1 visit (in-person)

Short-term Follow-up

Participants are monitored for safety and effectiveness after treatment, including hospital discharge and 3-month assessments

3 months

Multiple visits (in-person and virtual)

Long-term Follow-up

Participants are monitored for long-term safety and effectiveness, with assessments at 12, 18, and 24 months post-surgery

24 months

Periodic visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

• Autologous Mononuclear Cells

Trial Overview The study tests the effects of injecting autologous mononuclear cells directly into the heart muscle during Fontan surgery on kids with single right ventricle defects. It aims to see if this treatment improves heart structure and function compared to those not receiving stem cells.

How Is the Trial Designed?

Group I: Treatment Arm Experimental Treatment 1 Intervention

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.

Group II: Control Arm Active Control 1 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 Is Running the Clinical Trial?

Timothy J Nelson, MD, PhD

Lead Sponsor

Trials

5

Recruited

170+

Children's of Alabama

Collaborator

Trials

10

Recruited

3,000+

University of Oklahoma

Collaborator

Trials

484

Recruited

95,900+

Children's Hospital of Philadelphia

Collaborator

Trials

749

Recruited

11,400,000+

Children's Hospital Los Angeles

Collaborator

Trials

257

Recruited

5,075,000+

Children's Hospitals and Clinics of Minnesota

Collaborator

Trials

67

Recruited

5,022,000+

Children's Hospital Colorado

Collaborator

Trials

121

Recruited

5,135,000+

Ochsner Health System

Collaborator

Trials

97

Recruited

91,900+

Children's Hospital Medical Center, Cincinnati

Collaborator

Trials

844

Recruited

6,566,000+

The Hospital for Sick Children

Collaborator

Trials

724

Recruited

6,969,000+

Published Research Related to This Trial

The study successfully demonstrated the feasibility and safety of transplanting autologous umbilical cord blood mononuclear cells (UCB-MNCs) into the right ventricle of juvenile porcine hearts, with no mortalities and minimal complications observed over a 3-month monitoring period.

The results indicate that UCB-MNCs can be safely delivered in a pediatric setting, providing a promising foundation for future clinical trials aimed at using cell-based therapies for congenital heart diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and feasibility for pediatric cardiac regeneration using epicardial delivery of autologous umbilical cord blood-derived mononuclear cells established in a porcine model system. Cantero Peral, S., Burkhart, HM., Oommen, S., et al.[2018]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12044795/

Autologous umbilical cord blood mononuclear cell therapy ... There was an unfavorable outcome regarding cardiac function, as the product failed to improve right ventricular cardiac strain in the short and ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40312733/

Autologous umbilical cord blood mononuclear cell therapy ... Autologous umbilical cord blood mononuclear cell therapy for hypoplastic left heart syndrome: a nonrandomized control trial of the efficacy and ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03779711

Study Details | NCT03779711 | Intramyocardial Injection of ... Long-term improvement in cardiac function, reaching growth and developmental milestones, reaching Stage III surgical repair pre-op work-up, prolonging time to ...

4. stemcellres.biomedcentral.com stemcellres.biomedcentral.com/articles/10.1186/s13287-015-0044-y

Human umbilical cord blood-derived mononuclear cells ... Herein, studies were performed to evaluate the cardiovascular safety and beneficial effect of mononuclear cells (MNCs) isolated from human ...

5. thelancet.com thelancet.com/journals/ebiom/article/PIIS2352-3964(24)00528-0/fulltext

Feasibility and safety of autologous cord blood derived cell ... This phase-1 clinical trial has shown UCBC collection and reinfusion was feasible in approximately 70% of extremely preterm infants and was well ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01883076

Safety Study of Autologous Umbilical Cord Blood Cells for ... This study is a Phase I trial to determine the safety of autologous mononuclear cells (MNC) derived from umbilical cord blood for intramyocardial delivery into ...

7. mayo.edu mayo.edu/research/clinical-trials/cls-20112829

A Study of Autologous Umbilical Cord Blood Derived ... This is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of ...