Stem Cell Therapy for Congenital Heart Disease

Recruiting in Palo Alto (17 mi)

+5 other locations

Overseen byWaldemer Carlo, M.D.

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Timothy J Nelson, MD, PhD

No Placebo Group

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

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.

+9 more

Participant Groups

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.

2Treatment groups

Experimental Treatment

Active Control

Group I: Treatment ArmExperimental Treatment1 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 ArmActive Control1 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.