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Stem Cell Therapy

Stem Cell Therapy for Congenital Heart Disease

Phase 1

Waitlist Available

Led By Jason Maynes, MD

Research Sponsored by Timothy J Nelson, MD, PhD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of a congenital heart 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

Must not have

Individuals with severe heart failure that requires heart transplantation

Individuals with refractory or worsening arrhythmia

Timeline

Screening 3 weeks

Treatment Varies

Follow Up within 2 years post fontan surgery

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether injecting stem cells into the heart during surgery can improve heart function.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects aren't specified but may include typical risks associated with stem cell injections such as immune reactions or complications at the injection site. The procedure's impact will be closely monitored against any adverse changes in cardiac function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I need a heart transplant due to severe heart failure.

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My heart rhythm problem is not improving with treatment.

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I had complications from heart surgery that could worsen my heart function.

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I have had cancer in the past.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ within 2 years post fontan surgery

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~within 2 years post fontan surgery

This trial's timeline: 3 weeks for screening, Varies for treatment, and within 2 years post fontan surgery for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Short-term safety

Secondary study objectives

Cumulative days hospitalization

High sensitivity Troponin T

Long term safety

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

0 / 6Eligibility criteria met