← Back to Search

Stem Cell Therapy

c-kit+ Cells for Hypoplastic Left Heart Syndrome (CHILD Trial)

Phase 1

Recruiting

Led By Ming-Sing Si, MD

Research Sponsored by University of Miami

Eligibility Criteria Checklist

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

Must have

Subjects with hypoplastic left heart syndrome (all types) requiring Stage I Norwood operation.

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

CHILD Trial Summary

This trial will test if it is safe to inject c-kit+ cells into the heart during Stage II BDCPA surgery, and if doing so has any positive effects on clinical outcome measures.

Who is the study for?

This trial is for children with a heart condition called Hypoplastic Left Heart Syndrome who need the Stage I Norwood operation. It's not for kids without this specific syndrome, those needing immediate heart support before Stage II surgery, or if their mother has certain infections like HIV or hepatitis.Check my eligibility

What is being tested?

The study tests injecting c-kit+ cells directly into the heart muscle during a pediatric heart surgery (Stage II BDCPA) to see if it helps improve heart function, reduces complications, and improves overall health outcomes in children with Hypoplastic Left Heart Syndrome.See study design

What are the potential side effects?

Since this is an early-stage study focusing on feasibility and safety, potential side effects are being closely monitored but may include issues related to the injection procedure itself and reactions from the body to these new cells.

CHILD Trial Eligibility Criteria

Inclusion Criteria

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

Select...

I have hypoplastic left heart syndrome and need a Stage I Norwood operation.

CHILD Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in Right Ventricular End-diastolic Volume

Change in Right Ventricular End-systolic Volume

Change in Tricuspid Regurgitation

+4 more

Secondary outcome measures

Change in Infant Toddler Quality of Life Survey (ITQOL)

Change in somatic growth velocity

Transplantation

+1 more

Side effects data

From 2020 Phase 2 trial • 125 Patients • NCT02501811

15%

Ventricular tachycardia

Cardiac failure acute

Upper respiratory tract infection

Cardiac failure

Hyperglycaemia

Angina pectoris or angina unstable

Atrial fibrillation

Bronchitis

Respiratory failure

Thrombocytopenia

Lung neoplasm malignant

Septic shock

Head injury

Anaemia or Iron Deficiency Anaemia

Cardiac failure chronic

Cardiac tamponade

Hepatocellular injury

Enterocolitis infectious

Cholelithiasis

Vascular access site pseudoaneurysm

Acute kidney injury

Rash

Hidradenitis

Anaemia

Ventricular arrhythmia

Pneumonia

Joint dislocation

Bladder transitional cell carcinoma

Syncope

Urinary bladder haemorrhage

Chronic obstructive pulmonary disease

Hypoxia

Pulmonary embolism

Ventricular assist device insertion

Cerebrovascular accident

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

MSCs + CPCs

MSCs

CPCs

CHILD Trial Design

3Treatment groups

Experimental Treatment

Active Control

Group I: Open label C-kit+ cells Group AExperimental Treatment1 Intervention

Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.

Group II: C-kit+ cells Group BActive Control1 Intervention

Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.

Group III: No Intervention GroupActive Control1 Intervention

Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

c-kit+ cells

2015

Completed Phase 2

~130

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of MiamiLead Sponsor

900 Previous Clinical Trials

409,900 Total Patients Enrolled

Ming-Sing Si, MDPrincipal InvestigatorMichigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital

Richard Ohye, MDPrincipal InvestigatorMichigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital

Media Library

c-kit+ cells (Stem Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03406884 — Phase 1

Hypoplastic Left Heart Syndrome Research Study Groups: Open label C-kit+ cells Group A, C-kit+ cells Group B, No Intervention Group

Hypoplastic Left Heart Syndrome Clinical Trial 2023: c-kit+ cells Highlights & Side Effects. Trial Name: NCT03406884 — Phase 1

c-kit+ cells (Stem Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03406884 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research endeavor continue to accept new participants?

"Yes, according to clinicaltrials.gov the trial that first launched on October 16th 2019 is actively recruiting participants. 32 patients are expected for this study at 3 different medical sites."

Answered by AI

How many individuals have been included in this research project thus far?

"Affirmative. According to information provided on clinicaltrials.gov, this ongoing medical study was first released in October 16th 2019 and recently updated on September 7th 2022; 32 patients are needed from 3 separate sites for participation purposes."

Answered by AI

Is the FDA aware of c-kit+ cells and their therapeutic potential?

"Taking into account that this is a Phase 1 trial, our group at Power gave c-kit+ cells a score of 1 due to the scarcity of evidence regarding their safety and effectiveness."

Answered by AI

What are the central aims of this investigation?

"The primary purpose of this trial, which is conducted over a 12-month time frame, is to investigate the Number of Incidence of Treatment Related Major Adverse Cardiac Events. Assessing other markers such as Serious Adverse Events (including all-cause mortality and cardiovascular morbidity), Infant Toddler Quality Of Life Survey scores, and somatic growth velocity will additionally be carried out."

Answered by AI

Does this trial offer opportunities for volunteers?

"In this particular trial, 32 neonates diagnosed with hypoplastic left heart syndrome are being accepted. The participants must be between 1 day and 21 days of age and have been identified as requiring the Norwood Stage I operation for their condition."

Answered by AI