The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

NCT03406884 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: 20190743
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 4

Brief Summary

The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.

Conditions

Hypoplastic Left Heart Syndrome

Keywords

stem cells; pediatric

Outcome Measures

Primary Outcomes (7)

• Number of Incidence of Treatment Related Major Adverse Cardiac Events

  Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.

  30 days

• Number of C-kit+ Products

  Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects

  Day 1

• Number of Participants Completing Magnetic Resonance Imaging (MRI)

  Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.

  Baseline, 6 Months, 12 months

• Change in Right Ventricular Function (RVEF)

  Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.

  Baseline, 6 Months, 12 months

• Change in Right Ventricular End-diastolic Volume (RVEDV)

  Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.

  Baseline, 6 months, 12 months

• Change in Right Ventricular End-systolic Volume (RVESV)

  Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.

  Baseline, 6 months, 12 months

• Change in Tricuspid Regurgitation

  Efficacy will be reported as the change tricuspid regurgitation assessed as a percentage and will be measured using serial echocardiograms and MRI scan.

  Baseline, 6 months, 12 months

Secondary Outcomes (6)

• Number of Incidence of Serious Adverse Events

  Up to 12 months

• Change in Somatic Growth Velocity - Length (cm)

  Baseline, 6 months, 12 months

• Change in Somatic Growth Velocity - Weight (kg)

  Baseline, 6 months, 12 months

• Change in Somatic Growth Velocity - Head Circumference (cm)

  Baseline, 6 months, 12 months

• Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health

  Baseline, 12 months

Study Arms (3)

Open label C-kit+ cells Group A

EXPERIMENTAL

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.

Biological: c-kit+ cells

C-kit+ cells Group B

ACTIVE COMPARATOR

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.

Biological: c-kit+ cells

No Intervention Group

NO 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.

Interventions

c-kit+ cells BIOLOGICAL

The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.

Also known as: Autologous c-kit-positive cells (c-kit+ cells)

C-kit+ cells Group B; Open label C-kit+ cells Group A

Eligibility Criteria

• Age: 1 Day - 21 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• Candidates will be excluded from the study if any of the following conditions are met:
• Subjects undergoing the Stage I Norwood operation who do not have HLHS.
• Subjects requiring mechanical circulatory support immediately prior to Stage II BDCPA operation (within 5 days).
• Parent or guardian unwilling or unable to comply with necessary follow-up(s).
• Mother is serum positive for HIV 1/2, hepatitis B surface antigen or viremic hepatitis C and Treponema pallidum.

Sponsors & Collaborators

• Joshua M Hare: lead

Study Sites (4)

Emory University Children's Healthcare of Atlanta - Egleston Campus

Atlanta, Georgia, 30322, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Maryland - Division of Cardiac Surgery

Baltimore, Maryland, 21201, United States

Location

Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

Related Publications (3)

• Kaushal S, Hare JM, Mahle WT, Khan A, Ohye RG, Slesnick TC, Chai PJ, Shashidharan S, Robinson JD, Jone PN, Doman T, Si MS, Lu JC, Bacallao K, Nettina AE, Lamazares R, Saltzman RG, Simpson LM, Li R, Bettencourt JE, Mansoor K, Davis BR, Deatrick KB, Yang J, Mishra R, Everett AD, Lai D, Davis ME. Phase I Randomized Study of Cardiac Stem Cells in Patients With Hypoplastic Left Heart Syndrome: The CHILD Trial. JACC Heart Fail. 2026 Jan;14(1):102723. doi: 10.1016/j.jchf.2025.102723. Epub 2025 Nov 19.

  PMID: 41258851 DERIVED

• Kaushal S, Hare JM, Shah AM, Pietris NP, Bettencourt JL, Piller LB, Khan A, Snyder A, Boyd RM, Abdullah M, Mishra R, Sharma S, Slesnick TC, Si MS, Chai PJ, Davis BR, Lai D, Davis ME, Mahle WT. Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study). Pediatr Cardiol. 2022 Oct;43(7):1481-1493. doi: 10.1007/s00246-022-02872-6. Epub 2022 Apr 8.

  PMID: 35394149 DERIVED

• Ali MK, Ichimura K, Spiekerkoetter E. Promising therapeutic approaches in pulmonary arterial hypertension. Curr Opin Pharmacol. 2021 Aug;59:127-139. doi: 10.1016/j.coph.2021.05.003. Epub 2021 Jun 30.

  PMID: 34217109 DERIVED

Related Links

• Interdisciplinary stem cell institute at the University of Miami website

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome

Condition Hierarchy (Ancestors)

Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Joshua M. Hare, MD
• Organization: University of Miami

jhare@med.miami.edu

305-243-5579

Study Officials

• William Mahle, MD

  Emory University

  PRINCIPAL INVESTIGATOR

• Kristopher Deatrick, MD

  University of Maryland

  PRINCIPAL INVESTIGATOR

• Richard Ohye, MD

  Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Model Details: The first 10 participants will be enrolled in Group A to assess safety and feasibility. An additional 22 participants will be enrolled in Group B and will be randomized to either the Treatment or Control Group.

Study Record Dates

• First Submitted: December 20, 2017
• First Posted: January 23, 2018
• Study Start: October 16, 2019
• Primary Completion: July 1, 2024
• Study Completion: July 1, 2024
• Last Updated: January 23, 2026
• Results First Posted: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)