The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

NCT ID: NCT03406884

Last Updated: 2026-01-23

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-16

Study Completion Date

2024-07-01

Brief Summary

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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.

Detailed Description

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Conditions

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Hypoplastic Left Heart Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open label C-kit+ cells Group A

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 Type EXPERIMENTAL

c-kit+ cells

Intervention Type 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.

C-kit+ cells Group B

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 Type ACTIVE_COMPARATOR

c-kit+ cells

Intervention Type 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.

No Intervention Group

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.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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c-kit+ cells

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.

Intervention Type BIOLOGICAL

Other Intervention Names

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Autologous c-kit-positive cells (c-kit+ cells)

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* Candidates will be excluded from the study if any of the following conditions are met:

1. Subjects undergoing the Stage I Norwood operation who do not have HLHS.
2. Subjects requiring mechanical circulatory support immediately prior to Stage II BDCPA operation (within 5 days).
3. Parent or guardian unwilling or unable to comply with necessary follow-up(s).
4. Mother is serum positive for HIV 1/2, hepatitis B surface antigen or viremic hepatitis C and Treponema pallidum.
5. Subjects who are unsuitable for inclusion in the study in the opinion of the investigator(s).
Minimum Eligible Age

1 Day

Maximum Eligible Age

21 Days

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Joshua M Hare

OTHER

Sponsor Role lead

Responsible Party

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Joshua M Hare

Professor

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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William Mahle, MD

Role: PRINCIPAL_INVESTIGATOR

Emory University

Kristopher Deatrick, MD

Role: PRINCIPAL_INVESTIGATOR

University of Maryland

Richard Ohye, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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Emory University Children's Healthcare of Atlanta - Egleston Campus

Atlanta, Georgia, United States

Site Status

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

Chicago, Illinois, United States

Site Status

University of Maryland - Division of Cardiac Surgery

Baltimore, Maryland, United States

Site Status

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

Ann Arbor, Michigan, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 41258851 (View on PubMed)

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.

Reference Type DERIVED
PMID: 35394149 (View on PubMed)

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.

Reference Type DERIVED
PMID: 34217109 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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http://ISCI.MED.MIAMI.EDU

Interdisciplinary stem cell institute at the University of Miami website

Other Identifiers

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20190743

Identifier Type: -

Identifier Source: org_study_id

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