Clinical trial NCT03406884: The CHILD Trial: Hypoplastic Left Heart Syndrome Study.
Official title: Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hypoplastic Left Heart Syndrome
Keywords: stem cells
pediatric
Lead sponsor: University of Miami
Sponsor class: OTHER
First posted: 2018-01-23
Enrollment: 25
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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 purpose: TREATMENT

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 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.


Eligibility criteria: Inclusion Criteria:

* For inclusion in the study, subjects must meet all of the inclusion criteria:

  1. Subjects with hypoplastic left heart syndrome (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 BsAg or viremic hepatitis C and Treponema pallidum.
  5. Subjects who are unsuitable for inclusion in the study in the opinion of the investigator(s).

Primary outcomes:
measure: Number of Incidence of Treatment Related Major Adverse Cardiac Events
timeFrame: 30 days
measure: Number of c-kit+ products
timeFrame: Day 1
measure: Number of participants completing Magnetic Resonance Imaging (MRI)
timeFrame: 12 months
measure: Change in right ventricular function
timeFrame: Baseline, 12 months
measure: Change in Right Ventricular End-diastolic Volume
timeFrame: Baseline, 12 months
measure: Change in Right Ventricular End-systolic Volume
timeFrame: Baseline, 12 months
measure: Change in Tricuspid Regurgitation
timeFrame: Baseline, 12 months
Secondary outcomes:
measure: Number of Incidence of Serious Adverse Events
timeFrame: Up to 12 months
measure: Change in somatic growth velocity
timeFrame: Baseline, 12 months
measure: Change in Infant Toddler Quality of Life Survey (ITQOL)
timeFrame: Baseline, 12 months
measure: Incidence of mortality or need for transplantation
timeFrame: Up to 12 months
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University Children's Healthcare of Atlanta - Egleston Campus
state: Georgia
city: Baltimore
country: United States
facility: University of Maryland - Division of Cardiac Surgery
state: Maryland
city: Ann Arbor
country: United States
facility: Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital
state: Michigan
Study officials:
affiliation: Emory University
name: William Mahle, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Maryland
name: Kristopher Deatrick, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital
name: Richard Ohye, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.