Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome

NCT02398604 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: 20140718
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

Brief Summary

This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.

Conditions

Hypoplastic Left Heart Syndrome

Keywords

Pediatrics; HLHS

Outcome Measures

Primary Outcomes (1)

• Monitor major adverse cardiac events

  Monitoring of events includes death, sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support, aggravation of heart failure, new myocardial infarction, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.

  1 month after injection

Secondary Outcomes (4)

• Composite Change from baseline in right ventricular function, right ventricular end-diastolic volume, right ventricular end-systolic volume, right ventricular end-systolic diameter and tricuspid regurgitation

  baseline, 24 weeks and 1 year

• Number of participants with incidence of mortality or need for transplantation after the BDCPA operation

  One year after injections

• Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation

  12 months post operative

• Assessment of Co-morbidity

  up to 12 months follow-up

Study Arms (2)

Group A - Allo-hMSCs

EXPERIMENTAL

Group A: Fifteen (20) patients will be treated with Allogenic human mesenchymal stem cells (Allo-hMSCs): A concentration of 5 million cells/ml delivered in a dose of 2.5 x 10\^5 cells per kg of recipient (5 million/20kg) Allo-hMSCs. The entire dose of the cells will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation.

Drug: Allo-hMSCs

Group B

PLACEBO COMPARATOR

Group B: Fifteen (10) patients will be treated with a placebo comparator. The placebo will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation.

Drug: Placebo

Interventions

Allo-hMSCs DRUG

Allogeneic Human Mesenchymal Stem Cells

Also known as: stem cells; Allogeneic Human Mesenchymal Stem Cells

Group A - Allo-hMSCs

Placebo DRUG

Placebo

Also known as: Placebo comparator

Group B

Eligibility Criteria

• Age: Up to 30 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.

You may not qualify if:

• Have HLHS and restrictive or intact atrial septum.
• Be undergoing the Norwood procedure that do not have HLHS.
• Have significant coronary artery sinusoids.
• Require mechanical circulatory support prior to surgery.
• Have an underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
• Parent or guardian unwilling or unable to comply with necessary follow-up(s)
• Be serum positive for HIV, hepatitis BsAg (B Surface Antigen) or viremic hepatitis C.
• Need for concomitant surgery for aortic coarctation or tricuspid valve repair.

Sponsors & Collaborators

• Longeveron Inc.: lead

Study Sites (2)

University of Maryland Medical Center

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Related Links

• Interdisciplinary Stem Cell Institute

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

Study Officials

• Anthony Oliva, MD

  Longeveron Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 20 patients and 10 patients will be controls with a total of 30 HLHS patients.

Study Record Dates

• First Submitted: December 24, 2014
• First Posted: March 25, 2015
• Study Start: April 1, 2015
• Primary Completion: March 1, 2019
• Study Completion: March 1, 2019
• Last Updated: July 19, 2021

Record last verified: 2021-07

Locations

US (2)