NCT02398604

Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome

Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Longeveron Inc. · Industry
• Sex: All
• Age: 30 Days
• Healthy volunteers: Not accepted

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.

Detailed description

This is an open label study, intended as a safety and efficacy assessment prior to a full comparator study. In this Phase I study, cells administered will be delivered in 6-10 intramyocardial injections that will be tested in 20 patients and 10 patients will be controls with a total of 30 HLHS patients. A total of 30 patients with HLHS will be enrolled in a staged enrollment process. In this open-labeled study, a maximum of 20 patients will eventually receive intramyocardial injection of the allogeneic mesenchymal stem cells and 10 control patients with no cell injection. The enrollment of the patients will occur in two stages groups: Group A and Group B. In Group A, 10 consecutive HLHS patients will be initially enrolled in the allogenic MSCs treatment arm to determine feasibility and safety. After 6 months of the last enrolled patient in Group A, all Group A patients will be assessed in order to determine whether it is feasible and safe, including the harvesting, processing, and administering of the allogeneic MSCs. Thereafter, Group B will start enrolling a total of 20 HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio, respectively, in order to have 10 allogeneic MSCs-treated patient and 10 control patients. At the completion of this Phase I clinical study, the total enrolled cohort will be 20 patients treated with allogeneic MSCs and 10 patients in the control arm.

Conditions

• Hypoplastic Left Heart Syndrome

Interventions

Type	Name	Description
DRUG	Allo-hMSCs	Allogeneic Human Mesenchymal Stem Cells
DRUG	Placebo	Placebo

Timeline

• Start date: 2015-04-01
• Primary completion: 2019-03-01
• Completion: 2019-03-01
• First posted: 2015-03-25
• Last updated: 2021-07-19

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study