Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome (ELPIS)
Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
연구 개요
상세 설명
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.
연구 유형
등록 (실제)
단계
- 1단계
연락처 및 위치
연구 장소
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Maryland
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Baltimore, Maryland, 미국, 21287
- Johns Hopkins Hospital
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Baltimore, Maryland, 미국, 21201
- University of Maryland Medical Center
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.
Exclusion Criteria:
- 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.
- Be unsuitable for inclusion in the study, in the opinion of the investigator.
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: Group A - Allo-hMSCs
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.
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Allogeneic Human Mesenchymal Stem Cells
다른 이름들:
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위약 비교기: Group B
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.
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위약
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Monitor major adverse cardiac events
기간: 1 month after injection
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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.
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1 month after injection
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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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
기간: baseline, 24 weeks and 1 year
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as measured by serial echocardiograms and MRI scans.
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baseline, 24 weeks and 1 year
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Number of participants with incidence of mortality or need for transplantation after the BDCPA operation
기간: One year after injections
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Assessed through the number of participants with adverse events, hospitalizations, or transplantations.
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One year after injections
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Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation
기간: 12 months post operative
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Changes in weight, height, and head circumference will be measured
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12 months post operative
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Assessment of Co-morbidity
기간: up to 12 months follow-up
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Assessment of cardiovascular mortality, all-cause morbidity, Cardiovascular morbidity, re-hospitalizations, need for transplantation.
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up to 12 months follow-up
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공동 작업자 및 조사자
스폰서
수사관
- 수석 연구원: Anthony Oliva, MD, Longeveron Inc.
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (추정)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
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