- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02398604
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.
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
-
-
Maryland
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Baltimore, Maryland, United States, 21287
- Johns Hopkins Hospital
-
Baltimore, Maryland, United States, 21201
- University of Maryland Medical Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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.
|
Allogeneic Human Mesenchymal Stem Cells
Other Names:
|
Placebo Comparator: 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.
|
Placebo
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Monitor major adverse cardiac events
Time Frame: 1 month after injection
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
Time Frame: baseline, 24 weeks and 1 year
|
as measured by serial echocardiograms and MRI scans.
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baseline, 24 weeks and 1 year
|
Number of participants with incidence of mortality or need for transplantation after the BDCPA operation
Time Frame: One year after injections
|
Assessed through the number of participants with adverse events, hospitalizations, or transplantations.
|
One year after injections
|
Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation
Time Frame: 12 months post operative
|
Changes in weight, height, and head circumference will be measured
|
12 months post operative
|
Assessment of Co-morbidity
Time Frame: up to 12 months follow-up
|
Assessment of cardiovascular mortality, all-cause morbidity, Cardiovascular morbidity, re-hospitalizations, need for transplantation.
|
up to 12 months follow-up
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Anthony Oliva, MD, Longeveron Inc.
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 20140718
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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