Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome (ELPIS)
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|ClinicalTrials.gov Identifier: NCT02398604|
Recruitment Status : Recruiting
First Posted : March 25, 2015
Last Update Posted : September 24, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hypoplastic Left Heart Syndrome||Drug: Allo-hMSCs Drug: Placebo||Phase 1|
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 :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||20 patients and 10 patients will be controls with a total of 30 HLHS patients.|
|Masking:||None (Open Label)|
|Masking Description:||HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio|
|Official Title:||Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.|
|Study Start Date :||April 2015|
|Estimated Primary Completion Date :||October 2020|
|Estimated Study Completion Date :||October 2022|
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 Name: stem cells; Allogeneic Human Mesenchymal Stem Cells
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.
Other Name: Placebo comparator
- 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.
- 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.
- 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.
- 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
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02398604
|Contact: Joshua M Hare, MDfirstname.lastname@example.org|
|Contact: Sunjay Kaushal, MD||410-328-0061||SKaushal@som.umaryland.edu|
|United States, Maryland|
|University of Maryland Medical Center||Recruiting|
|Baltimore, Maryland, United States, 21201|
|Contact: Sunjay Kaushal, MD, PhD 410-328-5842 email@example.com|
|Contact: Helina Mehta 410-328-2750 HGMehta@som.umaryland.edu|
|Principal Investigator: Sunjay Kaushal, MD, Ph.D.|
|Johns Hopkins Hospital||Recruiting|
|Baltimore, Maryland, United States, 21287|
|Contact: Naru Hibino, MD 410-955-2800 firstname.lastname@example.org|
|Contact: William Ravekes, MD 410-955-5987 email@example.com|
|Principal Investigator: Narutishi Hibino, MD|
|Principal Investigator:||Sunjay Kaushal, MD, Ph.D.||University of Maryland|