- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03525418
Lomecel-B Delivered During Stage II Surgery for Hypoplastic Left Heart Syndrome (ELPIS) (ELPIS)
Lomecel-B Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B (formerly LMSCs) as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS, which is typically performed at 4 - 6 months after birth. Lomecel-B will be delivered via intramyocardial injections.
A total of 30 patients will be enrolled in 2 stages with 3 Cohorts.
In the first stage, 10 consecutive HLHS patients will be enrolled and treated with Lomecel-B (Cohort A). The first 3 patients will be treated no less than 5 days apart, and will be evaluated for any treatment-emergent adverse events (TE-AEs) (e.g., induced myocardial infarction or perforation). These patients will undergo full evaluation for 5 days to demonstrate safety prior to proceeding with the remainder of the cohort. After 6 months post-treatment of the last patient of Cohort A, a formal safety review will be conducted prior to proceeding to the next phase.
The second stage is double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Lomecel-B (Cohort B, 10 patients), or will receive no cells and no injection (Cohort C, 10 patients).
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
-
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Georgia
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Atlanta, Georgia, United States, 30307
- Emory University/Childen's Healthcare of Atlanta
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Maryland
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Baltimore, Maryland, United States, 21201
- University of Maryland Medical Center
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Baltimore, Maryland, United States, 21287
- Johns Hopkins University Hospital
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Utah
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Salt Lake City, Utah, United States, 84113
- University of Utah/Heart Center-Primary Children's Hospital
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria: all patients must have HLHS (all types) requiring BDCPA surgery.
Exclusion Criteria: all patients must not have any of the following.
- Significant coronary artery sinusoids.
- Requirement for mechanical circulatory support prior to BDCPA surgery.
- Underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
- HLHS and restrictive or intact atrial septum.
- Undergoing the Stage I (Norwood) procedure that does not have HLHS.
- Serum positivity for: HIV; hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV).
- Parent/guardian that is unwilling or unable to comply with necessary follow-up.
- Unsuitability for the study based on the Investigator's clinical opinion.
- Documented chromosomal abnormalities
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort A - Phase 1 (Open Label)
10 consecutive HLHS patients will be enrolled and treated with Longeveron Mesenchymal Stem Cells (LMSCs).
A single administration of LMSCs will be performed via intramyocardial injections during the Stage II (BDCPA) surgery.
Dosing is based on body weight.
Each LMSC-treated patient will be given 2.5 x 105 LMSCs per kg of body weight.
The entire dose of the cells will be roughly 600 microliters.
|
Allogeneic bone marrow-derived mesenchymal stem cell
Other Names:
|
Experimental: Cohort B - Phase 2 Treatment Group
Double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Longeveron Mesenchymal Stem Cells (LMSCs) (Cohort B, 10 patients) performed via intramyocardial injections during the Stage II (BDCPA) surgery, or will receive no cells and no injection (Cohort C, 10 patients) during the Stage II (BDCPA) surgery.
The second stage is to obtain preliminary safety and efficacy data the will enable and guide a subsequent larger Phase 2 trial.
|
Allogeneic bone marrow-derived mesenchymal stem cell
Other Names:
|
No Intervention: Cohort C - Phase 2 Control Group
Double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Longeveron Mesenchymal Stem Cells (LMSCs) (Cohort B, 10 patients) performed via intramyocardial injections during the Stage II (BDCPA) surgery, or will receive no cells and no injection (Cohort C, 10 patients) during the Stage II (BDCPA) surgery.
The second stage is to obtain preliminary safety and efficacy data the will enable and guide a subsequent larger Phase 2 trial.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety: To evaluate the safety and feasibility of intramyocardial injection of LMSCs during the Stage II (BDCPA) operation for HLHS via incidence of Treatment-Emergent Serious Adverse Events.
Time Frame: Evaluated through 1 year post-treatment.
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The incidence of Treatment-Emergent Serious Adverse Events will be evaluated, including: sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support; aggravation of heart failure; myocardial infarction; unplanned cardiovascular operation for cardiac tamponade; infection during the first month post-treatment; and death.
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Evaluated through 1 year post-treatment.
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Efficacy: Change from baseline in right ventricular ejection fraction (%).
Time Frame: Evaluated through 1 year post-treatment.
|
Used to assess cardiac function.
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Evaluated through 1 year post-treatment.
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Efficacy: Change from baseline in right ventricular end-systolic volume.
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess cardiac function.
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Evaluated through 1 year post-treatment.
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Efficacy: Change from baseline in right ventricular end-diastolic volume.
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess cardiac function.
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Evaluated through 1 year post-treatment.
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Efficacy: Change from baseline in right ventricular end-diastolic diameter.
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess cardiac function.
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Evaluated through 1 year post-treatment.
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Efficacy: Change from baseline tricuspid regurgitation.
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess cardiac function.
Measured by serial echocardiograms and MRI.
|
Evaluated through 1 year post-treatment.
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Efficacy: Change in weight (in kilograms).
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess change in somatic growth.
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Evaluated through 1 year post-treatment.
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Efficacy: Change in height (in centimeters).
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess change in somatic growth.
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Evaluated through 1 year post-treatment.
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Efficacy: Change in head circumference (in centimeters).
Time Frame: Evaluated through 1 year post-treatment.
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Used to assess change in somatic growth.
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Evaluated through 1 year post-treatment.
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Efficacy: Number of patients with Treatment-Emergent Adverse Events, and total number of occurrences of Treatment-Emergent Adverse Events, through-out participation in trial.
Time Frame: Evaluated through 1 year post-treatment.
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Treatment-Emergent Adverse Events will be assessed via incidence of co-morbidity, which include: cardiovascular morbidity; need for transplantation; re-hospitalizations; cardiovascular mortality; and all-cause mortality.
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Evaluated through 1 year post-treatment.
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
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
- 00-0000-05
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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