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Lomecel-B Delivered During Stage II Surgery for Hypoplastic Left Heart Syndrome (ELPIS) (ELPIS)

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ClinicalTrials.gov Identifier: NCT03525418
Recruitment Status : Completed
First Posted : May 15, 2018
Last Update Posted : March 7, 2022
Sponsor:
Information provided by (Responsible Party):
Longeveron Inc.

Study Description
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Brief Summary:
This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS. Lomecel-B will be delivered via intramyocardial injections

Condition or disease Intervention/treatment Phase
HLHS Biological: Longeveron Mesenchymal Stem Cells Phase 1 Phase 2

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 LMSCs (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 LMSCs (Cohort B, 10 patients), or will receive no cells and no injection (Cohort C, 10 patients).

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

Phase 1: 10 patient safety run-in: all patients treated with LMSCs during Stage II surgery.

Phase 2: 20 patients randomized 1:1 to receive either LMSCs or no cells (controls) during Stage II surgery.
Masking: Double (Participant, Investigator)
Masking Description: Phase 1: no masking. Phase 2: HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio
Primary Purpose: Treatment
Official Title: Lomecel-B Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)
Actual Study Start Date : February 21, 2018
Actual Primary Completion Date : December 31, 2021
Actual Study Completion Date : December 31, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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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.
 Biological: Longeveron Mesenchymal Stem Cells
Allogeneic bone marrow-derived mesenchymal stem cell
Other Name: LMSCs
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.
 Biological: Longeveron Mesenchymal Stem Cells
Allogeneic bone marrow-derived mesenchymal stem cell
Other Name: LMSCs
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.


Outcome Measures
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Primary Outcome Measures :
  1. 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. ]
    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.


Secondary Outcome Measures :
  1. Efficacy: Change from baseline in right ventricular ejection fraction (%). [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess cardiac function.

  2. Efficacy: Change from baseline in right ventricular end-systolic volume. [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess cardiac function.

  3. Efficacy: Change from baseline in right ventricular end-diastolic volume. [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess cardiac function.

  4. Efficacy: Change from baseline in right ventricular end-diastolic diameter. [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess cardiac function.

  5. Efficacy: Change from baseline tricuspid regurgitation. [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess cardiac function. Measured by serial echocardiograms and MRI.

  6. Efficacy: Change in weight (in kilograms). [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess change in somatic growth.

  7. Efficacy: Change in height (in centimeters). [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess change in somatic growth.

  8. Efficacy: Change in head circumference (in centimeters). [ Time Frame: Evaluated through 1 year post-treatment. ]
    Used to assess change in somatic growth.

  9. 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. ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   up to 1 Year   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria: all patients must have HLHS (all types) requiring BDCPA surgery.

Exclusion Criteria: all patients must not have any of the following.

  1. Significant coronary artery sinusoids.
  2. Requirement for mechanical circulatory support prior to BDCPA surgery.
  3. Underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
  4. Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
  5. HLHS and restrictive or intact atrial septum.
  6. Undergoing the Stage I (Norwood) procedure that does not have HLHS.
  7. Serum positivity for: HIV; hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV).
  8. Parent/guardian that is unwilling or unable to comply with necessary follow-up.
  9. Unsuitability for the study based on the Investigator's clinical opinion.
  10. Documented chromosomal abnormalities
Contacts and Locations
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Information from the National Library of Medicine

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): NCT03525418


Locations
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United States, Georgia
Emory University/Childen's Healthcare of Atlanta
Atlanta, Georgia, United States, 30307
United States, Maryland
University of Maryland Medical Center
Baltimore, Maryland, United States, 21201
Johns Hopkins University Hospital
Baltimore, Maryland, United States, 21287
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Utah
University of Utah/Heart Center-Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Sponsors and Collaborators
Longeveron Inc.
More Information
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Responsible Party: Longeveron Inc.
ClinicalTrials.gov Identifier: NCT03525418    
Other Study ID Numbers: 00-0000-05
First Posted: May 15, 2018    Key Record Dates
Last Update Posted: March 7, 2022
Last Verified: March 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Longeveron Inc.:
Pediatrics
Additional relevant MeSH terms:
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Hypoplastic Left Heart Syndrome
Heart Defects, Congenital
Cardiovascular Abnormalities
 Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities