Clinical trial NCT03525418: Lomecel-B Delivered During Stage II Surgery for Hypoplastic Left Heart Syndrome (ELPIS)
Official title: Lomecel-B Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: HLHS
Keywords: Pediatrics
Lead sponsor: Longeveron Inc.
Sponsor class: INDUSTRY
First posted: 2018-05-15
Enrollment: 10
Sex: ALL
Minimum age (years): 1
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
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.
Primary purpose: TREATMENT

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

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).

Eligibility 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

Primary outcomes:
measure: 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.
timeFrame: Evaluated through 1 year post-treatment.
Secondary outcomes:
measure: Efficacy: Change from baseline in right ventricular ejection fraction (%).
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change from baseline in right ventricular end-systolic volume.
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change from baseline in right ventricular end-diastolic volume.
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change from baseline in right ventricular end-diastolic diameter.
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change from baseline tricuspid regurgitation.
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change in weight (in kilograms).
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change in height (in centimeters).
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Change in head circumference (in centimeters).
timeFrame: Evaluated through 1 year post-treatment.
measure: Efficacy: Number of patients with Treatment-Emergent Adverse Events, and total number of occurrences of Treatment-Emergent Adverse Events, through-out participation in trial.
timeFrame: Evaluated through 1 year post-treatment.
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University/Childen's Healthcare of Atlanta
state: Georgia
city: Baltimore
country: United States
facility: University of Maryland Medical Center
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins University Hospital
state: Maryland
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Salt Lake City
country: United States
facility: University of Utah/Heart Center-Primary Children's Hospital
state: Utah
Study officials:
References: