NCT03525418

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

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2018

Longer than P75 for phase_1

Geographic Reach
1 country

5 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 19, 2018

Completed
2 days until next milestone

Study Start

First participant enrolled

February 21, 2018

Completed
3 months until next milestone

First Posted

Study publicly available on registry

May 15, 2018

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2024

Completed
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2025

Completed
Last Updated

October 4, 2023

Status Verified

October 1, 2023

Enrollment Period

6.4 years

First QC Date

February 19, 2018

Last Update Submit

October 2, 2023

Conditions

HLHS

Keywords

Pediatrics

Outcome Measures

Primary Outcomes (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.

    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.

    Evaluated through 1 year post-treatment.

Secondary Outcomes (9)

  • Efficacy: Change from baseline in right ventricular ejection fraction (%).

    Evaluated through 1 year post-treatment.

  • Efficacy: Change from baseline in right ventricular end-systolic volume.

    Evaluated through 1 year post-treatment.

  • Efficacy: Change from baseline in right ventricular end-diastolic volume.

    Evaluated through 1 year post-treatment.

  • Efficacy: Change from baseline in right ventricular end-diastolic diameter.

    Evaluated through 1 year post-treatment.

  • Efficacy: Change from baseline tricuspid regurgitation.

    Evaluated through 1 year post-treatment.

  • +4 more secondary outcomes

Study Arms (3)

Cohort A - Phase 1 (Open Label)

EXPERIMENTAL

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

Cohort B - Phase 2 Treatment Group

EXPERIMENTAL

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

Cohort C - Phase 2 Control Group

NO INTERVENTION

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.

Interventions

Longeveron Mesenchymal Stem CellsBIOLOGICAL

Allogeneic bone marrow-derived mesenchymal stem cell

Also known as: LMSCs
Cohort A - Phase 1 (Open Label)Cohort B - Phase 2 Treatment Group

Eligibility Criteria

Age1 Day - 1 Year
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Emory University/Childen's Healthcare of Atlanta

Atlanta, Georgia, 30307, United States

Location

University of Maryland Medical Center

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins University Hospital

Baltimore, Maryland, 21287, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Utah/Heart Center-Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

Related Publications (1)

  • Kaushal S, Hare JM, Hoffman JR, Boyd RM, Ramdas KN, Pietris N, Kutty S, Tweddell JS, Husain SA, Menon SC, Lambert LM, Danford DA, Kligerman SJ, Hibino N, Korutla L, Vallabhajosyula P, Campbell MJ, Khan A, Naioti E, Yousefi K, Mehranfard D, McClain-Moss L, Oliva AA, Davis ME. Intramyocardial cell-based therapy with Lomecel-B during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: the ELPIS phase I trial. Eur Heart J Open. 2023 Jan 11;3(2):oead002. doi: 10.1093/ehjopen/oead002. eCollection 2023 Mar.

    PMID: 36950450DERIVED

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Masking Details
Phase 1: no masking. Phase 2: HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: 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.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 19, 2018

First Posted

May 15, 2018

Study Start

February 21, 2018

Primary Completion

June 30, 2024

Study Completion

December 31, 2025

Last Updated

October 4, 2023

Record last verified: 2023-10

Locations

US(5)