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Trial record 2 of 4 for:    longeveron

Longeveron Mesenchymal Stem Cells (LMSCs) Delivered During Stage II Surgery for Hypoplastic Left Heart Syndrome (ELPIS) (ELPIS)

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ClinicalTrials.gov Identifier: NCT03525418
Recruitment Status : Recruiting
First Posted : May 15, 2018
Last Update Posted : October 8, 2018
Sponsor:
Information provided by (Responsible Party):
Longeveron LLC

Brief Summary:
This study is designed to assess the safety, tolerability, and efficacy of LMSCs as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS. LMSCs 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 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. LMSCs 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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 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: Allogeneic Human MEsenchymal Stem Cell (MSC) Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)
Actual Study Start Date : February 21, 2018
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2022

Resource links provided by the National Library of Medicine


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.



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.



Information from the National Library of Medicine

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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

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


Contacts
Contact: Suzanne L Page (305) 909-0840 spage@longeveron.com
Contact: Geoff Green (305) 909-0840 ggreen@longeveron.com

Locations
United States, Georgia
Emory University/Childen's Healthcare of Atlanta Not yet recruiting
Atlanta, Georgia, United States, 30307
Contact: Nikita Rao, MPH    404-785-0996    nikita.rao@choa.org   
Principal Investigator: William T Mahle, MD         
United States, Maryland
University of Maryland Medical Center Recruiting
Baltimore, Maryland, United States, 21201
Contact: Sunjay Kaushal, MD, PhD    410-328-5842    skaushal@som.maryland.edu   
Contact: Manal Al-Suqi    (410) 328-9409    MaAl-Suqu@som.umaryland.edu   
Johns Hopkins University Hospital Recruiting
Baltimore, Maryland, United States, 21287
Contact: Narutoshi Hibino, MD, PhD    410-287-9193    nhibino1@jhmi.edu   
Contact: Judi Willhide, RN, BSN    (410) 955-3597    Jwillhi3@jhmi.edu   
United States, Michigan
C.S Mott Children's Hospital/University of Michigan Congenital Heart Center Not yet recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Tammy Doman, RN    734-763-6109    tpaterso@med.umich.edu   
Principal Investigator: Ming-Sing Si, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Alyssa Rohde    513-803-8920    alyssa.rohde@cchmc.org   
Principal Investigator: James S Tweddell, MD         
United States, Utah
University of Utah/Heart Center-Primary Children's Hospital Not yet recruiting
Salt Lake City, Utah, United States, 84113
Contact: Linda Lambert, MSN    801-587-7523    linda.lambert@hsc.utah.edu   
Principal Investigator: S. Adil Husain, MD         
Sponsors and Collaborators
Longeveron LLC
Investigators
Principal Investigator: Joshua M Hare, MD Longeveron LLC

Responsible Party: Longeveron LLC
ClinicalTrials.gov Identifier: NCT03525418     History of Changes
Other Study ID Numbers: 00-0000-05
First Posted: May 15, 2018    Key Record Dates
Last Update Posted: October 8, 2018
Last Verified: October 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Longeveron LLC:
Pediatrics

Additional relevant MeSH terms:
Hypoplastic Left Heart Syndrome
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities