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HLHSPhase 1/2ELPISNCT03525418

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

A Phase 1/2 study testing Longeveron Mesenchymal Stem Cells in people with HLHS, run by Longeveron Inc. at 5 sites in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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 Read more about this study

Who is it for?
Ages 1 day to 1 year
Conditions the study is for
HLHS
What is being tested?
Longeveron Mesenchymal Stem Cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
5 sitesUnited States
Study dates
21 Feb 2018to 31 Dec 2025
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 day to 1 year
  • All sexes
  • No healthy volunteers
  • Child

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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • Emory University/Childen's Healthcare of Atlanta

    Atlanta, Georgia, 30307

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201

  • Johns Hopkins University Hospital

    Baltimore, Maryland, 21287

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

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

    Salt Lake City, Utah, 84113

Other trials in United States

About this study

Official title: Lomecel-B Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)

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

Read the 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).

Conditions
HLHS
Being tested
Longeveron Mesenchymal Stem Cells
Keywords
Pediatrics

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Longeveron Mesenchymal Stem CellsBiological

    Also called LMSCs

    Allogeneic bone marrow-derived mesenchymal stem cell

Study groups in NCT03525418
Study groupRoleWhat they receive
Cohort A - Phase 1 (Open Label)Experimental10 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.
Receives: Biological: Longeveron Mesenchymal Stem Cells
Cohort B - Phase 2 Treatment GroupExperimentalDouble-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.
Receives: Biological: Longeveron Mesenchymal Stem Cells
Cohort C - Phase 2 Control GroupNo interventionDouble-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 being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Evaluated through 1 year post-treatment.

Secondary outcomes (9)

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

    Used to assess cardiac function.

    Measured: Evaluated through 1 year post-treatment.

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

    Used to assess cardiac function.

    Measured: Evaluated through 1 year post-treatment.

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

    Used to assess cardiac function.

    Measured: Evaluated through 1 year post-treatment.

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

    Used to assess cardiac function.

    Measured: Evaluated through 1 year post-treatment.

  5. Efficacy: Change from baseline tricuspid regurgitation.

    Used to assess cardiac function. Measured by serial echocardiograms and MRI.

    Measured: Evaluated through 1 year post-treatment.

  6. Efficacy: Change in weight (in kilograms).

    Used to assess change in somatic growth.

    Measured: Evaluated through 1 year post-treatment.

  7. Efficacy: Change in height (in centimeters).

    Used to assess change in somatic growth.

    Measured: Evaluated through 1 year post-treatment.

  8. Efficacy: Change in head circumference (in centimeters).

    Used to assess change in somatic growth.

    Measured: Evaluated through 1 year post-treatment.

  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.

    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.

    Measured: Evaluated through 1 year post-treatment.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 May 2018
  2. Study start21 Feb 2018
  3. Primary completion30 Jun 2024 (estimated)
  4. Study completion31 Dec 2025 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
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 blind
Who is masked
Participant, Investigator
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03525418
Sponsor's protocol ID
00-0000-05
Acronym
ELPIS
Status last verified
Oct 2023
First submitted
19 Feb 2018
Last updated
4 Oct 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 36950450

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Longeveron Mesenchymal Stem Cells in people with HLHS, run by Longeveron Inc. at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 4 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome 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. (time frame: Evaluated through 1 year post-treatment.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03525418, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2023.

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