10 Participants Needed

Lomecel-B for Hypoplastic Left Heart Syndrome

(ELPIS Trial)

Recruiting at 4 trial locations
Age: < 18
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Longeveron Inc.
Stay on Your Current MedsYou can continue your current medications while participating
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you are on anti-arrhythmia therapy, you may not be eligible to participate.

What data supports the effectiveness of the treatment Lomecel-B for Hypoplastic Left Heart Syndrome?

Research suggests that mesenchymal stem cells, like those in Lomecel-B, may help improve heart function in patients with Hypoplastic Left Heart Syndrome by delivering biological signals that support the heart's right ventricle. Studies in similar treatments have shown potential benefits in heart function, which is promising for this condition.12345

Is Lomecel-B safe for humans?

The ELPIS trial is testing the safety of Lomecel-B (mesenchymal stem cells) in children with hypoplastic left heart syndrome by monitoring for major heart-related issues. If no serious problems occur in the first 10 patients, the study will continue to further test its safety and effectiveness.23467

How is the treatment Lomecel-B different from other treatments for hypoplastic left heart syndrome?

Lomecel-B is unique because it is a regenerative medicine approach, which aims to repair or replace damaged heart tissue, potentially offering a curative option rather than just managing symptoms like traditional surgeries. This treatment may involve using stem cells or similar regenerative techniques to improve heart function, which is different from the standard surgical interventions currently used for hypoplastic left heart syndrome.14589

What is the purpose of this trial?

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

Eligibility Criteria

This trial is for patients with Hypoplastic Left Heart Syndrome (HLHS) who need stage II surgery. It's not for those with certain HLHS complications, positive tests for HIV/HBV/HCV, chromosomal abnormalities, arrhythmias needing treatment, or additional heart issues requiring other surgeries.

Inclusion Criteria

I have HLHS and need BDCPA surgery.

Exclusion Criteria

My cancer has specific genetic changes.
I have large blood vessels in my heart's artery.
I need a device to help my heart pump blood before my heart surgery.
See 7 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment - Stage I

10 HLHS patients in Cohort A receive Lomecel-B via intramyocardial injections during Stage II surgery. Initial 3 patients evaluated for 5 days for safety.

5 days
In-hospital stay for surgery and initial evaluation

Safety Review

Formal safety review conducted 6 months post-treatment of the last patient in Cohort A.

6 months

Treatment - Stage II

20 HLHS patients randomized in a double-blind manner to receive Lomecel-B or no treatment during Stage II surgery.

During surgery
In-hospital stay for surgery

Follow-up

Participants are monitored for safety and effectiveness after treatment, including cardiac function and somatic growth assessments.

1 year

Treatment Details

Interventions

  • Lomecel-B
Trial Overview The study evaluates the safety and potential benefits of Lomecel-B stem cells given as an extra treatment during standard stage II surgery for HLHS. The cells are injected directly into the heart muscle.
Participant Groups
3Treatment groups
Experimental Treatment
Active Control
Group I: Cohort B - Phase 2 Treatment GroupExperimental Treatment1 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.
Group II: Cohort A - Phase 1 (Open Label)Experimental Treatment1 Intervention
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.
Group III: Cohort C - Phase 2 Control GroupActive Control1 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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Longeveron Inc.

Lead Sponsor

Trials
10
Recruited
480+

Findings from Research

Regenerative medicine approaches for treating hypoplastic left heart syndrome (HLHS) offer a promising alternative to current palliative care methods, potentially leading to curative treatments.
Despite the potential, significant challenges remain in translating these innovative strategies into clinical practice, necessitating further scientific and technological advancements.
The Potential Role of Regenerative Medicine on the Future Management of Hypoplastic Left Heart Syndrome.Kelly, JM., Anderson, C., Breuer, CK.[2023]
The ELPIS trial is investigating the safety and feasibility of injecting bone marrow-derived mesenchymal stem cells (MSCs) in 10 children with hypoplastic left heart syndrome (HLHS) to improve right ventricular function, with a focus on monitoring for major adverse cardiac events.
If the initial phase is successful, a larger phase IIb trial will compare MSC injection to standard surgical care in 20 patients, aiming to assess improvements in cardiac function and reduce the need for transplantation.
Study design and rationale for ELPIS: A phase I/IIb randomized pilot study of allogeneic human mesenchymal stem cell injection in patients with hypoplastic left heart syndrome.Kaushal, S., Wehman, B., Pietris, N., et al.[2018]
Stem cell therapy shows promise as a novel treatment for managing right ventricular dysfunction in patients with hypoplastic left heart syndrome, a serious congenital heart condition that often leads to significant morbidity after surgical palliation.
Current research indicates that the therapeutic effects of stem cells may primarily result from their paracrine effects—where they release signaling molecules—rather than from direct integration into heart tissue, which is a shift in understanding that could influence future clinical applications.
Stem Cell Therapy for Hypoplastic Left Heart Syndrome: Mechanism, Clinical Application, and Future Directions.Bittle, GJ., Morales, D., Deatrick, KB., et al.[2020]

References

The Potential Role of Regenerative Medicine on the Future Management of Hypoplastic Left Heart Syndrome. [2023]
Study design and rationale for ELPIS: A phase I/IIb randomized pilot study of allogeneic human mesenchymal stem cell injection in patients with hypoplastic left heart syndrome. [2018]
Stem Cell Therapy for Hypoplastic Left Heart Syndrome: Mechanism, Clinical Application, and Future Directions. [2020]
Characterization of an iPSC line NCHi006-A from a patient with hypoplastic left heart syndrome (HLHS). [2022]
Intracoronary autologous cardiac progenitor cell transfer in patients with hypoplastic left heart syndrome: the TICAP prospective phase 1 controlled trial. [2015]
Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study). [2022]
NOTCH1-Dependent Nitric Oxide Signaling Deficiency in Hypoplastic Left Heart Syndrome Revealed Through Patient-Specific Phenotypes Detected in Bioengineered Cardiogenesis. [2018]
[Favourable results with surgical treatment in 43 children with hypoplastic left-heart syndrome or similar disorders, 1999-2005]. [2007]
Differential changes in TGF-β/BMP signaling pathway in the right ventricular myocardium of newborns with hypoplastic left heart syndrome. [2022]
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