VentriGel for Hypoplastic Left Heart Syndrome

Overseen By William Mahle, MD

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: Emory University

Must not be taking: Antiarrhythmics, Immunosuppressants

Disqualifiers: Tumor, Coagulation disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: Immediate during surgery

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called VentriGel (Ventrix Bio Extracellular Matrix) to determine its safety for children with Hypoplastic Left Heart Syndrome (HLHS). The study will evaluate whether this treatment can improve heart function when injected during the Stage II Glenn operation. Children with HLHS scheduled for this surgery might be suitable candidates for the trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications. However, if you are on medications that cause immunosuppression or antiarrhythmic drugs, you may need to discuss this with the trial team.

Is there any evidence suggesting that VentriGel is safe for treating Hypoplastic Left Heart Syndrome?

Research has shown that VentriGel, a special gel for the heart, has been tested for safety in people. Studies have found it safe when injected into patients. Currently, the research focuses on ensuring its safety, with scientists still gathering safety information. Early results suggest that VentriGel is well-tolerated, and no serious side effects have been reported so far. However, participants should understand any possible risks and discuss them with their healthcare team. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Ventrix Bio Extracellular Matrix is unique because it introduces a bioengineered extracellular matrix directly into the heart muscle, aiming to regenerate heart tissue in patients with Hypoplastic Left Heart Syndrome (HLHS). Unlike traditional treatments that focus on surgical reconstruction of heart pathways, this approach targets tissue repair at a cellular level. Researchers are excited about this treatment because it could enhance heart function by promoting natural tissue growth and healing, potentially reducing the need for future surgeries and improving long-term outcomes for HLHS patients.

What evidence suggests that VentriGel might be an effective treatment for Hypoplastic Left Heart Syndrome?

Research shows that VentriGel, a special gel, has potential in treating heart problems. In earlier studies with heart attack patients, researchers found VentriGel to be safe and practical when injected into the heart. This trial will investigate VentriGel's use for Hypoplastic Left Heart Syndrome (HLHS), injecting it into the right ventricle during the Stage II Glenn Operation. Although direct information on its use for HLHS is limited, its ability to help tissue regenerate offers hope for improved heart function in individuals with this condition. Overall, early results suggest possible benefits, but further research is needed to confirm its effectiveness specifically for HLHS. ^{1 2 3 6 7}

Who Is on the Research Team?

William Mahle, MD

Principal Investigator

Emory University

Are You a Good Fit for This Trial?

This trial is for pediatric patients with Hypoplastic Left Heart Syndrome (HLHS). Specific eligibility details are not provided, but typically participants must meet certain health criteria and may be excluded based on factors that could interfere with the study or their safety.

Inclusion Criteria

My child has HLHS and needs a Stage II Glenn operation before turning one.

Exclusion Criteria

I have a heart rhythm problem known as a conduction abnormality.

I have heart rhythm problems from heart medication.

Parent or guardian unwilling or unable to comply with necessary follow-up(s)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive VentriGel injections during the Stage II Glenn Operation

Immediate during surgery

1 visit (in-person, during surgery)

Post-operative Monitoring

Participants are monitored for adverse events and cardiac function post-operation

30 days

Regular monitoring (in-person)

Follow-up

Participants are monitored for changes in cardiac function through echocardiograms and MRI scans

12 months

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Ventrix Bio Extracellular Matrix

Trial Overview The trial is testing VentriGel, an extracellular matrix product injected into the heart muscle. It's a Phase I Open-Label study to check if it's safe for children with HLHS and to see any initial improvements in heart function.

How Is the Trial Designed?

Group I: Ventrix Bio Extracellular Matrix Experimental Treatment 1 Intervention

Treatment intervention of a total of 8 injections into the right ventricle of the heart will occur before the patient comes off of the heart-lung bypass machine used during the Stage II Glenn Operation. The treatment will be conducted according to clinical guidelines set in place while the patient is undergoing a surgical intervention before the patient comes off the heart/lung bypass machine required for the stage II Glenn Procedure.

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials

1,735

Recruited

2,605,000+

University of California, San Diego

Collaborator

Trials

1,215

Recruited

1,593,000+

Ventrix, Inc.

Industry Sponsor

Trials

2

Recruited

30+

Published Research Related to This Trial

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06461676?cond=Hypoplastic%20left%20heart%20syndrome&viewType=Table&rank=4

Study of Intramyocardial Injection of Ventrix Bio ... A group of independent scientists who monitor the safety and scientific integrity of a clinical trial. The DMC can recommend to the sponsor that the trial be ...

2. ctv.veeva.com ctv.veeva.com/study/study-of-intramyocardial-injection-of-ventrix-bio-extracellular-matrix-ventrigel-to-assess-the-saf

Study of Intramyocardial Injection of Ventrix Bio Extracellular ... The goal of this Phase I Open-Label study is to demonstrate the safety and feasibility of VentriGel injection in children with Hypoplastic ...

3. withpower.com withpower.com/trial/phase-1-hypoplastic-left-heart-syndrome-9-2024-c2b0c

VentriGel for Hypoplastic Left Heart Syndrome A study on VentriGel, a cardiac extracellular matrix hydrogel, showed it was safe and feasible when injected into patients who had heart attacks, suggesting it ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10485645/

Cardiac tissue engineering for the treatment of hypoplastic ... It has been hypothesized that stem cell-based regenerative therapies could have a role in promoting cardiac tissue regeneration in HLHS patients who are ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S2589004223020576

Statistical modeling of extracellular vesicle cargo to predict ... Article. Statistical modeling of extracellular vesicle cargo to predict clinical trial outcomes for hypoplastic left heart syndrome.

6. delta.larvol.com delta.larvol.com/Products/?ProductId=be7beac2-2c71-4d40-a044-45c7ba589aff

VentriGel (extracellular matrix hydrogel) / Ventrix Study of intramyocardial injection of Ventrix bio extracellular matrix (VentriGel) to assess the safety and feasibility in pediatric patients with hypoplastic ...

7. clinconnect.io clinconnect.io/trials/NCT06461676

Study of Intramyocardial Injection of Ventrix Bio - ClinConnect The goal of this Phase I Open-Label study is to demonstrate the safety and feasibility of VentriGel injection in children with Hypoplastic Left Heart ...