Study of Intramyocardial Injection of Ventrix Bio Extracellular Matrix (VentriGel) to Assess the Safety and Feasibility in Pediatric Patients with Hypoplastic Left Heart Syndrome (HLHS)

NCT ID: NCT06461676

Last Updated: 2025-03-10

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-08-31
• Study Completion Date: 2028-09-30

Brief Summary

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 Syndrome (HLHS).

The main questions it aims to answer are:

• Whether VentriGel is safe in treating patients with HLHS
• Whether there are any preliminary improvements in measures of cardiac function following Ventrigel injection

Detailed Description

HLHS, a type of congenital heart disease, presents a unique challenge where the left ventricle fails to develop, necessitating the right ventricle to manage both systemic and pulmonary blood flow. This condition, once fatal, has become manageable due to advancements in surgical techniques, with reported 5-year survival rates of 70-90%. However, these staged procedures, while lifesaving, can strain the right ventricle, leading to long-term issues such as weakened muscle and reduced cardiac function. Additionally, even with surgical intervention, patients often face a diminished quality of life.

Addressing these challenges, ongoing clinical trials explore regenerative therapies, particularly stem cell injections, aiming to improve heart function. Yet, concerns persist regarding the practicality and efficacy of these treatments, including issues with cell survival and coordination of injections within narrow timeframes.

Innovatively, the investigator and the team propose an alternative approach using VentriGel, an injectable hydrogel derived from decellularized porcine myocardium. Originally designed for treating heart failure post-myocardial infarction in adults, VentriGel has shown promising results in animal models, demonstrating significant improvements in cardiac function. Notably, its shelf-stable nature and flexible timing for administration offer advantages over traditional stem cell therapies.

Moreover, VentriGel's effectiveness in addressing right-ventricular failure, as demonstrated in recent studies, highlights its potential as a solution for HLHS patients. Leveraging approved extracellular matrix devices, such as Alloderm and SurgiSIS, further underscores the feasibility and safety of this approach, paving the way for potentially transformative treatments in congenital heart diseases.

Conditions

Hypoplastic Left Heart Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ventrix Bio Extracellular Matrix

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.

Group Type: EXPERIMENTAL

Ventrix Bio Extracellular Matrix

Intervention Type: DRUG

VentriGel will be administered by injection into the right ventricle of the heart in the following defined doses per injection. There will be a total of 8 injections. The proposed dose of up to 0.6 mL is administered as up to 8 sequential injections of 4 injections of 0.1 mL each and 4 injections of 0.05 mL each.

Interventions

Ventrix Bio Extracellular Matrix

VentriGel will be administered by injection into the right ventricle of the heart in the following defined doses per injection. There will be a total of 8 injections. The proposed dose of up to 0.6 mL is administered as up to 8 sequential injections of 4 injections of 0.1 mL each and 4 injections of 0.05 mL each.

Intervention Type: DRUG

Other Intervention Names

VentriGel

Eligibility Criteria

Inclusion Criteria

• Subjects with Hypoplastic Left Heart Syndrome (HLHS) requiring Stage II Glenn operation under one year of age

Exclusion Criteria

• Subjects undergoing the Stage II Glenn operations who do not have HLHS
• Subjects requiring mechanical circulatory support within 5 days pre pre-surgical intervention
• Parent or guardian unwilling or unable to comply with necessary follow-up(s)
• Immunosuppressive diseases or subjects who require treatment with interventions that cause immunosuppression
• A history of tumor or malignancy
• Coagulation disorders
• Chromosomal abnormalities that limit expected survival to < 1 year
• Abnormal lab values that may increase the risk of the study procedure (WBC >20,000 cells/ul or < 1,000 cells/ul; platelet count < 50,000 cells/ul; Hgb < 8.0 gm/dl; LFTs > 2x reference lab upper limit of normal) at the time of screening
• Subjects with conduction abnormalities, including atrioventricular block and bundle branch blocks
• Ventricular arrhythmias due to antiarrhythmic pharmacological therapy

• Maximum Eligible Age: 1 Year
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ventrix, Inc.

INDUSTRY

Sponsor Role: collaborator

University of California, San Diego

OTHER

Sponsor Role: collaborator

Emory University

OTHER

Sponsor Role: lead

Responsible Party

William T. Mahle

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

William Mahle, MD

Role: PRINCIPAL_INVESTIGATOR

Emory University

Locations

Children's Healthcare of Altanta

Atlanta, Georgia, United States

Countries

United States

Central Contacts

William Mahle, MD

Role: CONTACT

MahleW@kidsheart.com

404-256-2593

Other Identifiers

STUDY00006965

Identifier Type: -

Identifier Source: org_study_id