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)

NCT06461676 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: STUDY00006965
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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

Conditions

Hypoplastic Left Heart Syndrome

Keywords

Stage II Glenn operation; HLHS

Outcome Measures

Primary Outcomes (9)

• Number of ventricular tachycardia events

  Number of sustained/symptomatic ventricular tachycardia incidences requiring intervention with inotropic support or anti-arrhythmics

  upto 30 days post operation

• Number of Cardiogenic shock events

  Cardiogenic shock (i.e. tissue hypoperfusion presented by hypotension due to decreased cardiac output, determined by rising lactate levels (5x normal levels in first 24 hours, 2.5x normal levels in first 30 days)

  Day 1, 30 days post operation

• Number of unplanned cardiovascular operation events

  Unplanned cardiovascular operation due to right ventricular intramyocardial injection site bleeding in the first 5 days after Stage II operation decreased cardiac output, determined by rising lactate levels (5x normal levels in first 24 hours, 2.5x normal levels in first 30 days)

  30 days post operation

• Number of patients requiring new permanent pacemaker

  Number of patients requiring new permanent pacemaker

  upto 30 days post operation

• Stroke or embolic event

  Stroke or embolic event to the brain determined by CT scan

  upto 30 days post operation

• Number of Adverse Events

  Number of adverse events occurring post op

  upto 30 days post operation

• Number of deaths

  Number of deaths occurring post op

  upto 30 days post operation

• Number of VentriGel products manufactured and delivered to subjects

  12 months

• Number of patients receiving Cardiac MRIs

  Baseline, 6 months, 12 months

Secondary Outcomes (9)

• Change in right ventricular ejection fraction

  Baseline, 6 months, 12 months post operation

• Change in right ventricular end diastolic volume

  Baseline, 6 months, 12 months post operation

• Change in right ventricular end systolic volume

  Baseline, 6 months, 12 months post operation

• Change in tricuspid regurgitation

  Baseline, 6 months,12 months post operation

• Change in right ventricular function Fractional Area Change

  Baseline, 6 months,12 months post operation

Study Arms (1)

Ventrix Bio Extracellular Matrix

EXPERIMENTAL

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.

Drug: Ventrix Bio Extracellular Matrix

Interventions

Ventrix Bio Extracellular Matrix 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.

Also known as: VentriGel

Ventrix Bio Extracellular Matrix

Eligibility Criteria

• Age: Up to 1 Year
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Emory University: lead
• Ventrix, Inc.: collaborator
• University of California, San Diego: collaborator

Study Sites (1)

Children's Healthcare of Altanta

Atlanta, Georgia, 30322, United States

Location

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome

Condition Hierarchy (Ancestors)

Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• William Mahle, MD

  Emory University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

William Mahle, MD

CONTACT

404-256-2593; MahleW@kidsheart.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 28, 2024
• First Posted: June 17, 2024
• Study Start: August 1, 2025
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: March 10, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)