Hypoplastic Left Heart SyndromePhase 1NCT06461676
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)
A Phase 1 study testing Ventrix Bio Extracellular Matrix in people with Hypoplastic Left Heart Syndrome, run by Emory University at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages Up to 1 year
- Conditions the study is for
- Hypoplastic Left Heart Syndrome
- What is being tested?
- Ventrix Bio Extracellular Matrix
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2025to Sep 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 Up to 1 year
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subjects with Hypoplastic Left Heart Syndrome (HLHS) requiring Stage II Glenn operation under one year of age
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Children's Healthcare of Altanta
Atlanta, Georgia, 30322
About this study
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
Read the 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
- Being tested
- Ventrix Bio Extracellular Matrix
- Keywords
- Stage II Glenn operation, HLHS
- Medical subject headings
- Hypoplastic Left Heart Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ventrix Bio Extracellular MatrixDrug
Also called VentriGel
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ventrix Bio Extracellular Matrix |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of ventricular tachycardia events
Number of sustained/symptomatic ventricular tachycardia incidences requiring intervention with inotropic support or anti-arrhythmics
Measured: 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)
Measured: 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)
Measured: 30 days post operation
Number of patients requiring new permanent pacemaker
Number of patients requiring new permanent pacemaker
Measured: upto 30 days post operation
Stroke or embolic event
Stroke or embolic event to the brain determined by CT scan
Measured: upto 30 days post operation
Number of Adverse Events
Number of adverse events occurring post op
Measured: upto 30 days post operation
Number of deaths
Number of deaths occurring post op
Measured: upto 30 days post operation
Number of VentriGel products manufactured and delivered to subjects
Measured: 12 months
Number of patients receiving Cardiac MRIs
Measured: Baseline, 6 months, 12 months
Secondary outcomes (9)
Change in right ventricular ejection fraction
This will be measured by serial echocardiograms and MRI scans
Measured: Baseline, 6 months, 12 months post operation
Change in right ventricular end diastolic volume
This will be measured by serial echocardiograms and MRI scans
Measured: Baseline, 6 months, 12 months post operation
Change in right ventricular end systolic volume
This will be measured by serial echocardiograms and MRI scans
Measured: Baseline, 6 months, 12 months post operation
Change in tricuspid regurgitation
This will be measured by serial echocardiograms and MRI scans. tricuspid regurgitation measured by tricuspid valve vena contracta width
Measured: Baseline, 6 months,12 months post operation
Change in right ventricular function Fractional Area Change
This will be measured by serial transthoracic echocardiogram (TTE)
Measured: Baseline, 6 months,12 months post operation
Change in right ventricular global longitudinal strain
This will be measured by serial transthoracic echocardiogram (TTE)
Measured: Baseline, 6 months,12 months post operation
Change in right ventricular global circumferential strain
This will be measured by serial transthoracic echocardiogram (TTE)
Measured: Baseline, 6 months,12 months post operation
Change in right ventricular Tricuspid Annular Plane Systolic Excursion (TAPSE)
This will be measured by serial transthoracic echocardiogram (TTE)
Measured: Baseline, 6 months,12 months post operation
Change in right atrial volume indexed
This will be measured by serial transthoracic echocardiogram (TTE)
Measured: Baseline, 6 months,12 months post operation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2024
- Study startAug 2025 (estimated)
- Primary completionSep 2028 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypoplastic Left Heart SyndromeStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Ventrix, Inc., University of California, San Diego
- Responsible party
- Principal investigator, William T. Mahle, Emory University
- Investigators
- William Mahle, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06461676
- Sponsor's protocol ID
- STUDY00006965
- Status last verified
- Mar 2025
- First submitted
- 28 May 2024
- Last updated
- 10 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06461676
- Acronym / sponsor ID
- STUDY00006965
- Conditions
- Hypoplastic Left Heart Syndrome
- Keywords
- Stage II Glenn operation, HLHS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Ventrix, Inc., University of California, San Diego
- Study start
- Aug 2025 (estimated)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Sep 2028 (estimated)
- First posted
- 17 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ventrix Bio Extracellular Matrix | Experimental | Drug: Ventrix Bio Extracellular MatrixTreatment 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 may qualify
- Ages
- Up to 1 year
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Subjects with Hypoplastic Left Heart Syndrome (HLHS) requiring Stage II Glenn operation under one year of age
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ventrix Bio Extracellular MatrixAlso: VentriGelVentriGel 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. | Drug | Ventrix Bio Extracellular Matrix |
Primary outcome
Number of ventricular tachycardia events
Time frame: upto 30 days post operation
Number of sustained/symptomatic ventricular tachycardia incidences requiring intervention with inotropic support or anti-arrhythmics
Number of Cardiogenic shock events
Time frame: Day 1, 30 days post operation
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)
Number of unplanned cardiovascular operation events
Time frame: 30 days post operation
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)
Number of patients requiring new permanent pacemaker
Time frame: upto 30 days post operation
Number of patients requiring new permanent pacemaker
Stroke or embolic event
Time frame: upto 30 days post operation
Stroke or embolic event to the brain determined by CT scan
Number of Adverse Events
Time frame: upto 30 days post operation
Number of adverse events occurring post op
Number of deaths
Time frame: upto 30 days post operation
Number of deaths occurring post op
Number of VentriGel products manufactured and delivered to subjects
Time frame: 12 months
Number of patients receiving Cardiac MRIs
Time frame: Baseline, 6 months, 12 months
9 secondary outcomes
Change in right ventricular ejection fraction
Time frame: Baseline, 6 months, 12 months post operation
Change in right ventricular end diastolic volume
Time frame: Baseline, 6 months, 12 months post operation
Change in right ventricular end systolic volume
Time frame: Baseline, 6 months, 12 months post operation
Change in tricuspid regurgitation
Time frame: Baseline, 6 months,12 months post operation
Change in right ventricular function Fractional Area Change
Time frame: Baseline, 6 months,12 months post operation
Change in right ventricular global longitudinal strain
Time frame: Baseline, 6 months,12 months post operation
Change in right ventricular global circumferential strain
Time frame: Baseline, 6 months,12 months post operation
Change in right ventricular Tricuspid Annular Plane Systolic Excursion (TAPSE)
Time frame: Baseline, 6 months,12 months post operation
Change in right atrial volume indexed
Time frame: Baseline, 6 months,12 months post operation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
William Mahle, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ventrix Bio Extracellular Matrix in people with Hypoplastic Left Heart Syndrome, run by Emory University at 1 site 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?
The sponsor lists 1 inclusion and 10 exclusion criteria. 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: opening soon, as of the sponsor's last update on 10 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact William Mahle, MD (404-256-2593, MahleW@kidsheart.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of ventricular tachycardia events (time frame: upto 30 days post operation). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Mar 2025.