Phase 2 Cardiomyopathy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People who have been confirmed to carry a harmful or likely harmful change (variant) in a gene called LAMP2
- People who are male
- People who are 8 years of age or older
- People whose heart shows abnormal thickening of the left ventricular wall (a part of the heart muscle)
- People whose heart is still pumping with at least 50% efficiency, as measured by a test called an ejection fraction
- People whose heart failure symptoms fall into a moderate range, classified as New York Heart Association (NYHA) Class II or III — meaning symptoms such as shortness of breath or fatigue with moderate or minimal physical activity (confirm with trial site)
- People who have a blood marker of heart stress (called high sensitivity Troponin I) measured at 20% or more above the normal upper limit
- People who are able to follow the study schedule, including receiving the study treatment and attending all follow-up visits
Who may not be able to join:
- People whose blood shows a high level of antibodies against a specific virus carrier (called AAV9) used in the study, at a level above 1:40 (confirm with trial site)
- People who have severe heart failure or who require advanced heart failure treatments
- People who have a history of blood clots inside the heart, stroke, a mini-stroke (TIA), a heart attack, or unstable chest pain caused by reduced blood flow to the heart
- People who have previously had a heart transplant or a transplant of another organ such as a lung or liver
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Joseph Rossano, MD, Children's Hospital of Philadelphia
Phone: 646-627-0033
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
Can't join this trial?
Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.