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New treatment option for Danon Disease
Official title A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
ClinicalTrials.gov ID: NCT06092034
What this study is testing
What is RP-A501?
RP-A501 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for danon disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 8 and older, men only
You may be able to join if
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- Male.
- Age ≥8 years.
- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
- Abnormal thickening of Left ventricular wall,
You likely can't join if
- Anti-AAV9 neutralizing antibody titer \>1:40.
- Severe heart failure or requirement for advanced therapies.
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome...
- Prior cardiac or other organ (lung, liver, other) transplantation.
See the full eligibility criteria
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- Male.
- Age ≥8 years.
- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
- Abnormal thickening of Left ventricular wall,
- Left ventricular ejection fraction (LVEF) ≥ 50%.
- New York Heart Association (NYHA) Class II to III.
- High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)
- Ability to comply with study procedures including investigational therapy and follow-up evaluations. Key
- Anti-AAV9 neutralizing antibody titer \>1:40.
- Severe heart failure or requirement for advanced therapies.
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
- Prior cardiac or other organ (lung, liver, other) transplantation.
The study team makes the final eligibility decision.
Where it's taking place
- La Jolla, California, United States
- Boston, Massachusetts, United States
- Philadelphia, Pennsylvania, United States
- Munich, Germany
- Florence, Italy
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 8 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include La Jolla, California, United States; Boston, Massachusetts, United States; Philadelphia, Pennsylvania, United States; Munich, Germany; Florence, Italy; London, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.