ClinicaliQ Trial Snapshot
- A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease — Recruiting • Phase II • Cardiology / Cardiovascular • NCT06092034.
- RP-A501 is a gene therapy using AAV9 vector to deliver the LAMP2B gene – designed to address the underlying genetic defect in Danon disease by restoring lysosomal function in affected tissues.
- Eligible patients are males with confirmed Danon disease – a rare X-linked lysosomal storage disorder; this Phase 2 trial evaluates both efficacy and safety in this population with limited current treatment options.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying
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.
Eligibility Snapshot
- Key Inclusion Criteria: 1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene. 2. Male. 3. Age ≥8 years. 4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: 1. Abnormal thickening of Left ventricular wall, 2. Left ventricular ejection fraction (LVEF) ≥ 50%. 5. New York Heart Association (NYHA) Class II to III. 6. High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN) 7. Ability to comply with study procedures including investigational therapy and follow-up evaluations. Key
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.