Tests a gene therapy, RP-A501, in boys with Danon disease
Tests the gene therapy RP-A501 in male patients with Danon disease.
Plain-language title generated from the registry record
Registry title: A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Key facts
- Where
- Florence, ItalySee the site
- Phase
- Phase 2
Where in Italy
- Meyer Children's HospitalFlorence, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Rocket Pharmaceuticals Inc.
- Trial code
- NCT06092034
- Record updated
- 6 October 2026
- Original description
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon disease.Read full original descriptionShow less
Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon disease.
Inclusion: 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) ≤ 12 months prior to screening; Ability to comply with study procedures including investigational therapy and follow-up evaluations.
Exclusion: Anti-AAV9 neutralizing antibody titer >1:40; Severe heart failure or requirement for advanced therapies (requirement for intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment, presence or requirement for mechanical circulatory support (MCS), and presence or requirement for mechanical ventilation); Prior cardiac or other organ (lung, liver, other) transplantation; History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.