A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

ConditionDanon Disease

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexMale

Age8+

SponsorRocket Pharmaceuticals Inc.

About this trial

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 criteria

Qualifiers

Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.

Male.

Age ≥8 years.

Abnormal thickening of Left ventricular wall,

Disqualifiers

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.