Adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene for Danon disease

Inclusion criteria

• {"criterion_text":"- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene."}
• {"criterion_text":"- Male gender."}
• {"criterion_text":"- Age ≥8 years."}
• {"criterion_text":"- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: a. For subjects < 18 years, z-score of the left ventricular posterior wall or interventricular septum at end diastole ≥+ 2, and for subjects ≥18 years, left ventricular posterior wall or interventricular septum at end diastole >13 mm (>12 mm if family history of clinically significant Danon disease), b. Left ventricular ejection fraction (LVEF) ≥ 50%."}
• {"criterion_text":"- New York Heart Association (NYHA) Class II to III."}
• {"criterion_text":"- hsTnI ≥20% above the ULN"}
• {"criterion_text":"- Ability to comply with study procedures including investigational therapy and follow-up evaluations."}
• {"criterion_text":"- Has received approved vaccination against Neisseria meningitidis ≥ 6 weeks before administration of RP-A501."}

Exclusion criteria

• {"criterion_text":"- Anti-AAV9 neutralizing antibody titer >1:40."}
• {"criterion_text":"- Intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment."}
• {"criterion_text":"- Presence or requirement for mechanical circulatory support (MCS)."}
• {"criterion_text":"- Presence or requirement for mechanical ventilation."}
• {"criterion_text":"- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina."}
• {"criterion_text":"- Prior cardiovascular (CV) surgery, percutaneous coronary intervention (PCI), or valvuloplasty."}
• {"criterion_text":"- Greater than moderate valvular stenosis or regurgitation on most recent echocardiographic assessment."}
• {"criterion_text":"- Prior cardiac or other organ (lung, liver, other) transplantation."}

Primary endpoints

• {"endpoint_text":"- Primary endpoint including myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI). Evaluation of efficacy associated with RP-A501.","definition_or_measurement_approach":"Primary endpoint comprises measurement of LAMP2 protein expression in myocardial tissue and assessment of left ventricular mass index (LVMI) to evaluate efficacy of RP-A501."}

Secondary endpoints

• {"endpoint_text":"- LAMP2 protein expression, LVMI, hsTnI, NT-proBNP, KCCQ, and NYHA class. Evaluation of efficacy associated with RP-A501.","definition_or_measurement_approach":"Assessments include myocardial LAMP2 expression, imaging-derived LVMI, serum biomarkers (hsTnI, NT-proBNP), patient-reported KCCQ scores and NYHA class to evaluate efficacy."}
• {"endpoint_text":"- Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization. Evaluation of efficacy associated with RP-A501.","definition_or_measurement_approach":"Event-free survival measured as time to first event (death, heart transplant, MCS, or heart failure hospitalization)."}
• {"endpoint_text":"- Incidence, severity and duration of treatment emergent safety events. Evaluation of safety.","definition_or_measurement_approach":"Safety assessed by recording incidence, severity and duration of treatment-emergent adverse events (TEAEs) and other safety signals."}

Germany

Earliest CTIS Part Ii Submission Date

07-12-2023

Latest Decision Or Authorization Date

25-02-2026

Processing Time Days

811

Number Of Sites

1

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

01-08-2024

Latest Decision Or Authorization Date

25-02-2026

Processing Time Days

573

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Rocket Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

CTI Clinical Trial and Consulting Services Europe GmbH

Responsibilities

sponsorDuties codes: [{"id":822914,"code":"1"},{"id":822915,"code":"10"},{"id":822916,"code":"12"},{"id":822917,"code":"5"},{"id":822918,"code":"6"}]

Name

Premier Research s.r.o.

Responsibilities

sponsorDuties codes: [{"id":822910,"code":"8"}]

Name

Eresearchtechnology Inc.

Responsibilities

sponsorDuties: [{"id":822920,"code":"15","value":"Central Imaging"}]

Third parties

• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"sponsorDuties codes: [{\"id\":822913,\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"sponsorDuties codes: [{\"id\":822919,\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"sponsorDuties codes: [{\"id\":822914,\"code\":\"1\"},{\"id\":822915,\"code\":\"10\"},{\"id\":822916,\"code\":\"12\"},{\"id\":822917,\"code\":\"5\"},{\"id\":822918,\"code\":\"6\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties: [{\"id\":822920,\"code\":\"15\",\"value\":\"Central Imaging\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Slovakia","full_name":"Premier Research s.r.o.","duties_or_roles":"sponsorDuties codes: [{\"id\":822910,\"code\":\"8\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Fisher Bioservices Inc.","duties_or_roles":"sponsorDuties: [{\"id\":822912,\"code\":\"15\",\"value\":\"Laboratory biorepository\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Early Development Laboratories Inc.","duties_or_roles":"sponsorDuties codes: [{\"id\":822911,\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}