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Gene Therapy Trial Report

Summary

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


NCTID NCT06092034 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Danon Disease
Disease Ontology Term DOID:0050437
Compound Name RP-A501
Sponsor Rocket Pharmaceuticals Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 14 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant LAMP2B
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 3.8E13 GC/kg
Dose 2 6.7E13 GC/kg
Dose 3 1.1E14 GC/kg (dose discontinued)

Study Record Dates


Current Stage Phase2
Submit Date 2023-10-11
Completion Date 2032-04
Last Update 2025-10-07

Participation Criteria


Eligible Age >=8 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
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 Exclusion Criteria: 1. Anti-AAV9 neutralizing antibody titer \>1:40. 2. Severe heart failure or requirement for advanced therapies. 3. History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina. 4. Prior cardiac or other organ (lung, liver, other) transplantation.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 6
Locations United States,Italy,United Kingdom,Germany

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates Clinical hold lifted August 2025

Resources/Links