A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
NCT ID: NCT05885412
Last Updated: 2024-10-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
9 participants
INTERVENTIONAL
2023-08-29
2026-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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RP-A601
Single ascending dose of RP-A601 in 2 consecutive cohorts
RP-A601
RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)
Interventions
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RP-A601
RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)
Eligibility Criteria
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Inclusion Criteria
2. Capable and willing to provide signed informed consent
3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
5. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
6. PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
7. Left ventricular ejection fraction by echocardiogram or CMR ≥50%
Exclusion Criteria
2. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
3. Previous participation in a study of gene transfer or gene editing
4. Severe Right ventricular (RV) dysfunction
5. New York Heart Association (NYHA) Class IV heart failure.
18 Years
ALL
No
Sponsors
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Rocket Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Barry Greenberg, MD
Role: PRINCIPAL_INVESTIGATOR
University of California, San Diego
Locations
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University of California, San Diego
La Jolla, California, United States
Duke University
Durham, North Carolina, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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RP-A601-0323
Identifier Type: -
Identifier Source: org_study_id
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