A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

Part of paid clinical trials in Los Angeles, California.

Sponsor
Prevail Therapeutics
Study ID
NCT05487599
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Gaucher Disease
  • Gaucher Disease, Type 1

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • LY3884961 — GENETIC
    • LY3884961 is a replication-incompetent recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.

Study Details

Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD. Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients. Following this, up to 6 patients may be enrolled in an expansion cohort. For each enrolled patient, the study will be approximately 5 years in duration, including up to a 60-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.

Key Dates

Start date
Dec 20, 2022
Status verified
May 2026
Primary completion
Aug 30, 2032
Completion
Aug 30, 2032

Study Design

Enrollment
15 participants (estimated)
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: LY3884961
    LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.

Primary Outcome Measure

Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: 5 years ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Cedars-SinaiLos AngelesCalifornia90048
Bobby Marker, CCRP
[email protected]
310-423-0901
Ann and Robert H Lurie Children's Hospital of ChicagoChicagoIllinois60611
Carolyn Rasmussen
[email protected]
312-227-6763
Duke University Health SystemDurhamNorth Carolina27710-3017
Gretchen Nichting
[email protected]
919-660-0757
Lysosomal & Rare Disorders Research and Treatment CenterFairfaxVirginia22030-6066
Lauren Noll
[email protected]
571-732-4655

Find similar trials in Los Angeles, CA

By research site
Cedars-Sinai· Los Angeles, CAAnn and Robert H Lurie Children's Hospital of Chicago· Chicago, ILDuke University Health System· Durham, NCLysosomal & Rare Disorders Research and Treatment Center· Fairfax, VA

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