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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
• 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.
Duke University Health System
Durham, North Carolina, United States
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States
Westmead Hospital-Cnr Hawkesbury and Darcy Rds
Westmead, New South Wales, Australia
SphinCS Clinical Science for LSD
Höchheim, Germany
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
Zaragoza, Spain
Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) with AE's graded as mild, moderate, or severe.
Time frame: 5 years
Spleen volume
Change and percent change from baseline
Time frame: 5 years
Platelet count
Change from baseline
Time frame: 5 years
GCase levels
Change from baseline
Time frame: 5 years
GlcSph levels
Change from baseline
Time frame: 5 years
Discontinuation of enzyme replacement therapy (ERT)/substrate reduction therapy (SRT)
Time from dosing to discontinuation of ERT/SRT
Time frame: 5 years
Re-initiation of ERT/SRT (if necessary)
Time to development of criteria requiring re-initiation of ERT/SRT (if necessary)
Time frame: 5 years
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