A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorPrevail Therapeutics
About this trial
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.
Eligibility criteria
Qualifiers
Age greater or equal to 18 years at the time of informed consent.
Bi-allelic pathogenic GBA1 variants must be centrally confirmed.
On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.
Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Disqualifiers
Clinically significant neurological signs and symptoms and/or behavioral disturbances.
Active and progressive bone disease expected to require surgical treatment in the next 6 months.
History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).
Splenomegaly > 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)
Trial design
Treatments tested in this trial
- LY3884961
Treatment groups
15 Participants
are divided into 1 treatment groupSponsors and collaborators
Prevail Therapeutics
Lead sponsor
Eli Lilly and Company
Collaborator
Source ClinicalTrials.gov