LY3884961 for Gaucher Disease
Phase-Based Progress Estimates
Effectiveness
Safety
Lysosomal Rare Disorders Research and Treatment Center, Fairfax, VA
Gaucher Disease
LY3884961 - BiologicalEligibility
18 - 65
All Sexes
What conditions do you have?
Select
Eligibility
18 - 65
All Sexes
What conditions do you have?
Select
Study Summary
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 into an expansion cohort. For each enrolled patient, the study will be approximately 5 years in duration, including up to a 45-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 up for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.
Treatment Effectiveness
Effectiveness Progress
Study Objectives
19 Primary · 7 Secondary · Reporting Duration: 5 years
5 years
Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Incidence and severity of clinically significant changes PR intervals as measured by 12-lead ECG
Incidence and severity of clinically significant changes QRS intervals as measured by 12-lead ECG
Incidence and severity of clinically significant changes QT intervals as measured by 12-lead ECG
Incidence and severity of clinically significant changes RR intervals as measured by 12-lead ECG
Incidence and severity of clinically significant changes in EuroQol Group 5 dimension, 5 level (EQ-5D-5L) questionnaire responses
Incidence and severity of clinically significant changes in Fatigue Severity Scale (FSS) questionnaire responses
Incidence and severity of clinically significant changes in GD1 Patient-Reported Outcome Measures (GD1-PROM)
Incidence and severity of clinically significant changes in heart rate as measured by 12-lead electrocardiogram (ECG)
Incidence and severity of clinically significant changes in physical examinations
Incidence and severity of clinically significant changes in results of abdominal MRI (liver volume)
Incidence and severity of clinically significant changes in results of abdominal MRI (spleen volume)
Incidence and severity of clinically significant changes in the bone marrow burden (BMB) score results of bone MRI
Incidence and severity of clinically significant changes in waist circumference
Incidence and severity of clinically significant changes in weight over time
Time from LY3884961 to enzyme replacement therapy (ERT)/substrate reduction therapy (SRT) discontinuation, if applicable
Time from discontinuation of ERT/SRT to re-initiation of ERT/SRT
Up to 24 months
Change in ELISPOT GCase or rAAV9
Change in anti-AAV9 antibody titers in blood
Change in anti-GCase antibody titers in blood
Up to 60 months
Change and percent change from baseline in spleen volume (MN) as determined by centrally read MRI (magnetic resonance imaging)
Change from baseline in GCase enzyme activity levels in blood
Change from baseline in GCase protein levels in blood
Change from baseline in GluSph levels in blood
Change from baseline in platelet count
Up to Month 3
Viral shedding in saliva, urine or stool, as measured by qPCR
Trial Safety
Safety Progress
Trial Design
4 Treatment Groups
High Dose LY3884961
1 of 4
Low Dose LY3884961
1 of 4
Medium Dose LY3884961
1 of 4
Expansion cohort
1 of 4
Experimental Treatment
15 Total Participants · 4 Treatment Groups
Primary Treatment: LY3884961 · No Placebo Group · Phase 1 & 2
High Dose LY3884961Experimental Group · 4 Interventions: Prednisone, Methylprednisolone, Sirolimus, LY3884961 · Intervention Types: Drug, Drug, Drug, Biological
Low Dose LY3884961Experimental Group · 4 Interventions: Prednisone, Methylprednisolone, Sirolimus, LY3884961 · Intervention Types: Drug, Drug, Drug, Biological
Medium Dose LY3884961Experimental Group · 4 Interventions: Prednisone, Methylprednisolone, Sirolimus, LY3884961 · Intervention Types: Drug, Drug, Drug, Biological
Expansion cohortExperimental Group · 4 Interventions: Prednisone, Methylprednisolone, Sirolimus, LY3884961 · Intervention Types: Drug, Drug, Drug, Biological
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisone
2020
Completed Phase 4
~2450
Methylprednisolone
2015
Completed Phase 4
~2370
Sirolimus
2013
Completed Phase 4
~2750
Trial Logistics
Trial Timeline
Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 5 years
Closest Location: Lysosomal Rare Disorders Research and Treatment Center · Fairfax, VA
N/AFirst Recorded Clinical Trial
1 TrialsResearching Gaucher Disease
0 CompletedClinical Trials
Who is running the clinical trial?
Eli Lilly and CompanyIndustry Sponsor
2,428 Previous Clinical Trials
3,118,229 Total Patients Enrolled
1 Trials studying Gaucher Disease
15 Patients Enrolled for Gaucher Disease
Prevail TherapeuticsLead Sponsor
3 Previous Clinical Trials
54 Total Patients Enrolled
1 Trials studying Gaucher Disease
15 Patients Enrolled for Gaucher Disease
Sarah Neuhaus, DOStudy DirectorPrevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company
Eligibility Criteria
Age 18 - 65 · All Participants · 10 Total Inclusion Criteria
Mark “yes” if the following statements are true for you:You have thrombocytopenia, with a platelet count of < 100 × 103 per μL.
Bone marrow infiltration as defined by total BMB score ≥ 7 on MRI.
You have osteopenia or osteoporosis.
You have two GBA1 mutations confirmed by the central laboratory.
Splenomegaly with spleen volume ≥ 3 MN as evaluated by centrally read abdominal MRI.
You have hepatomegaly with liver volume ≥ 1.2 MN as evaluated by centrally read abdominal MRI.
Patient has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations.
About The Reviewer
Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.
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