Multicenter, Open-label, Extension Study to Characterize the Long-term Efficacy and Safety of Early Versus Delayed Treatment With Venglustat (GZ/SAR402671) in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Trial Snapshot
- Phase
- Phase 3
- Status
- Terminated
- Sponsor
- Sanofi
- Enrollment
- 24
- Locations
- 11
- Primary Endpoint
- Percent Change in Total Kidney Volume (TKV)
Study Overview
Brief Summary
Primary Objective:
-To determine the effect of early versus delayed treatment with venglustat on the total kidney volume (TKV) in participants at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD).
Secondary Objective:
- To determine the effect of early versus delayed treatment with venglustat on the renal function (estimated glomerular filtration rate [eGFR] [Chronic Kidney Disease Epidemiology Collaboration {CKD-EPI} equation]).
- To characterize the safety profile of venglustat.
- To evaluate the effect of venglustat on the lens by ophthalmological examination.
- To evaluate the effect of venglustat on mood using Beck Depression Inventory-II (BDI-II).
Detailed Description
The planned study duration per participant was 25.5 months (maximal). Screening period (when applicable): up to 2 weeks. Core treatment period: 24 months. Follow-up: 30 days after final dose of the investigational medicinal product (IMP) (venglustat).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Venglustat
Participants were to be treated with venglustat 15 milligrams once daily orally for 24 months or until venglustat was commercially available, whichever came first.
Intervention: Venglustat GZ402671 (Drug)
Outcomes
Primary Outcomes
Percent Change in Total Kidney Volume (TKV)
Time Frame: From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823
Total kidney volume is a measure of disease progression in the ADPKD participants. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Due to the early termination of study LTS15823 and low enrollment numbers, efficacy analysis was not performed.
Secondary Outcomes
- Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)(From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)(From the first administration of the IMP in LTS15823 study up to the last IMP administration in LTS15823 study + 30 days (i.e., up to approximately 20 weeks))
- Change From Baseline in Lens Clarity During the Open-label Extension Treatment-emergent Period of LTS15823 Study(Baseline (EFC15392 study Baseline); from first administration of IMP up to last administration of IMP of open-label extension study LTS15823 + 30 days (i.e., up to approximately 20 weeks), in comparison to the EFC15392 study Baseline)
- Effect on Mood With Change From Baseline in Beck Depression Inventory (BDI-II) Score During the Open-label Extension Treatment-emergent Period of LTS15823 Study(Baseline (EFC15392 study Baseline); from first IMP administration up to 3 months in study LTS15823, in comparison to the EFC15392 study Baseline)