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About
Primary Objective:
To determine the effect of venglustat on the rate of total kidney volume (TKV) growth (Stage 1) and estimated glomerular filtration rate (eGFR) decline in participants at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) (Stage 2).
Secondary Objectives:
Full description
Study duration per participant was 26 months (maximal) that included a screening period of 15 days, run-in period of 2 weeks, a 24-month treatment period, and a follow-up 30 days after final dose of investigational medicinal product (IMP).
Enrollment
Sex
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Volunteers
Inclusion criteria
Male or female adult with ADPKD with age at the time the consent was signed:
Diagnosis of ADPKD in participants with a family history would be based on unified Pei criteria. In the absence of a family history, the diagnosis would be based on the presence of renal cysts bilaterally, totaling at least 20, in the absence of findings suggestive of other cystic renal diseases.
Mayo Imaging Classification of ADPKD Class 1C, 1D or 1E**
**Total kidney volume (TKV) had confirmed by a central reader prior to Visit 3.
Estimated glomerular filtration rate between 45 to 89.9 mL/min/1.73 m^2 during screening period* (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation) for Stage 1.
Estimated glomerular filtration rate between 30 to 89.9 mL/min/1.73 m^2 during screening period* (CKD-EPI equation) for Stage 2.
*Eligibility would be confirmed by eGFR value from one of the two first pre-randomization eGFR measurements.
Stable treatment regimen of antihypertensive therapy for at least 30 days prior to the screening visit for hypertensive participant.
Able to read, comprehend, and respond to the study questionnaires.
Participant had given voluntary written informed consent before performance of any study related procedures not part of standard medical care.
Participant had no access to tolvaptan at the time of study start or tolvaptan was not indicated for treatment of participant according to treating physician (participant does not meet recommended criteria for treatment, refuses to initiate or does not tolerate treatment with tolvaptan).
The participants, if female of childbearing potential, must have had a negative blood pregnancy test (β-human chorionic gonadotropin [β-hCG]) at the screening visit and a negative urine pregnancy test at the baseline visit.
Female participants of childbearing potential and male participants must have had agreed to practice true abstinence in line with their preferred and usual lifestyle or to use double-contraceptive methods (including a highly effective method of contraception for female participants of childbearing potential) for the entire duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of study drug.
Exclusion criteria
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Primary purpose
Allocation
Interventional model
Masking
478 participants in 5 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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