Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat

  • STATUS
    Recruiting
  • End date
    Nov 30, 2025
  • participants needed
    640
  • sponsor
    Sanofi
Updated on 2 May 2021

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 ADPKD.

Secondary Objective:

  • To determine the effect of early versus delayed treatment with venglustat on the renal function (estimated glomerular filtration rate [eGFR]).
  • 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).

Description

Study duration per participant is 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).

Details
Condition Congenital Cystic Kidney Disease
Treatment Venglustat GZ402671
Clinical Study IdentifierNCT04705051
SponsorSanofi
Last Modified on2 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age greater than or equal to 18 yrs?
Gender: Male or Female
Do you have any of these conditions: Do you have Congenital Cystic Kidney Disease??
Male or female adult with ADPKD who has completed the treatment period in Stage 1 or Stage 2 of Study EFC15392
The patient has an eGFR >30 mL/min/1.73 m2
measured at Visit 11 of the EFC15392 study for participant enrolled in the LTS15823 study at the time of Visit 12 (Month 24; end-of treatment visit) of the EFC15392 study
measured at Screening visit for participant enrolled in the LTS15823 study not concomitantly to the Visit 12 (Month 24; end-of treatment visit) of the EFC15392 study
Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
Male participants must agree to practice true abstinence in line with their preferred and usual lifestyle or to use double-contraceptive methods for the entire duration of the study and for at least 90 days following their last dose of IMP
Female participants must have a negative urine pregnancy test at the Baseline visit and agree 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 the entire duration of the study and for at least 6 weeks following their last dose of IMP
Capable of giving signed informed consent before performance of any study related procedures not part of standard medical care
Able to read, comprehend, and respond to the study questionnaires

Exclusion Criteria

For participants who have lag phase between the end of the EFC15392 study and Screening visit (Visit 0) in the LTS15823 study
The patient has a new clinically significant, uncontrolled medical condition that, in the opinion of the Investigator, would put the safety of the patient at risk through participation, or which would affect the efficacy or safety analysis if the condition exacerbated during the study, or that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
A history of drug abuse and/or alcohol abuse or alcohol dependence during the lag phase between the end of the EFC15392 study and Screening visit (Visit 0) in the LTS15823 study when applicable
Administration of tolvaptan or other polycystic kidney disease-modifying agents (somatostatin analogues) within 3 months prior to the Screening visit (Visit 0) in the LTS15823 study when applicable
The patient is currently receiving potentially cataractogenic medications, including a chronic regimen (more frequently than every 2 weeks) of any route of corticosteroids (including medium and high potency topical steroids), or any medication that may cause cataract, according to the Prescribing Information
The patient has received strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half lives, whichever is longer, prior to the Baseline visit (including consumption of grapefruit-containing products within 72 hours of starting venglustat administration)
Participation in another investigational interventional study or use of IMP, within 3 months or 5 half-lives, whichever is longer, before the Baseline visit (Visit 1) except participation in the EFC15392 study when applicable
Liver enzymes (alanine aminotransferase /aspartate aminotransferase) or total bilirubin >2 times the upper limit of normal unless the patient has the diagnosis of Gilbert syndrome. Patients with the Gilbert syndrome should have no additional symptoms or signs which suggest hepatobiliary disease and serum total bilirubin level no more than 3 mg/dL (51 mol/L) with conjugated bilirubin less than 20% of the total bilirubin fraction
For participants with or without lag phase between the end of EFC15392 study
and entry into LTS15823 study
The patient is pregnant or lactating
Presence of severe depression as measured by Beck Depression Inventory II >28 at Visit 1 (for participants enrolled in the LTS15823 study at the time of the end of treatment visit of the EFC15392 study) or at Visit 0 (for participants enrolled in the LTS15823 study after the end-of-treatment visit of the EFC15392 study)
Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
The above information is not intended to contain all considerations relevant
to a patient's potential participation in a clinical trial
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