A Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients


About this study

The primary purose of this study is to determine the effect of venglustat on the rate of total kidney volume (TKV) growth and estimated glomerular filtration rate (eGFR) decline in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria: 

  • Male or female adult with Autosomal Dominant Polycystic Kidney Disease (ADPKD) with age at the time the consent is signed:
    • between 18 to 50 years (both inclusive) for patients from Stage 1;
    • between 18 to 50 years (both inclusive) for patients from Stage 2 with eGFR between 45 and 89.9 mL/min/1.73 m^2 during screening period*;
    • between 18 to 55 years (both inclusive) for patients from Stage 2 with eGFR between 30 and 44.9 mL/min/1.73 m^2 during screening period*.
  • Diagnosis of AKPKD in patients with a family history will be based on unified Pei criteria. In the absence of a family history, the diagnosis will 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) must be 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]) for Stage 1.
  • Estimated glomerular filtration rate between 30 to 89.9 mL/min/1.73 m^2 during screening period* (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) for Stage 2.
  • *Eligibility will 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 patient.
  • Able to read, comprehend, and respond to the study questionnaires.
  • Patient has given voluntary written informed consent before performance of any study related procedures not part of standard medical care.
  • Patient does not have access to tolvaptan at the time of study start or tolvaptan is not indicated for treatment of patient according to treating physician (patient does not meet recommended criteria for treatment, refuses to initiate or does not tolerate treatment with tolvaptan).
  • The patient, if female of childbearing potential, must have 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 patients of childbearing potential and male patients must 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 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:

  • Systolic blood pressure > 160 mm Hg at Run-in and Baseline visits.
  • Administration within 3 months prior to the screening visit of tolvaptan or other Polycystic Kidney Disease-modifying agents (somatostatin analogues).
  • Current participation in another investigational interventional study or use of investigational medicinal product (IMP), within 3 months or 5 half lives, whichever is longer, before randomization.
  • The patient has a positive result of any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti HIV1 and anti HIV2 Ab). Patients with a positive hepatitis B surface antibody (HBsAb) test are eligible if other criteria are met (ie, negative tests for: HBsAg, hepatitis B core antibody [HBcAb]). Patients immune due to natural infection (positive hepatitis B surface antigen (HBsAb), negative hepatitis B surface antibody (HBsAg) and positive hepatitis B core antibody [HBcAb]) are eligible if they have negative HBV DNA test.
  • A history of drug and/or alcohol abuse within the past year prior to the screening visit.
  • The patient is scheduled for in-patient hospitalization including elective surgery, during the study.
  • The patient has a clinically significant, uncontrolled medical condition that, in the opinion of the investigator, would put the safety of the subject 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.
  • The patient, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (eg, has contraindications to pupillary dilation or unable to undergo magnetic resonance imaging (MRI) [For example: patient's weight exceeds weight capacity of the MRI, ferromagnetic metal prostheses, aneurysm clips, severe claustrophobia, large abdominal/back tattoos, etc]).
  • Any country-related specific regulation that would prevent the patient from entering the study.
  • The patients did not adhere to treatment (< 70% compliance rate) in the run-in.
  • The patient has, according to World Health Organization (WHO) Grading, a cortical cataract > one-quarter of the lens circumference (Grade cortical cataract-2 [COR-2]) or a posterior subcapsular cataract >2 mm (Grade posterior subcapsular cataract-2 [PSC-2]). Patients with nuclear cataracts will not be excluded.
  • 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 randomization. This also includes the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting venglustat administration.
  • The patient is pregnant, or lactating.
  • Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) 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 3mg/dl (51 μmol/L) with conjugated bilirubin less than 20% of the total bilirubin fraction.
  • Presence of severe depression as measured by Beck Depression Inventory-II (BDI-II) >28 and/or a history of a major affective disorder within 1 year of the screening visit.
  • Known hypersensitivity to venglustat or any component of the excipients.
  • The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.


Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Vicente Torres, M.D., Ph.D.

Open for enrollment

Contact information:

Kathleen Leistikow

(507) 266-1316


More information


Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

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