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


  • Study type

  • Study phase

  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 18-006865
    NCT ID: NCT03523728
    Sponsor Protocol Number: EFC15392

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. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria: 

  • Male or female adult with Autosomal Dominant Polycystic Kidney Disease (ADPKD) diagnosed by unified Pei criteria between ages of 18 to 50 years (both inclusive) at screening. 
  • Mayo Imaging Classification of ADPKD Class 1C, 1D or 1E (3). 
  • Estimated glomerular filtration rate between 45 to 90 mL/min/1.73 m2 at screening (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]).
  • 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 documented diagnosis of any of the following infections: hepatitis B, hepatitis C, human immunodeficiency virus 1 or 2. Patients with a positive hepatitis B surface antibody (HBsAb) test with a history of prior hepatitis B immunization are eligible if other criteria are met (ie, negative tests for: HBsAg, hepatitis B core antibody [HBcAb]).Patients with positive hepatitis B core antibody [HBcAb], negative HBsAg and negative hepatitis B surface antibody (HBsAb) tests are eligible if they have HBV DNA negative 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 (e.g., 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 us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

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

Open for enrollment

Contact information:

Lisa Bungum CCRP



More information


Publications are currently not available

Study Results Summary

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Supplemental Study Information

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