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

Study duration per participant is 26 months (maximal) per stage, including 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).

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 (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.