Effect of Venglustat in Patients With Renal Impairment

Genzyme

Status and phase

Completed

Phase 1

Conditions

Healthy Volunteers

Polycystic Kidney, Autosomal Dominant

Treatments

Drug: Venglustat GZ/SAR402671

Study type

Interventional

Funder types

Industry

Identifiers

NCT03687554

POP14499

U1111-1205-3215 (Other Identifier)

Details and patient eligibility

About

Primary Objective:

To study the effect of mild, moderate and severe renal impairment on the pharmacokinetics (PK) of Venglustat following a single dose.

Secondary Objective:

To assess the tolerability of Venglustat given as a single dose in subjects with mild, moderate and severe renal impairment in comparison with matched subjects with normal renal function.

Full description

Approximately 41 days, including a 21-day screening period, a 1-day treatment period, followed by a 9-day period of plasma sampling for assessment of primary endpoints.

Enrollment

24 patients

Sex

All

Ages

18 to 79 years old

Volunteers

Accepts Healthy Volunteers

Inclusion criteria

For all Subjects:

Male and/or female subjects, between 18 and 79 years of age, inclusive.
Body weight between 50.0 and 115.0 kg, inclusive, if male, and between 40.0 and 100.0 kg, inclusive, if female, body mass index between 18.0 and 34.9 kg/m2, inclusive
Normal electrocardiogram (ECG)
Having given written informed consent prior to undertaking any study-related procedure
Not under any administrative or legal supervision
Male subject, whose partners are of childbearing potential (including lactating women), must accept to use, during sexual intercourse, a double contraception method according to the following algorithm: (condom) plus (spermicide or intra-uterine device or hormonal contraceptive) from the inclusion up to 4 months after the last dosing
Male subject, whose partners are pregnant, must use, during sexual intercourse, a condom from the inclusion up to 4 months after the last dosing
Male subject has agreed not to donate sperm from the inclusion up to 4 months after the last dosing
Female subject must use a double contraception method including a highly effective method of birth control from at least 30 days prior to the inclusion to 30 days after the last IMP administration, except if she has undergone sterilization (documented) at least 3 months earlier or is postmenopausal

Specific for subjects with renal impairment:

Stable chronic renal impairment
Vital signs and laboratory parameters within acceptable range for subjects with renal impairment

Specific for matched healthy subjects:

Normal renal function
Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical exam)
Normal vital signs and laboratory parameters

Exclusion criteria

Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month)
Blood donation, any volume, within 2 months before inclusion
Symptomatic postural hypotension, irrespective of the decrease in blood pressure, or asymptomatic postural hypotension judged clinically relevant by the Investigator
Any significant change in chronic treatment medication within 14 days before inclusion
Any drug which could impact by any mechanism of action, the pharmacokinetics of the investigational medicinal product, including moderate and strong cytochrome P3A (CYP3A) inhibitors or inducers; any vaccination within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion
Positive result on any of the following tests: hepatitis B surface (HBs Ag) antigen, anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti HIV2 Ab)
Positive result on urine drug screen or plasma alcohol test
Active hepatitis, hepatic insufficiency
If female, pregnancy [defined as positive β-Human Chorionic Gonadotropin (β-HCG) blood test], breast-feeding

Specific for subjects with renal impairment:

Uncontrolled clinically relevant cardiovascular, pulmonary, gastrointestinal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, or infectious disease, or signs of acute illness
Acute renal failure (de novo or superimposed on preexisting chronic renal impairment), nephrotic syndrome
History of or current hematuria of urologic origin that limits the subject's participation in the study
Subjects requiring dialysis during the study

Specific for matched healthy controls:

Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female) or infectious disease, or signs of acute illness

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.