Polycystic kidney, autosomal dominant
Conditions
Interventions
Venglustat:Venglustat
Sponsors
Beijing Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Chinese male or female (approximately 50% of each gender) subjects, between 18 and 45 years of age, inclusive. - Body weight between 55.0 and 100 kg, inclusive, if male, and between 50.0 and 90.0 kg, inclusive, if female; body mass index (BMI) between 19.0 and 27.9 kg/m2, inclusive. - Normal vital signs, electrocardiogram, and laboratory parameters. - Male or female are required to either practice true abstinence consistent with their preferred and usual lifestyle, or use double contraceptive methods for the entire duration of the treatment until 6 weeks after the IMP dosing for women and until 90 days for men.
Exclusion criteria
Exclusion criteria: - Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, or infectious disease, or signs of acute illness which might affect the drug safety, tolerability, or pharmacokinetics, or safety of a participant during the study - Frequent (more than once a month) headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month) - Blood donation, any volume, within 3 months before inclusion. - Symptomatic postural hypotension, irrespective of the decrease in blood pressure, or asymptomatic postural hypotension defined as a decrease in SBP =20 mmHg within 3 minutes when changing from supine to standing position. - Presence or history of drug hypersensitivity, or allergic disease diagnosed and treated by a physician. - History of within 6 months of the study or presence of drug or alcohol abuse (alcohol consumption more than 40 g per day on a regular basis, which is about 500 mL red wine or 1000 mL beer or 100 mL 50% white wine). - Smoking regularly more than 5 cigarettes or equivalent per week within 6 months of the study, unable to stop smoking during the study (occasional smoker can be enrolled). Excessive consumption of beverages containing xanthine bases (more than 4 cups or glasses per day). - If female, pregnancy (defined as positive beta human chorionic gonadotropin [ß-HCG] test), breast-feeding. - Any medication (including St Johns Wort, herbal supplements or Chinese traditional medicine, moderate and strong CYP3A4 inhibitors or inducers) within 14 days before inclusion or within 5 times the elimination half-life or PD half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy; 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 antigen (HBs Ag), anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus (anti-HIV) antibodies. - Positive result on urine drug screen (amphetamines/methamphetamines, ketamine, benzodiazepines, cannabinoids, cocaine, opiates). - Positive alcohol breath test. - Any consumption of citrus (grapefruit, orange, etc) or their juices within 5 days before inclusion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Assessment of pharmacokinetic (PK) parameter of venglustat: Cmax;Assessment of PK parameter of venglustat: tmax;Assessment of PK parameter of venglustat: AUClast;Assessment of PK parameter of venglustat: AUC;Assessment of PK parameter of venglustat: AUC0-24;Assessment of PK parameter of venglustat: MRT;Assessment of PK parameter of venglustat: T1/2z;Assessment of PK parameter of venglustat: Vss/F;Assessment of PK parameter of venglustat: CL/F; | — |
Secondary
| Measure | Time frame |
|---|---|
| Assessment of safety-adverse events (AE); | — |
Countries
China
Contacts
Public ContactAixin Shi
Beijing Hospital
Outcome results
None listed