Conditions: Fabry Disease
Keywords: Venglustat
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 11
Healthy volunteers: no

Brief summary:
Primary Objective:

To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.

Detailed description:
The total duration of study per participant was 7 to 8 months for participants who entered a planned extension study and approximately 13 to 14 months for participants who did not enter a planned extension study. A 2-year extension study was planned for eligible participants.

Arms:
arm 1: GZ/SAR402671 15 milligram (mg) once daily orally for 26 weeks.

Interventions: intervention 1: GZ/SAR402671
Intervention descriptions: intervention 1: Pharmaceutical form: Capsule; Route of administration: Oral

Protocol document: