NCT05819359

Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in Subjects With Parkinson's Disease With a Pathogenic Variant in the Glucocerebrosidase (GBA1) Gene

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 237 (estimated)
• Sponsor: Bial R&D Investments, S.A. · Industry
• Sex: All
• Age: 35 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this randomized, double-blind, placebo-controlled study is to assess the efficacy of BIA 28-6156 over placebo in delaying clinical meaningful motor progression over 78 weeks in subjects with Parkinson's disease who have a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD).

Detailed description

This is a 2-part (Part A \[Genetic Screening\] and Part B \[Double-Blind Treatment\]), Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, pharmacodynamics, and pharmacokinetics of 2 fixed dose levels of BIA 28-6156 (10 and 60 mg/day) in approximately 237 subjects with genetically confirmed GBA-PD. Part A (Genetic Screening) will identify individuals with a PD risk-associated variant in the GBA1 gene for potential enrolment into Part B (Double-Blind Treatment) of the study. Part B will consist of a screening period to ensure that all protocol inclusion/exclusion criteria for Part B of the study are met (up to 5 weeks). After screening period, eligible subjects will be randomized into 1 of 3 treatment arms (BIA 28-6156 10 mg/day, BIA 28-6156 60 mg/day, or placebo) in a 1:1:1 ratio, and enter a double-blind treatment period up to 78 weeks, followed by a 30-day (4 weeks) of safety follow-up period. Subjects must be receiving a stable dose of PD medication for at least 30 days before screening (for Part B \[Double-Blind Treatment\]) and will continue to receive their usual PD medications throughout the study.

Conditions

• Parkinson's Disease

Interventions

Type	Name	Description
DRUG	BIA 28-6156 10 mg	BIA 28-6156 10 mg, once daily, oral administration.
DRUG	BIA 28-6156 60 mg	BIA 28-6156 60 mg, once daily, oral administration.
DRUG	Placebo	Placebo, once daily, oral administration.

Timeline

• Start date: 2023-03-31
• Primary completion: 2026-03-31
• Completion: 2026-07-31
• First posted: 2023-04-19
• Last updated: 2025-12-10

Locations

95 sites across 11 countries: United States, Canada, France, Germany, Italy, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study