Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD
Research type
Research Study
Full title
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
IRAS ID
1006602
Contact name
Raquel Costa
Contact email
Sponsor organisation
BIAL R&D Investments S.A.
Eudract number
2022-501783-18
Research summary
BIA 28-6156 (investigational medicine) is being studied to determine if it can treat Parkinson's Disease (PD) by activating an enzyme called beta-glucocerebrosidase (Gcase) in PD patients who have a mutation in the glucocerebrosidase 1 (GBA1) gene (GBA-PD). Gcase is a lysosomal enzyme that is encoded by the GBA1 gene.
The efficacy, safety, tolerability, pharmacodynamics, and pharmacokinetics (PK) of 2 fixed doses of BIA 28-6156, as compared with placebo, for participants with GBA-PD, will be studied in this Clinical Trial.
The Trial is divided into the below 2 parts:
- Part A: Genetic Screening
- Two visits to identify patients with a PD risk-associated variant in the GBA1 gene for potential enrollment into Part B
- Part B: Double-Blind Treatment
- Screening Period - To check eligibility to join the study
- Treatment Period - 2 doses of BIA 28-6156. 237 participants randomized to one of two active treatments - 10mg tablet/day or 60 mg tablet/day - or matching placebo, in a 1:1:1 randomization.
- Safety follow-up period - To check participants overall health and safety
Part A/Visit 2 may be the same as Part B/Visit 1. Participant will sign the ICF for Part B & can begin the screening assessments for Part B.
Up to 3750 participants may undergo genetic screening in Part A of the study to randomize approximately 237 participants into Part B of the study. The study will take place at approximately 90 sites in North America and Europe.
The study medicine comes in a tablet form & is to be taken orally once daily, in the morning, with or without food for a maximum of 78 weeks.
Participation in the study will last up to 87 weeks, including screening (5 wks), double-blind treatment (78 wks), & safety follow-up (4 wks). Participation is voluntary. Participants will be expected to attend clinic visit whereby study assessments will take place e.g., blood and urine collection, vital signs monitoring, neurological examination, and completion of questionnaires.REC name
North of Scotland Research Ethics Committee 1
REC reference
23/NS/0052
Date of REC Opinion
8 Sep 2023
REC opinion
Further Information Favourable Opinion