Phase 1 Pharmacokinetics and Safety Study of BIIB132 in Adults with SCA3
Research type
Research Study
Full title
A Phase 1, Blinded, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of BIIB132 Administered Intrathecally to Adults With Spinocerebellar Ataxia 3
IRAS ID
1004052
Contact name
Mathew Stagray
Contact email
Sponsor organisation
Biogen Idec Research Limited
Eudract number
2021-002223-37
ISRCTN Number
ISRCTN94357046
Research summary
Spinocerebellar ataxia3 (SCA3) is a rare, autosomal mainly inherited (condition can be passed down from parent to child) neurodegenerative disorder, and currently no specific approved therapy is available to slow clinical progression.
This is a Phase 1, First In Human, blinded (participant and doctor will not know which medication will be given) study where participants will be randomly assigned to either the study drug, i.e. BIIB132, or placebo (looks like the study drug but contains no active ingredient). This study is being carried out to find out more about the study drug, BIIB067 as a potential treatment for people with
SCA3. The study drug is administered Intrathecally (injection into the spinal canal) and approximately, 48 adult participants will be enrolled.The study comprises 5 arms (cohorts) and participants will be randomized in a 3:1 ratio (BIIB132:placebo). The 5 dose levels of BIIB132 that will be investigated are:
• Cohort 1: BIIB132 10 mg (8 participants in total)
• Cohort 2: BIIB132 20 mg (8 participants in total)
• Cohort 3: BIIB132 40 mg (8 participants in total)
• Cohort 4: BIIB132 70 mg (12 participants in total)
• Cohort 5: BIIB132 100 mg (12 participants in total)The frequency of study treatment administration is projected to be every 4 weeks (total of 4 doses) for participants in Cohorts 1 to 4 and every 4 weeks (total of 4 doses), every 8 weeks (total of 2 doses), or every 12 weeks (total of 2 doses) for participants in Cohort 5. However, dose levels and frequency of administration may be revised based on the safety data from previous cohorts.
The total study duration for each participant will be up to 44 weeks:
• Screening period of up to 6 weeks
• Treatment period of 12 weeks
• Follow-up period of 26 weeks
Approximately 19 sites globally (US, UK, Europe) are planned with sites chosen based on the higher prevalence of SCA3 in these specific regions.REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
22/EE/0059
Date of REC Opinion
13 Apr 2022
REC opinion
Further Information Favourable Opinion