A pharmacokinetic and safety study of BIIB132 in adults with spinocerebellar ataxia 3
- Conditions
- Spinocerebellar ataxia 3Nervous System Diseases
- Registration Number
- ISRCTN94357046
- Lead Sponsor
- Biogen Idec Research Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing
- Sex
- All
- Target Recruitment
- 48
1. Diagnosis of SCA3 with CAG repeats =60 in ATXN3 gene
2. Symptomatic ataxia with a screening Scale for Assessment and Rating of Ataxia (SARA) score 3 to 15 (still ambulatory) and a minimum SARA gait subscore of 1
3. Able to ambulate 8 m independently without any assistive device
4. Treatment naïve or on a stable dose of symptomatic therapy for a minimum of 4 weeks prior to screening
1. Unstable psychiatric illness or untreated major depression within 90 days before screening
2. History or screening magnetic resonance imaging (MRI) results show evidence of structural abnormalities that couldcontribute to the participant’s clinical state other than findings typical of SCA3 or any finding that might pose a risk to the participant
3. MRI brain findings of prior cerebellar stroke or clinical stroke within 12 months before screening
4. History of brain surgery regardless of purpose
5. Any contraindications to undergoing brain MRI
6. History of, or ongoing, malignant disease, (with the exception of basal cell carcinomas and squamous cell carcinomas that have been completely excised and considered cured at least 12 months prior to screening). Participants with cancers in remission for longer than 5 years may be included
7. History of epilepsy or the occurrence of seizures within 3 years prior to screening
8. Evidence of untreated/unstable thyroid disease
9. Poorly controlled diabetes mellitus
10. History of alcohol or substance abuse within the past year prior to screening
11. Use of off-label drugs for ataxia within 4 weeks prior to screening
12. Prior enrollment in any interventional clinical study in which an investigational treatment or approved therapy forinvestigational use is administered within 5 half-lives or 3 months, whichever is longer, prior to the screening visit
13. Any antiplatelet (except for aspirin up to 100 mg/day) or anticoagulant medication that cannot be safely interrupted for a lumbar puncture (LP) procedure
14. Any contraindications to LP procedures
15. Participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study
16. Prior enrollment in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 3 months prior to screening visit
Note: other protocol-defined inclusion/exclusion criteria may apply
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <br> 1. Number of participants with adverse events (AEs) assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE, Version 5) from Day 1 to Day 267<br> 2. Number of participants with serious adverse events (SAEs) assessed using National Cancer Institute CTCAE, Version 5 from Screening to Day 267<br>
- Secondary Outcome Measures
Name Time Method