Spinocerebellar ataxia 3 Nervous System Diseases
Conditions
Interventions
Randomisation process:
Interactive Response Technology (IRT)
Study arms:
Experimental: BIIB132: Dose 1
Participants will receive BIIB1
Sponsors
Biogen Idec Research Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 2. Number of participants with serious adverse events (SAEs) assessed using National Cancer Institute CTCAE, Version 5 from Screening to Day 267 | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Area under the concentration-time curve (AUC) of BIIB132 measured using serum samples at pre-dose and multiple timepoints post-dose on Day 1 up to Day 85 2. Area under the concentration versus time curve, from time of dosing (time = 0) to infinity (AUCinf) of BIIB132 measured using serum samples at pre-dose and multiple timepoints post-dose on Day 1 up to Day 85 3. Area under the concentration versus time curve, from time of dosing (time = 0) to time of the last measurable effect (AUClast) of BIIB132 measured using serum samples at pre-dose and multiple timepoints post-dose on Day 1 up to Day 85 4. Maximum observed concentration (Cmax) of BIIB132 measured using serum samples at pre-dose and multiple timepoints post-dose on Day 1 up to Day 85 5. Time to reach maximum observed concentration (Tmax) of BIIB132 measured using serum samples at pre-dose and multiple timepoints post-dose on Day 1 up to Day 85 6. Elimination half-life (t½) of BIIB132 measured using serum samples at pre-dose and multiple timepoints post-dose on Day 1 up to Day 85 | — |
Countries
England, France, Germany, Israel, Netherlands, Portugal, United Kingdom
Outcome results
None listed