A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3 (NCT05160558) | Clinical Trial Compass
TerminatedPhase 1
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
Stopped: Sponsor's Decision
United States8 participantsStarted 2022-02-02
Plain-language summary
The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3). The secondary objective of this study is to characterize the multiple-dose pharmacokinetics (PK) of BIIB132 administered via IT injection to participants with SCA3.
Who can participate
Age range18 Years – 70 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key Inclusion Criteria:
* Diagnosis of SCA3 with CAG repeats ≥60 in ATXN3 gene.
* 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.
* Able to ambulate 8 m independently without any assistive device.
* Treatment naïve or on a stable dose of symptomatic therapy for a minimum of 4 weeks prior to screening.
Key Exclusion Criteria:
* Unstable psychiatric illness or untreated major depression within 90 days before screening.
* History or screening magnetic resonance imaging (MRI) results show evidence of structural abnormalities that could contribute to the participant's clinical state other than findings typical of SCA3 or any finding that might pose a risk to the participant.
* MRI brain findings of prior cerebellar stroke or clinical stroke within 12 months before screening.
* History of brain surgery regardless of purpose.
* Any contraindications to undergoing brain MRI.
* 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.
* History of epilepsy or the occurrence of seizures within 3 years prior to screening.
* Evidence of untreated/unstable thyroid disease.
* Poorly controlled diabetes mellitus.
* History of alcohol or substance abus…
What they're measuring
1
Number of Participants with Adverse Events (AEs)
Timeframe: Day 1 to Day 267
2
Number of Participants with Serious Adverse Events (SAEs)