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
- Conditions
- Machado-Joseph diseaseSCA310029305
- Registration Number
- NL-OMON51564
- Lead Sponsor
- Biogen
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 6
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 abuse within the past year prior to screening.
- Use of off-label drugs for ataxia within 4 weeks prior to screening.
- Prior enrollment in any interventional clinical study in which an
investigational treatment or approved therapy for investigational use is
administered within 5 half-lives or 3 months, whichever is longer, prior to the
screening visit.
- Any antiplatelet [except for aspirin up to 100 milligrams per day (mg/day)]
or anticoagulant medication that cannot be safely interrupted for an lumbar
puncture (LP) procedure.
- Any contraindications to LP procedures.
- Participants who are pregnant or currently breastfeeding and those intending
to become pregnant during the study.
- 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 <p>Number of Participants with Adverse Events (AEs)<br /><br>Number of Participants with Serious Adverse Events (SAEs)</p><br>
- Secondary Outcome Measures
Name Time Method <p>Area Under the Concentration-Time Curve (AUC) of BIIB132<br /><br>Area Under the Concentration Versus Time Curve, from Time of Dosing (Time = 0)<br /><br>to Infinity (AUCinf) of BIIB132<br /><br>Area Under the Concentration Versus Time Curve, from Time of Dosing (Time = 0)<br /><br>to Time of the Last Measurable Effect (AUClast) of BIIB132<br /><br>Maximum Observed Concentration (Cmax) of BIIB132<br /><br>Time to Reach Maximum Observed Concentration (Tmax) of BIIB132<br /><br>Elimination Half-Life (t*) of BIIB132</p><br>