NL-OMON51564撤回不适用
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 - 260SA101 (Mera)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- Biogen
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •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.
排除标准
- •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
- •- 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.
研究者
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