Phase IIb, Randomized, Double-blind, Placebo-controlled Study in Parallel Groups Assessing the Efficacy and Safety of Two Doses of SOM3355 in Patients Suffering From Huntington's Disease With Choreic Movements
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 139
- 试验地点
- 23
- 主要终点
- Change in Total Maximal Chorea (TMC) Score of the UHDRS® for Subjects Not Taking Neuroleptics During the Trial (mITT - N=122)
研究概览
简要总结
Phase IIb, randomized, double-blind, placebo-controlled study in parallel groups assessing the efficacy and safety of two doses of SOM3355 in patients suffering from Huntington's Disease with choreic movements.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 21 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females ≥21 years old, a diagnosis of Huntington's Disease determined by a movement disorders expert and confirmed by a number of HTT gene CAG repeats ≥36, a UHDRS® Total maximal chorea (TMC) score ≥10, and a UHDRS® Total Functional Capacity (TFC) ≥7.
排除标准
- •Onset of HD symptoms prior to age of 21 years (juvenile forms of HD), HD patients presenting rigid akinesia, and use of other VMAT2 inhibitors such as tetrabenazine, deutetrabenazine, or valbenazine, or other antichoreic treatment such as any neuroleptic, or amantadine, memantine, riluzole.
研究组 & 干预措施
Placebo
Placebo capsules were administered twice daily (BID) for at least 9 weeks at maintenance dose.
干预措施: Placebo capsules (Drug)
SOM3355 400 mg/day
SOM3355 200 mg capsules were administered twice daily (BID) for at least 9 weeks at maintenance dose.
干预措施: SOM3355 200 mg capsules (Drug)
SOM3355 600 mg/day
SOM3355 300 mg capsules were administered twice daily (BID) for at least 8 weeks at maintenance dose.
干预措施: SOM3355 300 mg capsules (Drug)
结局指标
主要结局
Change in Total Maximal Chorea (TMC) Score of the UHDRS® for Subjects Not Taking Neuroleptics During the Trial (mITT - N=122)
时间窗: From baseline to end of maintenance dose (10 weeks of treatment).
Pre-defined analysis of the primary efficacy endpoint (change in TMC score from baseline to the end of maintenance dose) performed with the 122 subjects of the mITT not taking neuroleptics during the trial. The TMC is part of the motor assessment of the Unified Huntington's Disease Rating Scale (UHDRS) and measures chorea in 7 different body parts, including the face, oral-buccal-lingual region, trunk, and each limb independently. The TMC score is the sum of the individual scores, ranging from 0 to 28. A decrease in TMC scores indicates improvement in chorea symptoms.
次要结局
- Change in the Clinical Global Impression (CGI) (mITT - N=139)(From baseline to end of maintenance dose (10 weeks of treatment).)
- Change in the Patient Global Impression (PGI) (mITT - N=139)(From baseline to end of maintenance dose (10 weeks of treatment).)