A Randomized, Open-label, Multicenter, Phase 2 Clinical Trial to Explore the Safety and Efficacy of Sepranolone in Pediatric and Adult Patients With Tourette Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 26
- 试验地点
- 2
- 主要终点
- To investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the The Yale Global Tic Severity Scale (YGTSS) Total Score.
研究概览
简要总结
The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone as a treatment for Tourette syndrome, by reducing the severity and frequency of tics. The total study duration from the Screening Visit to the final follow-up visit was approximately 26 weeks and included the following periods:
- A baseline period of 4 weeks between the screening visit (Visit 1) and randomization (4 weeks of baseline period were not needed in well-known adult subjects with stable Tourette syndrome history over the past at least 4 weeks). A school holiday/annual leave period of 2-6 weeks where no study-related activities were done.
- A randomized treatment period of 12 weeks
- A safety follow-up period of 4 weeks
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
- None
入排标准
- 年龄范围
- 12 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Tic severity score ≥ 20 Yale Global Tic Severity Scale (YGTSS) Total Score at baseline
- •The patient may have Obsessive-Compulsive Disease (OCD) as a comorbidity
排除标准
- •Patient has participated in a clinical study over the past 30 days
- •Evidence or history of neurological disease that may interfere with the study
- •Malignant disease
- •Unstable or clinical significant medical condition that could pose a risk
- •HIV/ongoing hepatitis
- •Clinical significant findings in vital signs
- •History of anaphylactic reactions
研究组 & 干预措施
Sepranolone
Sepranolone 10 mg sc twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment.
干预措施: Sepranolone (Drug)
结局指标
主要结局
To investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the The Yale Global Tic Severity Scale (YGTSS) Total Score.
时间窗: Change from baseline at week 4, 8 and 12.
The Yale Global Tic Severity Scale (YGTSS) is a validated scale for assessing the severity of motor and vocal tics in both children and adults with Tourette syndrome. Tics are scored based on a semi-structured interview, including scoring of the number, frequency, intensity, complexity, and interference of tics. The primary endpoint is the total tic score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome.
次要结局
- Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome(From randomization (day 1) until the end of study visit (week 16).)
- To evaluate the efficacy of Sepranolone to reduce the urge to tic in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Premonitory Urge for Tics Scale (PUTS) scale.(Change from baseline at week 4, 8 and 12.)
- To evaluate the effect on Global impression by Sepranolone in patients with Tourette syndrome at 12 weeks, as measured by the Tourette Syndrome-Clinical Global Impression (TS-CGI).(TS-CGI score at week 4, 8 and 12.)
- To evaluate the efficacy of Sepranolone to reduce tic related impairment in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Yale Global Tic Severity Scale (YGTSS) Impairment Score.(Change from baseline at week 4, 8 and 12.)
- To evaluate the effect of Sepranolone on activities of daily living in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the GTS-QoL ADL subscale.(Change from baseline at week 4, 8 and 12.)