Efficacy of Deutetrabenazine in Huntington's Disease Patients with Dysphagia: a Randomised, Placebo-controlled Pilot Study
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 36
- 试验地点
- 1
- 主要终点
- Efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
研究概览
简要总结
To determine the efficacy of deutetrabenazine to control symptoms of dysphagia associated with HD.
详细描述
The primary endpoint is the change in swallow function/dysphagia from baseline to maintenance therapy.
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
- Triple (Participant, 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
- •Confirmed diagnosis of HD
- •Symptoms of dysphagia
- •Must be able to swallow tablets
排除标准
- •Other confounding diseases that affect swallowing
- •Depression
- •Hepatic impairment
- •Renal impairment
研究组 & 干预措施
Deutetrabenazine
The participants randomized to this group will receive oral deutetrabenazine for 8 weeks. Week 1: 6 mg once daily; Week 2: 6 mg twice daily (BID); Week 3: 9 mg BID; Week 4: 12 BID; Week 5: 15 mg BID; Week 6: 18 mg BID; Week 7: 21 mg BID; Week 8: 24 mg BID.
干预措施: Deutetrabenazine Oral Tablet [Austedo] (Drug)
Placebo
The participants randomized to this group will receive oral placebo for 8 weeks. Week 1: 6 mg once daily; Week 2: 6 mg twice daily (BID); Week 3: 9 mg BID; Week 4: 12 BID; Week 5: 15 mg BID; Week 6: 18 mg BID; Week 7: 21 mg BID; Week 8: 24 mg BID.
干预措施: Placebo oral tablet (Drug)
结局指标
主要结局
Efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
时间窗: 18 months
The change in swallow function/dysphagia from baseline to maintenance therapy using the clinical swallow assessment Huntington's Disease Dysphagia Scale (HDDS). The Huntington's Disease Dysphagia Scale is an 11-item scale used to monitor swallowing difficulties in persons in early to advanced disease stages. The Huntington's Disease Dysphagia Scale includes questions related to the preparatory oral, oral, pharyngeal, and esophageal phases of swallow. The response options are the following: Question 1-4 and 6-11: 1 No, almost never; 2 Yes, seldom; 3 Yes, sometimes; 4 Yes, frequently; 5 Yes, almost always. Question 5: 1 Yes, almost always; 2 Yes, frequently; 3 Yes, sometimes; 4 Yes, seldom; 5 No, almost never. Higher scores mean a worse outcome.
Instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
时间窗: 18 months
Assessment of pharyngeal and esophageal dysphagia using the Videofluoroscopic Swallowing Study, also known as the Modified Barium Swallow Study. The instrumental assessment provides a description of any anatomical differences and physiological problems that may be associated with the patients' symptoms in all phases of the swallow. Results from the videoflourographic study are used to rate the severity of dysphagia according to the Dysphagia Outcome and Severity Scale. The Dysphagia Outcome and Severity Scale scores range from 1 to 7, with 7 representing normal swallowing and 1 representing severe dysphagia.
Non-instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
时间窗: 18 months
Non-instrumental assessment and symptoms of dysphagia using the Bedside Swallowing Assessment Scale. The Bedside Swallowing Assessment Scale interpretation: Level 1: irrelevant alterations, scores ≤ 19; level 2: average alterations, scores 20-23; level 3: severe alterations, scores ≥ 24. Higher scores mean a worse outcome.
次要结局
- Effect of deutetrabenazine on body weight in HD patients.(18 months)
- Effect of deutetrabenazine on body mass index in HD patients.(18 months)
- Effect of deutetrabenazine on fat distribution in HD patients.(18 months)
研究者
Sylvette Ayala-Pena
Scientific Official/Principal Investigator
Fundacion Huntington Puerto Rico