Phase IIa, Double-blind, Randomized, Placebo-controlled Study of the Efficacy and Safety of SOM3355 in Huntington's Disease (HD) Patients With Chorea Movements.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 4
- 主要终点
- UHDRS (Unified Huntington's Disease Rating Scale) Total Maximal Chorea (TMC) score
研究概览
简要总结
Phase IIa study to evaluate the efficacy and safety of SOM3355 in chorea movements associated with Huntington's disease
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject is at least 18 years of age at time of consent.
- •Diagnosis of HD definite by a movement disorders expert and confirmed by a number of HTT gene CAG repeats equal or greater than
- •Female of child bearing potential (FCBP) and non-vasectomized male agree to practice appropriate methods of birth control.
- •Ability to walk independently or with minimal assistance.
- •UHDRS TMC score equal or greater than
- •UHDRS TFC equal or greater than
- •Subject has provided written informed consent or through his/her legally authorized representative.
排除标准
- •Onset of HD symptoms prior to age 18 (Juvenile forms of HD).
- •Non-ambulatory patients.
- •A past medical history of clinically significant ECG abnormalities or a family history (grandparents, parents and siblings) of a prolonged QT-interval syndrome.
- •Pregnant or breastfeeding female patients, including those planning to conceive during the period of the trial.
- •Patients with psychiatric symptoms, or other impairments, that would interfere with their full compliance with the Investigator instructions and testing, unless there is an identified caregiver to support the patient.
- •Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the subject in case of participation in the study. The Investigator should make this determination in consideration of the subject's medical history and/or clinical laboratory test results at screening and baseline.
- •Known allergy/sensitivity/intolerance to the study drugs or their excipients.
- •Any significant laboratory results which, in the Investigator's opinion, would not be compatible with study participation or represent a risk for the subject while in the study.
- •Prescribed anti-hypertensive medication, tetrabenazine, deutetrabenazine or valbenazine within 15 days prior starting the investigational treatment.
- •Excluded concomitant medications: any anti-hypertensive medication, tetrabenazine, deutetrabenazine or valbenazine, all typical neuroleptics and all MAO inhibitors
- •Subject has a history of alcohol or substance abuse in the previous 12 months.
- •Patients with diabetic ketoacidosis or metabolic acidosis.
- •Patients with cardiogenic shock, congestive heart failure, pulmonary hypertension due to right-sided heart failure, severe sinus bradycardia, atrioventricular block (grades II and III) or sinoatrial block.
- •Subject has participated in an investigational drug or device trial within 30 days prior starting the investigational treatment.
研究组 & 干预措施
Group A
Administration of SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks, SOM3355 100mg BID for 6 weeks and placebo BID for 6 weeks.
干预措施: SOM3355 100mg BID (Drug)
Group A
Administration of SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks, SOM3355 100mg BID for 6 weeks and placebo BID for 6 weeks.
干预措施: SOM3355 200mg BID (Drug)
Group A
Administration of SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks, SOM3355 100mg BID for 6 weeks and placebo BID for 6 weeks.
干预措施: Placebo BID (Drug)
Group B
Administration of placebo BID for 6 weeks, SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks and SOM3355 100mg BID for 6 weeks.
干预措施: SOM3355 100mg BID (Drug)
Group B
Administration of placebo BID for 6 weeks, SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks and SOM3355 100mg BID for 6 weeks.
干预措施: SOM3355 200mg BID (Drug)
Group B
Administration of placebo BID for 6 weeks, SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks and SOM3355 100mg BID for 6 weeks.
干预措施: Placebo BID (Drug)
结局指标
主要结局
UHDRS (Unified Huntington's Disease Rating Scale) Total Maximal Chorea (TMC) score
时间窗: 6 months
UHDRS subscore used to measure the effectiveness of SOM3355 on HD chorea.
次要结局
- Columbia Suicide Severity Rating Scale (C-SSRS)(6 months)
- UHDRS Total Functional Capacity (TFC)(6 months)
- Clinical Global Impression of Change (CGIC) scale(6 months)
- Patient Global Impression of Change (PGIC) scale(6 months)
- UHDRS Functional Assessment(6 months)
- UHDRS Total Motor Score (TMS)(6 months)
- UHDRS Gait score(6 months)
- Safety (number of participants with adverse events)(6 months)