NCT03692312已完成2 期
A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 56
- 试验地点
- 14
- 主要终点
- Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
研究概览
简要总结
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
详细描述
This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.
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
- 6 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female children and adolescents aged ≥6 years and ≤16 years
- •Diagnosis of Congenital DM1 (also known as Steinert's disease)
- •Diagnosis must be genetically confirmed
- •One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:
- •Hypotonia
- •Generalized weakness
- •Respiratory insufficiency
- •Feeding difficulties
- •Clubfoot or another musculoskeletal deformity
- •Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
- •Written, voluntary informed consent must be obtained before any study related procedures are conducted.
- •Where a parent or LAR provides consent, there must also be assent from the subject
- •Subject's caregiver must be willing and able to support participation for duration of study
- •Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
排除标准
- •Not able to walk; (full time wheel chair use)
- •Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- •New or change in medications/therapies within 4 weeks prior to Screening
- •Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
- •Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
- •Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months
- •Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
- •Hypersensitivity to tideglusib and its excipients including allergy to strawberry
研究组 & 干预措施
Placebo
Placebo Comparator
Matching placebo, orally, once daily
干预措施: Placebo (Drug)
Tideglusib
Experimental
Weight adjusted tideglusib, orally, once daily
干预措施: Tideglusib (Drug)
Tideglusib
Experimental
Weight adjusted tideglusib, orally, once daily
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
时间窗: Baseline and week 20
The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1. The severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.
次要结局
- 10-meter Walk-run Test(20 weeks)
- Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores(Baseline and week 20)
- Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score(Baseline and week 20)
- Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)(Baseline and week 20)
- Clinical Global Impression - Severity Scale (CGI-S)(Baseline and week 20)
- Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study.(Between Screening to End of Study, up to 28 weeks)
- Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study.(Between Screening to End of Study, up to 28 weeks)
- CDM1-RS Independent Central Rater Score (CDM1-RS)(Baseline to week 20)
- CGI-I Independent Central Rater Score (CGI-I)(Baseline and week 20)
- Independent Rater Clinical Global Impression - Severity Scale (CGI-S)(Baseline and week 20)
研究者
研究点 (14)
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