An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 76
- 试验地点
- 14
- 主要终点
- Safety (Adverse Events)
研究概览
简要总结
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
详细描述
This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
- •Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM
- •Diagnosis must be genetically confirmed
- •Subjects must be male or female aged ≥6 years to ≤45 years at Screening
- •Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
- •Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)
- •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
- •Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:
- •Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
- •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 (as required by local regulations)
- •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
- •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 Eligibility/Baseline Visit
- •Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
- •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 other than the AMO-02- MD-2-003 study
- •Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results
- •Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry
研究组 & 干预措施
Tideglusib
Weight adjusted or weight banded tideglusib, orally, once daily
干预措施: Tideglusib (Drug)
结局指标
主要结局
Safety (Adverse Events)
时间窗: 52 Weeks
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.
Safety (Adverse Events) - With Optional Expanded Access
时间窗: Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from End of Treatment to End of Optional Extended Access, and End of Optional Extended Access to the End of Follow-up period.
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
时间窗: 52 Weeks
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.
次要结局
- Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access(Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132)
- Clinical Global Impressions Improvement Scale (CGI-I)(54 Weeks)
- Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded Access(Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132)
- Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score(54 weeks)
- Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access(Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132)
- Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)(52 weeks)
- Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded Access(Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132)
- Clinical Global Impressions Severity Scale (CGI-S)(54 weeks)
- Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded Access(Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132)
- Autism Behavior Inventory- Clinician (ABI-C)(52 Weeks)
- Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview(52 Weeks)
- 10-meter walk-run test(52 Weeks)
- Plasma Troponin T levels(52 Weeks)
- Plasma Troponin T levels - With Optional Expanded Access(Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132)
- Blood Leukocytes CTG Repeats(Baseline and Week 52)
- Blood Leukocytes CTG Repeats - With Optional Expanded Access(Week 52 or later)