NCT02858908已完成2 期
A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy
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
- 16
- 试验地点
- 1
- 主要终点
- Safety (Adverse Events)
研究概览
简要总结
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy. The pharmacokinetics of tideglusib and its primary metabolite will also be investigated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 12 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adolescents or adults with diagnosis of congenital or juvenile-onset type 1 myotonic dystrophy (DM-1)
- •Diagnosis must be genetically confirmed
- •Subjects must be male or female aged 12 years to 45 years
- •Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2)
- •Subjects must be ambulatory and able to complete the 10 metre walk/run test (splints allowed)
- •Subject's legally authorized representative (LAR) must provide written informed consent and there must be written consent or assent (as age applicable and developmentally appropriate) by the subject before any study-related procedures are conducted
排除标准
- •Non-ambulatory (full time) wheel chair user
- •Receiving stimulant medication
- •Receiving other medications/therapies not stable (changed) within 4 weeks prior to Run-in (V2)
- •Medical illness or other concern which would cause investigator to conclude subjects will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment.
- •Current enrolment in a clinical trial of an investigational drug or enrolment in a clinical trial of an investigational drug in the last 6 months
- •Women of child bearing potential who are pregnant, lactating or not willing to use a protocol defined acceptable contraception method if sexually active and not surgically sterile.
- •Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication and impact the interpretability of the study results
- •Current clinically significant (as determined by the investigator) cardiovascular, renal, hepatic, endocrine or respiratory disease
- •Clinically significant heart disease (in the opinion of the investigator) or second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia
- •A history of chronic liver disease with current out of range values for Alanine transaminase (ALT), clinically relevant hepatic steatosis or other clinical manifestations of ongoing liver disease
- •A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis)
- •A history of alcohol or substance use disorders
研究组 & 干预措施
Cohort 1 - Tideglusib
Experimental
1000 mg tideglusib, orally, once daily
干预措施: Tideglusib (Drug)
Cohort 2 - Tideglusib
Experimental
400 mg tideglusib, orally, once daily
干预措施: Tideglusib (Drug)
结局指标
主要结局
Safety (Adverse Events)
时间窗: 12 weeks
Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.
次要结局
- Plasma Concentration of Tideglusib(12 weeks)
- Blood Pharmacokinetics of Tideglusib(12 weeks)
- Area Under the Plasma Concentration vs. Time Curve of Tideglusib(12 weeks)
- 10 Metre Walk/Run Test(12 weeks)
- Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time(12 weeks)
- Respiratory Forced Vital Capacity (FVC)(12 weeks)
- Dual-energy X-ray Absorptiometry (DXA)(12 weeks)
- Clinical Global Impressions- Improvement (CGI-I)(12 weeks)
- Actigraphy (3-minute Bouts of Activity)(12 weeks)
- Actigraphy (>10-minute Bouts of Activity)(12 weeks)
- Actigraphy (Steps)(12 weeks)
- Nine Hole Peg Test (NHPT)(12 weeks)
- Top 3 Concerns Visual Analogue Scale (VAS) Score(12 weeks)
- Ohio State University (OSU) Autism Rating Scale (OARS)(12 weeks)
- Ohio State University (OSU) Autism Clinical Global Impression (CGI)(12 weeks)
- Clinical Global Impressions- Severity (CGI-S)(12 weeks)
- Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy(12 weeks)
- Peabody Picture Vocabulary Test (PPVT)(12 weeks)
- Biomarker - Lymphocyte GSK3β Levels and Activity(12 weeks)
研究者
研究点 (1)
Loading locations...
相似试验
尚未招募
2 期
Tislelizumab Monotherapy or Combined With Lenvatinib as Neoadjuvant Therapy for Resectable Hepatocellular Carcinoma.Resectable Hepatocellular CarcinomaNCT05807776Tianjin Medical University Cancer Institute and Hospital50
Unknown
2 期
A Study on Toripalimab Plus Nab-Paclitaxel With or Without Cisplatin as First-line Treatment of Urothelial CarcinomaUrothelial CarcinomaNCT04211012Jun Guo30
已完成
不适用
A Study of Tislelizumab (BGB-A317) in Combination With Chemotherapy as First Line Treatment in Participants With Advanced Esophageal Squamous Cell CarcinomaNCT03783442BeiGene649
Unknown
2 期
Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy of Nimacimab in Patients With Diabetic GastroparesisDiabetic GastroparesisNCT03900325Bird Rock Bio, Inc.6
Unknown
2 期
A Study of the C3 Inhibitor AMY-101 in Patients With ARDS Due to COVID-19 (SAVE)Acute Respiratory Distress Syndrome Due to SARS-CoV-2 Infection (Severe COVID19)NCT04395456Amyndas Pharmaceuticals S.A.144