EUCTR2016-004623-23-GB进行中（未招募）1 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 - Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 56
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •The subject will not be considered eligible for the study without meeting all of the criteria below:
- •1. Subjects under study must be children or adolescents with a diagnosis of DM1. For the purposes of this study, the following definitions apply.
- •In addition to the genetic confirmation of DM1, one or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first week after birth:
- •Hypotonia
- •Generalized weakness
- •Respiratory insufficiency
- •Feeding difficulties
- •Clubfoot or another musculoskeletal deformity
- •2. Diagnosis must be genetically confirmed
- •3. Subjects must be male or female children and adolescents aged =6 years and =16 years at Screening
- •4. Subjects must have a Clinical Global Impression – Severity (CGI-S) score of 4 or greater at Screening and start of Run-in (V2)
- •5. Subjects must be ambulatory and able to complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
- •6. 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)
- •7. Subject’s caregiver must be willing and able to support participation for duration of study
- •8. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 56
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range 0
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1. Non-ambulatory (full time) wheel chair use
- •2. BMI less than 13.5 kg/m² or greater than 40 kg/m²
- •3. Receiving other medications/therapies not stable (i.e. changed) within 4 weeks prior to Screening. For each enrollee, every effort should be made to maintain stable regimens (i.e. dose, as applicable, and frequency) of allowed concomitant medications (e.g. concomitant mexiletine or stimulants) and allowed non-medicine based therapies (e.g. occupational or physiotherapy) throughout the course of the study, from the time of commencement of Screening until the last study assessment
- •4. Use within 4 weeks prior to Baseline (V3) of strong CYP3A4 inhibitors. Examples include clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir
- •5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window e.g. warfarin and digitoxin
- •6. Medical illness or other concern 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
- •7. 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
- •8. Gastrointestinal disease which, in the opinion of the investigator, may interfere with the absorption, distribution, metabolism or excretion of the study medication and impact the interpretability of the study results
- •9. Current clinically significant (as determined by the investigator) neurological, cardiovascular, renal, hepatic, endocrine or respiratory disease that may impact the interpretability of the study results
- •10. 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
- •11. Implantation of a cardiac pacemaker within the 12 months preceding Screening
- •12. Average QTcF value of >450 msec at Screening or at Run-In (V2) (may repeat to confirm)
- •13. Clinically significant abnormalities in safety laboratory tests, vital signs or ECG, as determined by the Investigator at Screening or Run-In (V2) as applicable (may repeat to confirm)
- •14. Females 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
- •15. Males, engaged in sexual relations with a female of child-bearing potential, not using an acceptable contraceptive method if not surgically sterile
- •16. Kidney disease requiring ongoing treatment
- •17. A history of chronic liver disease with current out of range values for ALT, clinically relevant hepatic steatosis or other clinical manifestations of liver disease
- •18. ALT value > 2X ULN reference range at Screening (may repeat to confirm)
- •19. Total bilirubin value greater than the upper limit of the normal reference range at Screening (unless due to Gilbert’s syndrome) (may repeat to confirm). For subjects with a well known/well documented diagnosis of Gilbert's syndrome a total bilirubin
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