A Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary efficacy of DYN101 in subjects 2 to 17 years of age with centronuclear myopathy caused by mutations in MTM1 or DNM2. - Research Using an Investigational Treatment for CNM (DyNaMic)

Dynacure0 个研究点目标入组 9 人开始时间: 2021年5月27日最近更新: 2021年10月26日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方
入组人数: 9

研究概览入排标准研究者记录与标识符相似试验

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

性别: All

入选标准

•1.Subject must be male or female aged =2 to <18years on the date the main ICF is signed.
•2.Subject must have a clinically symptomatic CNM, with a documented MTM1orDNM2 mutation.
•3.Subjects must have impaired muscle function as evidenced by:
•-MFM20 score between 5% and 80% for subjects =2 and <6 years of age, or
•-MFM32 score between 5% and 80% for subjects =6 years of age.
•4.Subject should have sufficient skeletal muscle (vastus lateralis, gastrocnemius, or biceps brachii as last resort) to perform 2 open muscle biopsies during the trial, as determined by ultrasound imaging at screening.
•5.Subject must meet have platelet count >150,000/µLat screening.
•6.Parent(s) or legally-authorized representative must be able to provide written, signed and dated informed consent for their child to participate in the trial. Informed assent can be obtained from the child according to local regulations.
•7.Parent(s) or legally-authorized representative must be at or above the age of legal consent in the jurisdiction of the country in which the trial is taking place.
•8.Subject, parent(s), and/or legally-authorized representative must have an understanding, ability, and willingness to fully comply with visit frequency, trial procedures, videorecording of assessments where applicable, and restrictions, including contraceptive requirements.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 9
•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. Subject has evidence of clinically significant liver disease with:
•-alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyltransferase, or bilirubin values > 2x upper limit of normal (ULN); and/or
•-clinically significant abnormal liver ultrasound results;and/or-clinically significant abnormal hepatic elastography results.
•2.Subject has evidence of clinically significant renal disease with:
•-Cystatin C =ULN and urinary protein to creatinine ratio =150 mg/g or 15mg/mmol; and-Total daily protein >50 mg/24 hours.
•3. Presence of significant comorbidities or conditions other than CNM or clinically significant findings during screening of medical history, physical examination, clinical laboratory evaluation, vital signs, or ECG recording for which, in the opinion of the investigator and/or the medical monitor, participation would not be in the best interest of the subject (e.g.compromise the safety or well-being) or that could prevent, limit, or confound the protocol-specified assessments (e.g.taking a muscle biopsy).
•4. Currently enrolled in any interventional trial or scheduled to participate in such a trial whilst participating in the current trial.
•5. Has previously received gene therapy for CNM.
•6.Subject has severe muscle contractures that would preclude the ability to show improvement in the MFM32 assessment, in the opinion of the investigator.
•7. Subject has severe airway malacia which could impact the capacity to wean off ventilatory support.
•8. Subject requires oxygen supplementation.
•9. For female subjects of childbearing potential: pregnant, breastfeeding, or planning to become pregnant during the trial.
•10. Current or relevant history of physical or psychiatric illness, and/or any medical disorder that may require treatment or make the subject unlikely to fully complete the trial, or any condition that presents undue risk from the IMP or procedures.
•11. Intake of any disallowed therapies by the subject, within 12 weeks before the planned first IMP administration.
•12. Known or suspected intolerance or hypersensitivity to IMP ingredients or closely related compounds.
•13. Parent(s) or legally-authorized representative are legally incapacitated or have limited legal capacity, or have lack of mental capacity to fully understand the protocol requirements and ensure completion of all required trial procedures.

研究者

发起方

Dynacure

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