A Phase 1/2 randomized, placebo-controlled, double-blinded, single crossover study to determine the safety and efficacy of tamoxifen therapy for myotubular myopathy (XLMTM)

Hospital for Sick Children0 个研究点目标入组 16 人开始时间: 2021年4月21日最近更新: 2024年4月22日

适应症

试验速览

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•In order to be eligible to participate in this study, an individual must meet all of the following criteria:
•2. Patients aged 2 years and older may participate
•3. MTM resulting from a confirmed mutation in the MTM1 gene
•4. Patients over 18 years of age and parent(s)/legal guardian(s) of patients < 18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors, or at least 7 years of age when required by regulation
•5. Willing and able to comply with all protocol requirements and procedures

•An individual who meets any of the following criteria will be excluded from participation in this study:
•1. Other disease which may significantly interfere with the assessment of MTM and is clearly not related to the disease, at the discretion of the qualified investigator
•2. Has undergone surgery or hospitalisation < 3 months before starting TAM4MTM (at t = -3 months), or has surgery scheduled during the 18 months of participation in TAM4MTM, which will impede motor assessments in the opinion of the Investigator
•3. Has a history of thromboembolic events
•4. Has severe liver dysfunction
•5. Currently enrolled in a treatment study for XLMTM or receiving treatment with an experimental therapy other than pyridostigmine
•6. Treatment with pyridostigmine for < 6 weeks duration (must be greater than 6 weeks to be included in TAM4MTM)
•7. Use of concomitant medication known to inhibit CYP2D6 and/or CYP3A4, including clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, goldenseal and grapefruit, paroxetine, troleandomycin, rifampin, phenobarbital, aminoglutethimidine, medroxyprogesterone, amiodarone, haloperidol, indinavir, ritonavir, quinidine, rifampicin, or any selective serotonin reuptake inhibitor (SSRI)
•8. Subject has a contraindication to tamoxifen or its ingredients

发起方

Hospital for Sick Children