Status:
TERMINATED
Tamoxifen Therapy for Myotubular Myopathy
Lead Sponsor:
James Dowling
Collaborating Sponsors:
Canadian Institutes of Health Research (CIHR)
Cures Within Reach
Conditions:
X Linked Myotubular Myopathy
Eligibility:
MALE
6+ years
Phase:
PHASE1
PHASE2
Brief Summary
This is a phase 1 / 2, randomized, double-blinded, single cross-over study, with a washout period between treatment regimens, to test the efficacy and safety of tamoxifen therapy to improve motor and ...
Detailed Description
Pre-clinical studies in Mtm1 knockout mice (a model of XLMTM) demonstrated prolonged survival, increased motor function (including muscle strength), and improved muscle histopathology with tamoxifen t...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA
- Male
- Patients ages 6 months and older may participate.
- XLMTM resulting from a confirmed mutation in the Myotubularin 1 (MTM1) gene
- 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.
- Willing and able to comply with all protocol requirements and procedures.
- EXCLUSION CRITERIA
- Other disease which may significantly interfere with the assessment of myotubular myopathy (MTM) and is clearly not related to the disease, at the discretion of the qualified investigator.
- Has undergone surgery or hospitalization \< 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.
- Has a history of thromboembolic events
- Currently enrolled in a treatment study for XLMTM or receiving treatment with an experimental therapy other than pyridostigmine.
- Treatment with pyridostigmine for \< 6 weeks duration (must be greater than 6 weeks to be included in TAM4MTM).
- 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, aminoglutethimide, medroxyprogesterone, amiodarone, haloperidol, indinavir, ritonavir, quinidine, rifampicin, or any selective serotonin reuptake inhibitor (SSRI).
- Subject has a contraindication to tamoxifen or its ingredients
Exclusion
Key Trial Info
Start Date :
December 18 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 9 2024
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT04915846
Start Date
December 18 2020
End Date
May 9 2024
Last Update
September 19 2024
Active Locations (4)
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1
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
2
National Institutes of Health
Rockville, Maryland, United States, 20892
3
Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8
4
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3JH