December 2024 - TAM4MTM XLMTM community webinar
On 3rd December 2024, the Myotubular Trust, a UK-based patient organization, hosted a community webinar to share updates on the discontinuation of the TAM4MTM clinical trial. Dr James Dowling, SickKids Hosptial Toronto, is the lead Principal Investigator and Sponsor of the study outlined the objectives and reasons why the study was terminated.
Study purpose: The TAM4MTM study explored re-purposing tamoxifen as a new treatment for XLMTM. Pre-clinical laboratory experiments suggested tamoxifen could improve the symptoms of mice with XLMTM.
Outcomes from the trial: Two participants experienced significant liver issues: one had hepatobiliary disorder and worsening liver function, while the other developed acute cholestasis. Both recovered after stopping tamoxifen. The trial was terminated due to these safety concerns, and patients/families/clinicians are strongly advised against “off-label” use of tamoxifen for XLMTM.
Future plans: A full report of the clinical outcome data is anticipated in 2025. Further work to understand the natural history of liver disease in XLMTM is ongoing, including why some patients get liver disease, how it develops, and how it can be treated.