Overview

Tamoxifen Therapy for Myotubular Myopathy

Status:
Recruiting
Trial end date:
2023-12-31
Target enrollment:
Participant gender:
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 respiratory function in males with XLMTM.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
James Dowling
Collaborators:
Canadian Institutes of Health Research (CIHR)
Cures Within Reach
Great Ormond Street Hospital Charity
Mogford Campbell Family Chair Fund
Myotubular Trust
Sparks
The Joshua Frase Foundation USA
Will Cure USA
Treatments:
Tamoxifen