TAM4MTM- Safety and Efficacy of Tamoxifen Therapy for XLMTM
Research type
Research Study
Full title
A Phase 1/2 Randomised, Placebo-Controlled, Double-Blinded, Single Crossover Study to Determine the Safety and Efficacy of Tamoxifen Therapy for Myotubular Myopathy (XLMTM)
IRAS ID
286790
Contact name
Giovanni Baranello
Contact email
Sponsor organisation
The Hospital for Sick Children
Eudract number
2020-004443-86
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
This is a Phase 1/2 randomised, double-blinded, single crossover 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. 16 participants across all participating centres will be enrolled. Eligible participants will consist of males of age ≥ 2 years with myotubular myopathy, resulting from a confirmed mutation in MTM1 gene. All participants will receive tamoxifen (Apo-Tamox) for approximately 6 months (6 months +/-1 week), and placebo for approximately 6 months (6 months +/- 1 week). Depending on randomisation, drug or placebo will be dispensed at the end of the t=0 study visit (Phase 1). At the end of Phase 1, participants will enter a ‘washout’ period, when they will cease treatment. After approximately 3 months of washout, participants will cross-over to the other treatment regimen and receive the other IMP for the final 6 months of their study participation (Phase 2).
REC name
London - Central Research Ethics Committee
REC reference
20/LO/1145
Date of REC Opinion
21 Dec 2020
REC opinion
Further Information Favourable Opinion