NCT04915846: TAM4MTM

Tamoxifen Therapy for Myotubular Myopathy

Terminated Phase 1, PHASE2 Last updated 19 September 2024

What this trial tests

Phase 1, PHASE2 trial testing ApoTamox 10mg in X Linked Myotubular Myopathy in 6 participants. Terminated before completion.

Timeline

18 December 2020

Primary endpoint
9 May 2024

9 May 2024

Quick facts

Lead sponsor	James Dowling
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	quadruple
Primary purpose	treatment
Enrollment	6
Start date	18 December 2020
Primary completion	9 May 2024
Estimated completion	9 May 2024
Sites	4 locations across United Kingdom, Canada, United States

Drugs / interventions tested

• ApoTamox 10mg — full drug profile →
• Placebo

Conditions studied

• X Linked Myotubular Myopathy — all drugs for X Linked Myotubular Myopathy →

Sponsor

James Dowling — full company profile →

Who can join

6 Months and older, male only, with X Linked Myotubular Myopathy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Common Pathogenic Mechanisms in Centronuclear and Myotubular Myopathies and Latest Treatment Advances.
   Gómez-Oca R, Cowling BS, Laporte J. · · 2021 · cited 45× · PMID 34768808 · DOI 10.3390/ijms222111377
2. X-linked myotubular myopathy is associated with epigenetic alterations and is ameliorated by HDAC inhibition.
   Volpatti JR, Ghahramani-Seno MM, Mansat M, Sabha N, et al · · 2022 · cited 19× · PMID 35844027 · DOI 10.1007/s00401-022-02468-7
3. Molecular mechanisms and therapeutic strategies for neuromuscular diseases.
   Zambon AA, Falzone YM, Bolino A, Previtali SC. · · 2024 · cited 7× · PMID 38678519 · DOI 10.1007/s00018-024-05229-9
4. Natural history study and statistical modeling of disease progression in a preclinical model of myotubular myopathy.
   Buono S, Monseur A, Menuet A, Robé A, et al · · 2022 · cited 5× · PMID 35642830 · DOI 10.1242/dmm.049284
5. microRNA-133a as an indicator of disease progression and treatment response in X-linked myotubular myopathy.
   Maani N, Gustafson D, MacDonald P, Pannia E, et al · · 2025 · cited 1× · PMID 40212776 · DOI 10.1016/j.omtn.2025.102507