drugset / Trial / NCT04915846
Tamoxifen Therapy for Myotubular Myopathy
Phase 1/2
Terminated
6 enrolled
James Dowling
Canadian Institutes of Health Research (CIHR) · collabCures Within Reach · collabGreat Ormond Street Hospital Charity · collabMogford Campbell Family Chair Fund · collabMyotubular Trust · collabSparks · collabThe Joshua Frase Foundation USA · collabWill Cure USA · collab
RandomizedCrossoverQuadruple-blindTreatment
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.
Timeline
- Start
- 2020-12-18
- Primary completion
- 2024-05-09
- Completion
- 2024-05-09
Publications
- Background Amburgey K, Tsuchiya E, de Chastonay S, Glueck M, Alverez R, Nguyen CT, Rutkowski A, Hornyak J, Beggs AH, Dowling JJ. A natural history study of X-linked myotubular myopathy. Neurology. 2017 Sep 26;89(13):1355-1364. doi: 10.1212/WNL.0000000000004415. Epub 2017 Aug 25.
- Background Maani N, Sabha N, Rezai K, Ramani A, Groom L, Eltayeb N, Mavandadnejad F, Pang A, Russo G, Brudno M, Haucke V, Dirksen RT, Dowling JJ. Tamoxifen therapy in a murine model of myotubular myopathy. Nat Commun. 2018 Nov 19;9(1):4849. doi: 10.1038/s41467-018-07057-5.
- Background Gayi E, Neff LA, Massana Munoz X, Ismail HM, Sierra M, Mercier T, Decosterd LA, Laporte J, Cowling BS, Dorchies OM, Scapozza L. Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy. Nat Commun. 2018 Nov 19;9(1):4848. doi: 10.1038/s41467-018-07058-4.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Tamoxifen | Small molecule | 10 mg | Oral |