Trials for patients with XLMTM
Many clinical trials and studies are specific to patients with a certain genetic mutation or other eligibility criteria. You will need a genetic confirmation to participate in clinical trials. Below is a list of studies for patients with X-linked myotubular myopathy:
Upcoming studies:
Title | A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition (EXCEL) More information can be found using the following link, on clinicaltrials.gov |
Status | Recruiting |
Sponsor | Astellas Gene Therapies |
Study Type | Observational Study |
Duration | 2025/01/31 – 2026/08/31 |
Location(s) | North America and United Kingdom |
Active/ongoing studies:
Title | Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study More information can be found using the following link, on clinicaltrials.gov |
Status | Recruiting |
Sponsor | Laurent Servais |
Study Type | Observational Study |
Duration | 2020/07/10 – 2026/03 |
Location(s) | Belgium |
Title | Molecular and Genetic Studies of Congenital Myopathies More information can be found using the following link, on clinicaltrials.gov |
Status | Recruiting |
Sponsor | Boston Children’s Hospital |
Study Type | Observational Study |
Duration | 2003/08 - 2050/01 |
Location(s) | United States |
Title | The Natural History and Muscle Fatigability of Patients With Congenital Myopathies. (READYCOM) More information can be found using the following link, on clinicaltrials.gov |
Status | Recruiting |
Sponsor | Radboud University Medical Center |
Study Type | Observational Study |
Duration | 2024/03/28 – 2026/11 |
Location(s) | Netherlands |
Title | Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment (COMPIS) More information can be found using the following link, on clinicaltrials.gov |
Status | Enrolling by invitation |
Sponsor | Vastra Gotaland Region |
Study Type | Interventional Study |
Duration | 2021/10/25 – 2025/12/31 |
Location(s) | Sweden |
Previous studies:
Title | Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies (NatHis-CNM) More information can be found using the following link, on clinicaltrials.gov |
Status | Completed |
Sponsor | Institut de Myologie, France |
Study Type | Natural History Study |
Duration | 2017/05/28 – 2021/09/30 |
Location(s) | Belgium, France, Germany, Italy, Spain |
Title | Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) (MTM) More information can be found using the following link, on clinicaltrials.gov |
Status | Completed |
Sponsor | Valerion Therapeutics, LLC |
Study Type | Natural History Study |
Duration | 2014/02 – 2017/06/26 |
Location(s) | United States, Canada, Belgium, France, Germany, Italy, Spain |
Title | Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM) More information can be found using the following link, on clinicaltrials.gov |
Status | Terminated |
Sponsor | James Dowling |
Study Type | Interventional Study |
Duration | 2020/12/18 – 2024/05/09 |
Location(s) | North America and United Kingdom |
Title | A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM) (ASPIRO) More information can be found using the following link, on clinicaltrials.gov |
Status | On hold indefinitely |
Sponsor | Astellas Gene Therapies |
Study Type | Interventional Study |
Duration | 2017/08/02 – 2021/09/09 |
Location(s) | United States, Canada, France, Germany |
Title | Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (Unite-CNM) More information can be found using the following link, on clinicaltrials.gov |
Status | Terminated |
Sponsor | Dynacure |
Study type | Interventional Study |
Duration | 2020/01/09 - 2022/06/22 |
Location(s) | Belgium, Denmark, France, Germany, Netherlands, United Kingdom |