Myopathies, Structural, Congenital clinical trials
The registry lists 24 studies for Myopathies, Structural, Congenital across more than 10 countries. 10 are recruiting or about to start. 20 of them are mainly about Myopathies, Structural, Congenital (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.
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Who runs them
Astellas Gene Therapies (6) · Dynacure (3) · Cure CMD (2) · Centre Hospitalier Universitaire de Liege (1) · Boston Children's Hospital (1) · University of Missouri-Columbia (1) · University of Nevada, Las Vegas (1) · University of Erlangen-Nürnberg Medical School (1)
What is being tested here
Drugs under test in Myopathies, Structural, Congenital studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026The Natural History and Muscle Fatigability of Patients With Congenital Myopathies. · Radboud University Medical Center
- Oct 2027Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators · Astellas Gene Therapies
Who these studies take
Of the 7 Myopathies, Structural, Congenital studies recruiting now, 4 (57%) set no upper age limit and 2 (29%) stop at 75 or younger. 7 (100%) take children under 18. 0 (0%) are for women only and 2 (29%) for men only; 2 (29%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (71%) | 6 (86%) |
| 18 | 5 (71%) | 5 (71%) |
| 30 | 5 (71%) | 5 (71%) |
| 40 | 5 (71%) | 5 (71%) |
| 50 | 5 (71%) | 5 (71%) |
| 65 | 5 (71%) | 5 (71%) |
| 75 | 5 (71%) | 5 (71%) |
| 85 | 4 (57%) | 4 (57%) |
Across all 24 studies, recruiting or not: 38% stop at 75 or younger and 38% at 65; 92% take children; 0% are for women only and 38% for men only; 8% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 24 of 24. search them.
- Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need VentilatorsRecruitingNCT07052929Phase 1/22 countries
- A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957Not yet recruitingNCT07876804Phase 1/2
- A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle ConditionRecruitingNCT065811463 countries
- Molecular and Genetic Studies of Congenital MyopathiesRecruitingNCT002728831 country
- Smoking Cessation Intervention for Young Adults Using MTM: A Randomized Controlled TrialNot yet recruitingNCT07104006Not applicable1 country
- Multispectral Optoacoustic Tomography for Advanced Imaging of Centronuclear MyopathyRecruitingNCT070218201 country
- Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next StudyRecruitingNCT05982119Not applicable7 countries
- Myotubular and Centronuclear Myopathy Patient RegistryRecruitingNCT040643071 country
- The Prevalence of RYR1-related DiseaseNot yet recruitingNCT067913691 country
- The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.RecruitingNCT061572681 country
- Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseActive, not recruitingNCT07478172Not applicable1 country
- A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)Active, not recruitingNCT03199469Phase 2/34 countries
- Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no TreatmentCompletedNCT05099107Not applicable1 country
- Tamoxifen Therapy for Myotubular MyopathyTerminatedNCT04915846Phase 1/23 countries
- Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear MyopathiesTerminatedNCT04033159Phase 1/26 countries
- Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear MyopathiesCompletedNCT03351270Not applicable5 countries
- A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) SubjectsCompletedNCT027042735 countries
- A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)CompletedNCT022316972 countries
- Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear MyopathiesWithdrawnNCT04743557Phase 1/22 countries
- Natural History Study of Patients With Centronuclear MyopathiesWithdrawnNCT04977648
- Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)CompletedNCT024531521 country
- Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)CompletedNCT020577057 countries
- Myotubular Myopathy Event StudyCompletedNCT018406571 country
- Myotubular Myopathy Genetic Testing StudyCompletedNCT018179461 country