Clinical trials: Myotubular Myopathy
X-Linked Myotubular Myopathy
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition.
- Trial name
- ASPIRO (NCT03199469)
- Testing
- Resamirigene bilparvovec
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 27 people
- Sponsor
- Astellas Gene Therapies
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety and tolerability as well as pharmacokinetics (PK), pharmacodynamics (PD) and…
- Trial name
- DyNaMic (NCT04743557)
- Testing
- DYN101
- Where
- 2 sites in France, Germany
- Sponsor
- Dynacure
Males With X-linked Myotubular Myopathy (XLMTM)
A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)
This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical manifestations and recorded…
- Trial name
- NCT02231697
- Testing
- Non-interventional, retrospective medical chart review
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 161 people
- Sponsor
- Astellas Gene Therapies
X-linked Myotubular Myopathy
A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger.
- Trial name
- INCEPTUS (NCT02704273)
- Where
- 8 sites in United States, Canada and 3 more
- Volunteers
- 34 people
- Sponsor
- Astellas Gene Therapies
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)/preliminary efficacy of…
- Trial name
- Unite-CNM (NCT04033159)
- Testing
- DYN101
- Where
- 8 sites in Belgium, Denmark and 4 more
- Volunteers
- 14 people
- Sponsor
- Dynacure
X-Linked Myotubular Myopathy
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07052929
- Testing
- ASP2957, Methylprednisolone, Prednisolone
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
X-linked Myotubular Myopathy
A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07876804
- Testing
- ASP2957
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Myotubular Myopathy
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe.
- Trial name
- MTM (NCT02057705)
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 48 people
- Sponsor
- Valerion Therapeutics, LLC
X-Linked Myotubular Myopathy
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness.
- Trial name
- EXCEL (NCT06581146)
- Testing
- No Intervention
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Astellas Gene Therapies
Centronuclear Myopathy
Natural History Study of Patients With Centronuclear Myopathies
This is a prospective, longitudinal study of the natural disease course intended to recruit approximately 60 patients with centronuclear myopathies (CNM) in Europe and the United States.
- Trial name
- NatHis-CNM (NCT04977648)
- Sponsor
- Dynacure