Clinical trials: Myotubular Myopathy
Central Core Disease, Multi-Minicore Disease
The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.
Core myopathies (CCD/MmD), nemaline myopathies (NEM) and centronuclear myopathies (CNM) are three types of rare congenital myopathies.
- Conditions
- Central Core Disease, Multi-Minicore Disease, Nemaline Myopathy, Centronuclear Myopathy
- Trial name
- READYCOM (NCT06157268)
- Testing
- Natural history and non therapeutical therapy
- Where
- 2 sites in Netherlands
- Volunteers
- 100 people
- Sponsor
- Radboud University Medical Center
X-linked Myotubular Myopathy
Myotubular Myopathy Event Study
X-Linked myotubular myopathy (XLMTM), a form of centronuclear myopathy (CNM) is the result of a mutation in the MTM1 (myotubularin) gene which leads to altered myotubularin. Myotubularin is essential for optimum muscle function.
- Trial name
- MTMES (NCT01840657)
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Cure CMD
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