The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2+
SponsorRadboud University Medical Center
About this trial
Core myopathies (CCD/MmD), nemaline myopathies (NEM) and centronuclear myopathies (CNM) are three types of rare congenital myopathies. Not much is known about the natural history and no curative treatment is available for these groups. Also patients report fatigability as one of their symptoms. The goal of this observational study is to study the natural history during 24 months to achieve trial readiness and to study the muscle fatigability in CCD/MmD, NEM and CNM.
Eligibility criteria
Qualifiers
2 years or older
Willing and able to complete the measurement protocol
Willing and able to travel to Nijmegen and Utrecht
Dutch-speaking
Disqualifiers
None
Trial design
Treatments tested in this trial
- Natural history and non therapeutical therapy
Treatment groups
100 Participants
are divided into 3 treatment groupsSponsors and collaborators
Radboud University Medical Center
Lead sponsor
UMC Utrecht
Collaborator
Source ClinicalTrials.gov