Nemaline Myopathy Clinical Research Network (NM-CTRN)
ConditionNemaline Myopathy
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-18
SponsorStanford University
About this trial
The goal of this study is to establish a research network to help define the natural disease history and clinical outcome measures for Nemaline Myopathy (NM).
Eligibility criteria
Qualifiers
0-18 years of age at recruitment
Confirmation of Nemaline Myopathy (pathogenic or likely pathogenic mutations in ACTA1 (AD) or NEB (AR)
Patient and/or parent or legal guardian must be willing and able to provide informed consent
Disqualifiers
Clinically significant medical finding on the physical examination, other than NM, that the Investigator deems unsuitable for participation in and/or completion of the study procedures
Any confirmed chronic or acute condition or disease affecting any system(s), which could interfere with the results of the study and/or the compliance with the study procedures. This will be subject to the clinical judgement of the Principal Investigator (PI)
Participants of ongoing (interventional) clinical trials that assess the efficacy of potential treatments will be excluded
Safety concerns
Trial design
Treatments tested in this trial
- Not listed
Trial groups
50 Participants
are grouped into 1 trial groupSponsors and collaborators
Stanford University
Lead sponsor
A Foundation Building Strength
Collaborator
Source ClinicalTrials.gov