Clinical Trial Readiness for the Dystroglycanopathies
ConditionMuscular Dystrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorKatherine Mathews
About this trial
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Elevated CK (creatine kinase)
Evidence of a dystroglycanopathy as determined by review of muscle pathology OR documented mutation in one of the known genes OR abnormal alpha-dystroglycan glycosylation in cultured fibroblasts
Dystroglycanopathies are predicted to affect all racial and ethnic backgrounds, and all patients with dystroglycanopathies will be eligible for participation.
Participants may be of any age, including children, and males and females will be recruited equally.
Disqualifiers
There are no exclusion criteria.
Trial population
neuromuscular care clinic
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
10 meter walk
time to walk 10 meters without assistive device
Time frame
through study completion, an average of 1 yeaSecondary outcomes
1
4 stair climb
time to climb 4 stairs with or without use of rails
Time frame
through study completion, an average of 1 yeaSponsors and contacts
Click on the lead sponsor to view all of their trials.
Katherine Mathews
Lead sponsor
University of Iowa
Sponsor institution
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
Source ClinicalTrials.gov