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Clinical Trial Readiness for the Dystroglycanopathies

RecruitingObservational study

Who can join

All ages · All sexes

Full eligibility criteria
Inclusion Criteria:

* 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.
* Patients will have varying degrees of muscular weakness, but otherwise should be in relatively good health.

Exclusion Criteria:

* There are no exclusion criteria.

About the study

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.

Sponsor: Katherine Mathews · Participants: 190 · Started: 2006-04

Contact the study team

Official record on ClinicalTrials.gov — NCT00313677

Locations in the U.S.

IowaUniversity of Iowa, 200 Hawkins Drive, Iowa City

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.