Clinical Trial Readiness for the Dystroglycanopathies

Description

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.

Conditions

Muscular Dystrophy

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Study Overview

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Study Details

Study overview

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.

Clinical Trial Readiness for the Dystroglycanopathies

Clinical Trial Readiness for the Dystroglycanopathies

Condition
Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Iowa City

University of Iowa, 200 Hawkins Drive, Iowa City, Iowa, United States, 52242

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * There are no exclusion criteria.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Katherine Mathews,

Katherine Mathews, M.D., PRINCIPAL_INVESTIGATOR, University of Iowa

Kevin Campbell, Ph.D., STUDY_DIRECTOR, Co-Investigator

Steven A. Moore, M.D. Ph.D., STUDY_DIRECTOR, Co-Investigator

Study Record Dates

2026-07