RECRUITING

Clinical Trial Readiness for the Dystroglycanopathies

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Conditions

Muscular DystrophyMDfukutin-related protein genelimb girdleFKRP genecongenital muscular dystrophychildhood onset LGMDadult onset LGMDPOMT1POMT2POMGnT1LARGEalpha dystroglycandystroglycanopathyISPD/CRPPADPM 1, 2 or 3GMPPBB3GNT1/B4GAT1B3GALNT2GTDC2/POMGnT2TMEM5/RXYLT1FukutinDAG1POMK/SGK196DOLKGO5R2TRAPPC11

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

Clinical Trial Readiness for the Dystroglycanopathies

Quick Facts

Study Start:2006-04
Study Completion:2026-07
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT00313677

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Elevated CK (creatine kinase)
  2. * 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
  3. * Dystroglycanopathies are predicted to affect all racial and ethnic backgrounds, and all patients with dystroglycanopathies will be eligible for participation.
  4. * Participants may be of any age, including children, and males and females will be recruited equally.
  5. * Patients will have varying degrees of muscular weakness, but otherwise should be in relatively good health.
  1. * There are no exclusion criteria.

Contacts and Locations

Study Contact

Carrie Stephan, R.N. M.A.
CONTACT
(319) 356-2673

Principal Investigator

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 Locations (Sites)

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

Collaborators and Investigators

Sponsor: 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

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2006-04
Study Completion Date2026-07

Study Record Updates

Study Start Date2006-04
Study Completion Date2026-07

Terms related to this study

Keywords Provided by Researchers

  • muscular dystrophy
  • MD
  • fukutin-related protein gene
  • limb girdle
  • FKRP gene
  • congenital muscular dystrophy
  • childhood onset LGMD
  • adult onset LGMD
  • POMT1
  • POMT2
  • POMGnT1
  • LARGE
  • alpha dystroglycan
  • dystroglycanopathy
  • ISPD/CRPPA
  • DPM 1, 2 or 3
  • GMPPB
  • B3GNT1/B4GAT1
  • B3GALNT2
  • GTDC2/POMGnT2
  • TMEM5/RXYLT1
  • Fukutin
  • DAG1
  • POMK/SGK196
  • DOLK
  • GO5R2
  • TRAPPC11

Additional Relevant MeSH Terms

  • Muscular Dystrophy