RECRUITING

Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy

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Conditions

LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)Early Phase 1ObservationalNatural HistoryNeuromuscularFunctional Assessments

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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 goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires

Official Title

Establishing Clinical Trial Readiness for Children 0-5 Years With Congenital Muscular Dystrophy Secondary to LAMA2 Mutations (READY CMD LAMA2)

Quick Facts

Study Start:2025-05-12
Study Completion:2028-09
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06503367

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 to 5 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable).
  2. * Subject must be aged birth to less than 5.0 years of age at time of consent.
  3. * A confirmed diagnosis of LAMA2-RD confirmed via:
  4. * Absence of another confirmed genetic disease.
  5. * Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study.
  6. * Willingness to comply with the study protocol, including but not limited to, all study procedures and visits.
  1. * Acute medical illness or hospitalization within 30 days prior to informed consent.
  2. * Participation in a previous trial of any investigational agent for LAMA2-RD within 1 month prior to informed consent, or use of any other investigational therapy (including off-label use of Losartan) within 30 days prior to informed consent, or participation in other clinical studies, within 30 days (or 3 half-lives, whichever is longer) prior to informed consent, which in the opinion of the PI, may potentially confound results from this study.
  3. * Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD.

Contacts and Locations

Study Contact

Kevin Warf
CONTACT
(614) 355-2765
ReadyCMDLAMA2@nationwidechildrens.org

Principal Investigator

Anne M Connolly, MD
PRINCIPAL_INVESTIGATOR
Nationwide Childrens Hospital

Study Locations (Sites)

University of California, Los Angeles
Los Angeles, California, 90095
United States
Stanford University
Stanford, California, 94305
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
United States
National Institute of Neurological Disorders and Stroke, NIH
Bethesda, Maryland, 20892
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
University of Minnesota
Saint Paul, Minnesota, 55108
United States
Washington University in St. Louis
St. Louis, Missouri, 63130
United States
University of Rochester
Rochester, New York, 14627
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States
University of Texas Southwestern
Dallas, Texas, 75235
United States
University of Utah
Salt Lake City, Utah, 84112
United States

Collaborators and Investigators

Sponsor: Nationwide Children's Hospital

  • Anne M Connolly, MD, PRINCIPAL_INVESTIGATOR, Nationwide Childrens Hospital

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 Date2025-05-12
Study Completion Date2028-09

Study Record Updates

Study Start Date2025-05-12
Study Completion Date2028-09

Terms related to this study

Keywords Provided by Researchers

  • Early Phase 1
  • Observational
  • Natural History
  • Neuromuscular
  • Functional Assessments

Additional Relevant MeSH Terms

  • LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)