RECRUITING

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

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

Conditions

LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
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Related Conditions:

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

Study Overview

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Study Details

Study overview

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

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

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

Condition
LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Intervention / Treatment

-

Contacts and Locations

Los Angeles

University of California, Los Angeles, Los Angeles, California, United States, 90095

Stanford

Stanford University, Stanford, California, United States, 94305

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Iowa City

University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States, 52242

Bethesda

National Institute of Neurological Disorders and Stroke, NIH, Bethesda, Maryland, United States, 20892

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Saint Paul

University of Minnesota, Saint Paul, Minnesota, United States, 55108

St. Louis

Washington University in St. Louis, St. Louis, Missouri, United States, 63130

Rochester

University of Rochester, Rochester, New York, United States, 14627

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

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

  • * Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable).
  • * Subject must be aged birth to less than 5.0 years of age at time of consent.
  • * A confirmed diagnosis of LAMA2-RD confirmed via:
  • * Absence of another confirmed genetic disease.
  • * Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study.
  • * Willingness to comply with the study protocol, including but not limited to, all study procedures and visits.
  • * Acute medical illness or hospitalization within 30 days prior to informed consent.
  • * 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.
  • * Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD.

Ages Eligible for Study

to 5 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Nationwide Children's Hospital,

Anne M Connolly, MD, PRINCIPAL_INVESTIGATOR, Nationwide Childrens Hospital

Study Record Dates

2028-09