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

Summary

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 \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS)

  collaborator NIH

• Ann & Robert H Lurie Children's Hospital of Chicago

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  collaborator OTHER

• University of Iowa

  collaborator OTHER

• Washington University School of Medicine

  collaborator OTHER

• Stanford University

  collaborator OTHER

• University of Utah

  collaborator OTHER

• University of Rochester

  collaborator OTHER

• University of Pittsburgh

  collaborator OTHER

• University of Texas, Southwestern Medical Center at Dallas

  collaborator OTHER

• University of Minnesota

  collaborator OTHER

• University of California, Los Angeles

  collaborator OTHER

• Boston Children's Hospital

  collaborator OTHER

• Nationwide Children's Hospital

  lead OTHER

Principal Investigators

• Anne M Connolly, MD · Nationwide Childrens Hospital

Eligibility

Max Age

5 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-05-12

Primary Completion

2028-09-30

Completion

2028-09-30

Countries

• United States

Study Locations