Actively Recruiting
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
Led by Nationwide Children's Hospital · Updated on 2026-04-08
44
Participants Needed
14
Research Sites
172 weeks
Total Duration
On this page
Sponsors
N
Nationwide Children's Hospital
Lead Sponsor
N
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent by the subject, parent(s), or legally authorized representative and/or assent by the subject when applicable
- Age from birth to less than 5 years at time of consent
- Confirmed diagnosis of LAMA2-RD by either two pathogenic variants in the LAMA2 gene or muscle biopsy showing absence of merosin with at least one pathogenic LAMA2 gene variant
- No other confirmed genetic diseases
- Willingness to maintain current exercise and/or physical therapy during the study
- Willingness to follow all study procedures and visit schedules
You will not qualify if you...
- Acute medical illness or hospitalization within 30 days before consent
- Participation in any investigational therapy trial for LAMA2-RD within 30 days before consent
- Use of any investigational therapy including off-label Losartan within 30 days before consent
- Participation in other clinical studies within 30 days or 3 half-lives prior to consent that may affect study results
- Other significant medical conditions that may interfere with study interpretation as judged by the Principal Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
4
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
Actively Recruiting
5
National Institute of Neurological Disorders and Stroke, NIH
Bethesda, Maryland, United States, 20892
Actively Recruiting
6
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
7
University of Minnesota
Saint Paul, Minnesota, United States, 55108
Actively Recruiting
8
Washington University in St. Louis
St Louis, Missouri, United States, 63130
Actively Recruiting
9
University of Rochester
Rochester, New York, United States, 14627
Actively Recruiting
10
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
11
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
12
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
13
University of Texas Southwestern
Dallas, Texas, United States, 75235
Actively Recruiting
14
University of Utah
Salt Lake City, Utah, United States, 84112
Not Yet Recruiting
Research Team
K
Kevin Warf
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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