Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 5
SponsorNationwide Children's Hospital
About this trial
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
Eligibility criteria
Qualifiers
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.
Absence of another confirmed genetic disease.
Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Not listed
Trial groups
No trial groups listed
Sponsors and collaborators
Nationwide Children's Hospital
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago
Collaborator
Children's Hospital Medical Center, Cincinnati
Collaborator
University of Iowa
Collaborator
Washington University School of Medicine
Collaborator
Stanford University
Collaborator
University of Utah
Collaborator
University of Rochester
Collaborator
University of Pittsburgh
Collaborator
University of Texas, Southwestern Medical Center at Dallas
Collaborator
University of Minnesota
Collaborator
University of California, Los Angeles
Collaborator
Boston Children's Hospital
Collaborator
Source ClinicalTrials.gov