Clinical trialNCT06503367RecruitingObservational

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

SponsorNationwide Children's HospitalEnrollment44LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Anne M Connolly, MD
Nationwide Childrens Hospital
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

14 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

University of California, Los Angeles
Los Angeles, California, United States
Perry Shieh, MD PhD
Recruiting
Stanford University
Stanford, California, United States
Carolina Tesi-Rocha, MD
Recruiting
Ann & Robert H. Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
Nancy Kuntz, MD
Recruiting
University of Iowa Stead Family Children'S Hospital
Iowa City, Iowa, United States
Katherine Mathews, MD
Recruiting
National Institute of Neurological Disorders and Stroke, Nih
Bethesda, Maryland, United States
Reghan Foley, MD
Recruiting
Boston Children'S Hospital
Boston, Massachusetts, United States
Partha Ghosh, MD
Recruiting
University of Minnesota
Saint Paul, Minnesota, United States
Peter Kang, MD
Recruiting
Washington University in St. Louis
St Louis, Missouri, United States
Craig Zaidman, MD
Recruiting
University of Rochester
Rochester, New York, United States
Bo Hoon Lee, MD
Recruiting
Cincinnati Children'S Hospital Medical Center
Cincinnati, Ohio, United States
Cuixia Tian, MD
Recruiting
Nationwide Children'S Hospital
Columbus, Ohio, United States
Anne M Connolly, MDLindsay Alfano, DPTMegan Waldrop, MD
Recruiting
Children'S Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Hoda Abdel-Hamid, MD
Recruiting
University of Texas Southwestern
Dallas, Texas, United States
Kaitlin Batley, MD
Recruiting
University of Utah
Salt Lake City, Utah, United States
Russell Butterfield, MD PhD
Not, yet, recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 12 May 2025
Primary completion: 30 Sept 2028
Completion: 30 Sept 2028
Last update posted: 19 Aug 2026
Registered enrollment: 44
Registry id: NCT06503367

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.