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

Part of paid clinical trials in Los Angeles, California.

Sponsor: Nationwide Children's Hospital
Study ID: NCT06503367
Status: Recruiting

Conditions

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

Eligibility Criteria

Sex: ALL
Age: N/A - 5 Years
Healthy Volunteers: Not accepted

Study Details

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

Key Dates

Start date: May 12, 2025
Status verified: Apr 2026
Primary completion: Sep 30, 2028
Completion: Sep 30, 2028

Study Design

Enrollment: 44 participants (estimated)

Primary Outcome Measure

Validate the change over 24 months using the Neuromuscular Gross Motor Outcome [ Time Frame: 24 months ]

Central Contacts

Kevin Warf
(614) 355-2765
[email protected]

Locations (14)

Facility	City	State	ZIP	Site coordinators
University of California, Los Angeles	Los Angeles	California	90095	Indumathi Mohanchandra [email protected] (310) 825-3264 Perry Shieh, MD PhD (PRINCIPAL_INVESTIGATOR)
Stanford University	Stanford	California	94305	Lin Karman [email protected] (650) 384-9417 Carolina Tesi-Rocha, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	60611	Raven Hill [email protected] (312) 227-2937 Nancy Kuntz, MD (PRINCIPAL_INVESTIGATOR)
University of Iowa Stead Family Children's Hospital	Iowa City	Iowa	52242	Laura Knosp [email protected] (319) 384-6277 Katherine Mathews, MD (PRINCIPAL_INVESTIGATOR)
National Institute of Neurological Disorders and Stroke, NIH	Bethesda	Maryland	20892	Dana MacDonald [email protected] (301) 402-2273 Reghan Foley, MD (PRINCIPAL_INVESTIGATOR)
Boston Children's Hospital	Boston	Massachusetts	02115	Elicia Estrella [email protected] (617) 919-7384 Partha Ghosh, MD (PRINCIPAL_INVESTIGATOR)
University of Minnesota	Saint Paul	Minnesota	55108	Ellen Poppy [email protected] (612) 625-2850 Peter Kang, MD (PRINCIPAL_INVESTIGATOR)
Washington University in St. Louis	St Louis	Missouri	63130	Julie Miner [email protected] (314) 362-1566 Craig Zaidman, MD (PRINCIPAL_INVESTIGATOR)
University of Rochester	Rochester	New York	14627	Kim Hart [email protected] (585) 275-3767 Bo Hoon Lee, MD (PRINCIPAL_INVESTIGATOR)
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	45229	Jessica Getz [email protected] (513) 803-9036 Cuixia Tian, MD (PRINCIPAL_INVESTIGATOR)
Nationwide Children's Hospital	Columbus	Ohio	43205	Kevin Warf [email protected] (614) 355-2765 Anne M Connolly, MD (PRINCIPAL_INVESTIGATOR) Megan Waldrop, MD (SUB_INVESTIGATOR) Lindsay Alfano, DPT (SUB_INVESTIGATOR)
Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania	15224	Heather Dicostanzo [email protected] (412) 692-7660 Hoda Abdel-Hamid, MD (PRINCIPAL_INVESTIGATOR)
University of Texas Southwestern	Dallas	Texas	75235	Shahera Ranjha [email protected] (214) 456-5959 Kaitlin Batley, MD (PRINCIPAL_INVESTIGATOR)
University of Utah	Salt Lake City	Utah	84112	Jessi Beyer [email protected] (801) 587-4865 Russell Butterfield, MD PhD (PRINCIPAL_INVESTIGATOR)

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By research site

University of California, Los Angeles· Los Angeles, CA Stanford University· Stanford, CA Ann & Robert H. Lurie Children's Hospital of Chicago· Chicago, IL University of Iowa Stead Family Children's Hospital· Iowa City, IA National Institute of Neurological Disorders and Stroke, NIH· Bethesda, MD Boston Children's Hospital· Boston, MA