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A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
Sponsor: Sarepta Therapeutics, Inc.
Summary
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy type 2C (LGMD2C/R5), or Limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 5 years after enrollment for participants with LGMD2C/R5, LGMD2D/R3, and LGMD2E/R4 with a North Star Assessment for Dysferlinopathy (NSAD) ≥ 25 at Baseline, up to 3 years for participants with LGMD2C/R5, LGMD2D/R3, and LGMD2E/R4 with a NSAD \< 25 at Baseline, and up to 3 years for participants with LGMD2A/R1. Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present.
Official title: Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), Type 2C (LGMD2C/R5), and Type 2A (LGMD2A/R1)
Key Details
Gender
All
Age Range
4 Years - Any
Study Type
OBSERVATIONAL
Enrollment
205
Start Date
2021-04-22
Completion Date
2030-09-05
Last Updated
2025-11-06
Healthy Volunteers
No
Conditions
Locations (26)
Barrow Neurological Institute
Phoenix, Arizona, United States
Arkansas Children's
Little Rock, Arkansas, United States
University of California San Diego
La Jolla, California, United States
University of California, Davis Health Dept of PM&R
Sacramento, California, United States
Anne & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Utah Hospital
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
University Hospital Gent
Ghent, Oost-Vlaanderen, Belgium
UZ Leuven
Leuven, Vlaams Brabant, Belgium
Hôpital Universitaire des Enfants Reine Fabiola
Brussels, Belgium
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
Children's Hospital - London Health Science Centre
London, Ontario, Canada
Zentrum für Kinderheilkunde und Jugendmedizin Uniklinikum Giessen Marburg (UKGM), Standort Giessen
Giessen, Hesse, Germany
Universitätsklinikum Essen
Essen, North Rhine-Westphalia, Germany
Istituto Giannina Gaslini
Genoa, Liguria, Italy
Azienda Ospedale Università Padova
Padova, Veneto, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Hospital Sant Joan de Déu Universidad de Barcelona
Barcelona, Spain
Karolinska Universitetssjukhuset Solna
Stockholm, Stockholm County, Sweden
Lokman Hekim Etlik Hastanesi
Ankara, Turkey (Türkiye)
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, United Kingdom
Institute of Genetic Medicine, International Centre for Life
Newcastle upon Tyne, United Kingdom