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
Public ClinicalTrials.gov record NCT04475926. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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)
Study identification
- NCT ID
- NCT04475926
- Recruitment status
- Active, not recruiting
- Study type
- Observational
- Phase
- Not listed
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Enrollment
- 205 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- 4 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 21, 2021
- Primary completion
- Sep 4, 2030
- Completion
- Sep 4, 2030
- Last update posted
- Nov 5, 2025
2021 – 2030
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| Arkansas Children's | Little Rock | Arkansas | 72202 | — |
| University of California San Diego | La Jolla | California | 92037 | — |
| University of California, Davis Health Dept of PM&R | Sacramento | California | 95817 | — |
| Anne & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Oregon Health and Science University | Portland | Oregon | 97068 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75201 | — |
| University of Utah Hospital | Salt Lake City | Utah | 84112 | — |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 15 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04475926, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 5, 2025 · Synced May 18, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04475926 live on ClinicalTrials.gov.