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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

StatusActive, Not Recruiting
PhaseNot specified
Started2021-04-22
View on ClinicalTrials.gov ↗

Amendment history

2025-11-04
Recruiting→Active, Not Recruiting Study Status, Study Description, Study Design, Contacts/Locations v19
Trial statusRecruiting→Active, Not Recruiting
Primary completion date2030-04-30→2030-09-05
Completion date2030-04-30→2030-09-05
Enrollment targetestimated 241→confirmed 205
Study description+152 characters
LGMD Type 2A, 2C, and 2D cohorts are now closed. Enrollment in the LGMD Type 2E cohort is closed and participants will be followed for up to five years.
Study sitesdetails revised at 26 of 26 sites
2024-10-16
Study Status, Study Description, Study Design, Outcome Measures, Eligibility, Contacts/Locations v18
Primary completion date2028-03-31→2030-04-30
Completion date2028-03-31→2030-04-30
Enrollment target225→241
Eligibility criteria+176 characters
Inclusion Criteria: - Male or female participant ≥ 4 years of age withwho confirmeddemonstrate symptoms of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1 in the opinion of the investigator (eg, muscle weakness, loss of function, delayed milestones). Confirmed clinical and genetic diagnosis of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LG [...]
Primary endpoints8 to 9 entries
NorthAnkle StarRange Assessmentof for DysferlinopathyMotion (ROM) NSAD) Total Score
Study sitesdetails revised at 19 of 26 sites
2024-03-26
Study Identification, Study Status, Study Description, Conditions, Study Design, Arms and Interventions, Eligibility, Contacts/Locations v17
Primary completion date2025-12-24→2028-03-31
Completion date2025-12-24→2028-03-31
Enrollment target90→225
Study arms3→4
Eligibility criteria+11 characters
[...] e with confirmed genetic diagnosis of LGMD2E/R4, LGMD2D/R3, or LGMD2C/R5, or LGMD2A/R1. Exclusion Criteria: Demonstrates cognitive delay or impair [...]
Study sitesdetails revised at 23 of 26 sites
Study title+10 characters
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
2023-04-06
Study Status, Contacts/Locations v16
Study sites25→26
2023-03-20
Study Status, Contacts/Locations v15
Study sites24→25
Show 14 earlier versions
2023-02-16
Study Status, Contacts/Locations v14
Study sites22→24
2023-01-09
Study Status, Contacts/Locations v13
Study sites15→22
2022-08-23
Study Status, Contacts/Locations v12
Study sites13→15
2022-07-14
Study Identification, Study Status, Study Description, Study Design, Arms and Interventions, Eligibility, Contacts/Locations v11
Enrollment target160→90
Study sites12→13
Eligibility criteria+10 characters
Inclusion Criteria: - Male or female patientparticipant ≥ 4 years of age with confirmed genetic diagnosis of LGMD2E [...] , in the opinion of the Investigator, that might compromise patientsparticipants ability to comply with study requirements. Is participating [...]
Study title+4 characters
A Study of the Natural History of PatientsParticipants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
2022-04-14
Study Status, Contacts/Locations v10
Study sites10→12
2022-03-25
Study Status, Conditions, Contacts/Locations v9
Study sites9→10
2022-02-18
Study Status, Contacts/Locations v8
Study sites8→9
2022-01-14
Study Status, Contacts/Locations v7
Study sites5→8
2021-06-18
Study Status, Contacts/Locations v6
Primary completion date2023-08-31→2025-12-24
Completion date2024-04-30→2025-12-24
Study sites4→5
2021-05-13
Study Status v5
Primary completion date2023-08→2023-08-31
Completion date2024-04→2024-04-30
Start dateestimated 2021-03→confirmed 2021-04-22
2021-03-02
Study Identification, Study Status, Study Description, Conditions, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v4
Primary completion date2023-04→2023-08
Completion date2023-12→2024-04
Start date2020-11→2021-03
Study sites2→4
Eligibility criteria+9 characters
Inclusion Criteria: Male or female patient ≥ 4 years of age with confirmed genetic diagnosis of LGMD2E/R4, LGMD2D/R3, or LGMD2C/R5. Exclusion Criteria: Demonstrates cognitive delay or impair [...]
Primary endpoints6 to 8 entries
Time of 10-Meter Walk/Run [10MWR] Timed Up and Go (TUG)
Study title+9 characters
A Study of the Natural History of Patients With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
2020-11-16
Study Status, Contacts/Locations v3
Start date2020-10→2020-11
Study sites1→2
2020-10-07
Study Status, Contacts/Locations v2
Start date2020-09→2020-10
Study sitesdetails revised at 1 of 1 site
2020-09-23
Not Yet Recruiting→Recruiting Study Status, Outcome Measures, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Start date2020-07→2020-09
Study sites0→1
Primary endpoints6 entries, revised
Time to Rise Fromfrom the Floor
2020-07-14
Original filing
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.
Trial Details
NCT Number NCT04475926
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Limb-girdle Muscular Dystrophy
Enrollment 205 participants
Start Date 2021-04-22
Primary Completion 2030-09-05 (estimated)
Study Completion 2030-09-05 (estimated)
Updated on ClinicalTrials.gov 2025-11-06