A Study of the Natural History of Patients With LGMD2E, LGMD2D, and LGMD2C, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
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ClinicalTrials.gov Identifier: NCT04475926 |
Recruitment Status :
Recruiting
First Posted : July 17, 2020
Last Update Posted : November 17, 2020
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Condition or disease |
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Limb-girdle Muscular Dystrophy |
Study Type : | Observational |
Estimated Enrollment : | 160 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | A Multi-Center, Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C (LGMD2C/R5) |
Estimated Study Start Date : | November 2020 |
Estimated Primary Completion Date : | April 2023 |
Estimated Study Completion Date : | December 2023 |

Group/Cohort |
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LGMD2E Cohort
Patients with LGMD2E will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to <8 years age range, 8 to <14 years age range, and ≥14 years age range through the course of the study.
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LGMD2D Cohort
Patients with LGMD2D will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to <8 years age range, 8 to <14 years age range, and ≥14 years age range through the course of the study.
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LGMD2C Cohort
Patients with LGMD2C will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to <8 years age range, 8 to <14 years age range, and ≥14 years age range through the course of the study.
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- Time to Rise from the Floor [ Time Frame: Baseline upto Week 144 ]
- Time to Ascend 4 Steps [ Time Frame: Baseline upto Week 144 ]
- Time of 100-Meter Walk/Run (100MWR) [ Time Frame: Baseline upto Week 144 ]
- North Star Assessment for Dysferlinopathy (NSAD) Total Score [ Time Frame: Baseline upto Week 144 ]
- Dimension of the Performance of the Upper Limb (PUL) [ Time Frame: Baseline upto Week 144 ]
- Pulmonary Function Test: Forced Vital Capacity (FVC) [ Time Frame: Baseline upto Week 144 ]

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Ages Eligible for Study: | 4 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- Male or female patient ≥ 4 years of age with confirmed genetic diagnosis of LGMD2E, LGMD2D, or LGMD2C.
Exclusion Criteria:
- Demonstrates cognitive delay or impairment that could confound motor development, in the opinion of the Investigator.
- Has a medical condition, in the opinion of the Investigator, that might compromise patients ability to comply with study requirements.
- Is participating in other interventional study(ies) at the time of enrollment in this study.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04475926
Contact: Medical Information | +1-800-690-2003 | clinicaltrials@sarepta.com |
United States, Arkansas | |
Arkansas Children's | Recruiting |
Little Rock, Arkansas, United States, 72202 | |
Contact: Annette Guy guyea@archildrens.org | |
Principal Investigator: Aravindhan Veerapandiyan | |
United States, Virginia | |
Children's Hospital of the King's Daughters | Recruiting |
Norfolk, Virginia, United States, 23507 | |
Contact: Cara Headrick 757-688-6417 cara.headrick@chkd.org | |
Principal Investigator: Crystal Proud, MD |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04475926 |
Other Study ID Numbers: |
SRP-LGMD-501-NHS |
First Posted: | July 17, 2020 Key Record Dates |
Last Update Posted: | November 17, 2020 |
Last Verified: | November 2020 |
LGMD-2E North Star Assessment for Dysferlinopathy (NSAD) Performance of Upper Limb (PUL) Pulmonary function tests (PFTs) Ambulatory Non-Ambulatory Limb-girdle LGMD |
sarcoglycanopathy β -sarcoglycan Muscular Dystrophy α - sarcoglycan γ - sarcoglycan LGMD-2D LGMD-2C |
Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Sarcoglycanopathies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases |
Nervous System Diseases Genetic Diseases, Inborn Respiration Disorders Respiratory Tract Diseases Cardiomyopathies Heart Diseases Cardiovascular Diseases |