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Filters: Condition: Limb-Girdle Muscular Dystrophy, Type 2E Clear all

Conditions: Limb-girdle Muscular Dystrophy
Interventions: SRP-9003, Glucocorticoid; Biological · Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 4
States / cities: La Jolla, California • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more

Conditions: Limb-Girdle Muscular Dystrophy, Type 2E
Interventions: SRP-9003; Genetic
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years to 15 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Limb-Girdle Muscular Dystrophy, Type 2E
Interventions: Not listed
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 3 Years to 15 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease
Interventions: Not listed
Lead sponsor: Hereditary Neuropathy Foundation; Government
Eligibility: Not listed
Enrollment: 10,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2029
U.S. locations: 1
States / cities: New York, New York

Conditions: Limb-Girdle Muscular Dystrophy Type 2A, Limb-Girdle Muscular Dystrophy, Type 2E
Interventions: Not listed
Lead sponsor: Lindsay Alfano; Other
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2025
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Limb-girdle Muscular Dystrophy
Interventions: Not listed
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years and older
Enrollment: 205 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2030
U.S. locations: 11
States / cities: Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 8 more

Conditions: LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
Interventions: Not listed
Lead sponsor: Virginia Commonwealth University; Other
Eligibility: 6 Years to 50 Years
Enrollment: 1,000 participants
Timeline: 2023 – 2029
U.S. locations: 1
States / cities: Richmond, Virginia

Conditions: Limb Girdle Muscular Dystrophy
Interventions: SRP-9003; Genetic
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years to 50 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 2
States / cities: Columbus, Ohio • Memphis, Tennessee

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A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Limb Girdle Muscular Dystrophy Type 2E Recruitment Study

Natural History Study for Charcot Marie Tooth Disease

Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

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

Long-Term Development of Muscular Dystrophy Outcome Assessments

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)