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Sarepta Therapeutics

Sarepta Therapeutics logo
🇺🇸United States
Ownership
Private, Public
Established
1980-01-01
Employees
1.3K
Market Cap
$13.4B
Website
http://www.sarepta.com
Introduction

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company, which is engaged in the discovery and development of therapeutics for the treatment of rare diseases. The company was founded on July 22, 1980, and is headquartered in Cambridge, MA.

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)

Phase 1
Active, not recruiting
Conditions
Limb Girdle Muscular Dystrophy
First Posted Date
2023-05-25
Last Posted Date
2024-12-24
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
6
Registration Number
NCT05876780
Locations
🇺🇸

Nationwide Children's Hospital, Columbus, Ohio, United States

🇺🇸

St. Jude Children's Research Hospital, Memphis, Tennessee, United States

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Phase 3
Completed
Conditions
Duchenne Muscular Dystrophy
First Posted Date
2021-10-27
Last Posted Date
2025-07-08
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
126
Registration Number
NCT05096221
Locations
🇺🇸

Arkansas Children's, Little Rock, Arkansas, United States

🇺🇸

UC San Diego Altman Clinical and Translational Research Institute, La Jolla, California, United States

🇺🇸

UCLA Medical Center, Los Angeles, California, United States

and more 39 locations

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Phase 1
Active, not recruiting
Conditions
Muscular Dystrophy, Duchenne
First Posted Date
2020-11-12
Last Posted Date
2024-08-26
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
55
Registration Number
NCT04626674
Locations
🇺🇸

Stanford University, Palo Alto, California, United States

🇺🇸

University of California, Davis, Sacramento, California, United States

🇺🇸

Washington University in St. Louis, Saint Louis, Missouri, United States

and more 2 locations

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

Recruiting
Conditions
Limb-girdle Muscular Dystrophy
First Posted Date
2020-07-17
Last Posted Date
2024-10-18
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
241
Registration Number
NCT04475926
Locations
🇺🇸

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

and more 23 locations

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Phase 2
Terminated
Conditions
Duchenne Muscular Dystrophy
Interventions
First Posted Date
2019-07-01
Last Posted Date
2025-03-10
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
62
Registration Number
NCT04004065
Locations
🇺🇸

University of California Davis Health, Sacramento, California, United States

🇺🇸

Connecticut Children's, Farmington, Connecticut, United States

🇺🇸

Northwest Florida Clinical Research Group, LLC, Gulf Breeze, Florida, United States

and more 22 locations

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Phase 3
Active, not recruiting
Conditions
Muscular Dystrophy, Duchenne
Interventions
First Posted Date
2019-06-20
Last Posted Date
2024-12-20
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
160
Registration Number
NCT03992430
Locations
🇺🇸

University of Alabama at Birmingham, Birmingham, Alabama, United States

🇺🇸

University of Florida, Gainesville, Florida, United States

🇺🇸

Rare Disease Research, LLC, Atlanta, Georgia, United States

and more 56 locations

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

Phase 2
Terminated
Conditions
Duchenne Muscular Dystrophy
Interventions
First Posted Date
2019-06-14
Last Posted Date
2023-08-18
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
15
Registration Number
NCT03985878
Locations
🇧🇪

UZ-Gent, Gent, Belgium

🇫🇷

Hopital Trousseau, Bâtiment lemariey, Paris, France

🇮🇹

Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile, Rome, Italy

and more 1 locations

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

Phase 1
Completed
Conditions
Muscular Dystrophy, Duchenne
First Posted Date
2018-12-07
Last Posted Date
2024-11-14
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
41
Registration Number
NCT03769116
Locations
🇺🇸

David Geffen School of Medicine at UCLA, Los Angeles, California, United States

🇺🇸

Nationwide Children's Hospital, Columbus, Ohio, United States

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

Phase 1
Terminated
Conditions
Muscular Dystrophy, Duchenne
Interventions
First Posted Date
2018-09-18
Last Posted Date
2023-09-28
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
15
Registration Number
NCT03675126
Locations
🇺🇸

Connecticut Children's Medical Center, Hartford, Connecticut, United States

🇺🇸

NW FL Clinical Research Group, LLC, Gulf Breeze, Florida, United States

🇺🇸

Center for Integrative Rare Disease Research (CIRDR), Atlanta, Georgia, United States

and more 5 locations

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

Phase 1
Active, not recruiting
Conditions
Limb-Girdle Muscular Dystrophy, Type 2E
First Posted Date
2018-08-29
Last Posted Date
2025-04-02
Lead Sponsor
Sarepta Therapeutics, Inc.
Target Recruit Count
6
Registration Number
NCT03652259
Locations
🇺🇸

Nationwide Children's Hospital, Columbus, Ohio, United States

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