DeepMed Research

Sarepta Therapeutics

🇺🇸United States

Country: 🇺🇸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.

Clinical Trials

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

Interventions: Genetic: SRP-9003

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: 🇺🇸
St. Jude Children's Research Hospital, Memphis, Tennessee, United States
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, 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

Interventions: Genetic: delandistrogene moxeparvovec
Genetic: placebo

First Posted Date: 2021-10-27

Last Posted Date: 2024-12-10

Lead Sponsor: Sarepta Therapeutics, Inc.

Target Recruit Count: 126

Registration Number: NCT05096221

Locations: 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸
UC San Diego Altman Clinical and Translational Research Institute, La Jolla, 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

Interventions: Genetic: delandistrogene moxeparvovec

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
🇺🇸
Washington University in St. Louis, Saint Louis, Missouri, United States
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, 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: Drug: Vesleteplirsen

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: 🇮🇹
A.O.U. Citta della Salute e della Scienza di Torino - SS Malattie Neuromuscolari, Department of Neurosciences, Torino, Italy
🇳🇱
Leiden University Medical Center, Leiden, Netherlands
🇪🇸
Hospital Sant Joan de Déu. U.B., Barcelona, Spain

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: Drug: Eteplirsen

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: 🇮🇳
Royal Institute of Child Neurosciences, Ahmedabad, India
🇩🇪
Charité Universitätsmedizin Berlin CVK, Berlin, Germany
🇮🇳
Aster RV Hospital, Bengaluru, India

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: Drug: Eteplirsen

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: 🇫🇷
Hopital Trousseau, Bâtiment lemariey, Paris, France
🇧🇪
UZ-Gent, Gent, Belgium
🇮🇹
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

Interventions: Genetic: delandistrogene moxeparvovec
Genetic: placebo

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: 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸
David Geffen School of Medicine at UCLA, Los Angeles, California, 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: Drug: SRP-5051

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
🇺🇸
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸
NW FL Clinical Research Group, LLC, Gulf Breeze, Florida, 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

Interventions: Genetic: SRP-9003

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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