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Conditions: Duchenne Muscular Dystrophy
Interventions: WVE-210201, Placebo; Drug
Lead sponsor: Wave Life Sciences USA, Inc.; Industry
Eligibility: 5 Years to 18 Years · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Duchenne Muscular Dystrophy
Interventions: WVE-210201 (suvodirsen), Placebo; Drug
Lead sponsor: Wave Life Sciences USA, Inc.; Industry
Eligibility: 5 Years to 12 Years · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 6
States / cities: New Haven, Connecticut • Atlanta, Georgia • Kansas City, Kansas + 3 more

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: Ultevursen, Sham-procedure; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 12 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 10
States / cities: San Diego, California • San Francisco, California • Miami, Florida + 7 more

Conditions: Duchenne Muscular Dystrophy
Interventions: SRP-4045, Placebo; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 7 Years to 21 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 3
States / cities: Los Angeles, California • Chicago, Illinois • Baltimore, Maryland

Conditions: Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
Interventions: Observational study with patients who may be treated with various disease-modifying therapies; Other
Lead sponsor: The Duchenne Registry; Other
Eligibility: Not listed
Enrollment: 2,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2072
U.S. locations: 10
States / cities: Little Rock, Arkansas • Sacramento, California • Aurora, Colorado + 6 more

Conditions: Muscular Dystrophy, Duchenne
Interventions: Eteplirsen; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years to 13 Years · Male only
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 3
States / cities: Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia

Conditions: Duchenne Muscular Dystrophy
Interventions: NS-065/NCNP-01, Placebo; Drug
Lead sponsor: NS Pharma, Inc.; Industry
Eligibility: 4 Years to 9 Years · Male only
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 6
States / cities: Sacramento, California • Gainesville, Florida • Chicago, Illinois + 3 more

Conditions: Non-Small Cell Lung Cancer Metastatic, Gastric Cancer, Esophageal Squamous Cell Carcinoma, Head and Neck Squamous Cell Carcinoma, Colorectal Cancer
Interventions: MCLA-129, Osimertinib, Chemotherapy; Drug
Lead sponsor: Merus B.V.; Industry
Eligibility: 18 Years and older
Enrollment: 194 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 4
States / cities: Orange, California • Nashville, Tennessee • West Valley City, Utah + 1 more

Conditions: Muscular Dystrophy, Duchenne
Interventions: SRP-5051; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years and older · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 7
States / cities: Hartford, Connecticut • Gulf Breeze, Florida • Atlanta, Georgia + 4 more

Conditions: Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 8 Years and older
Enrollment: 81 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 10
States / cities: San Francisco, California • Miami, Florida • Atlanta, Georgia + 7 more

Conditions: Metastatic Non-Small Cell Lung Cancer
Interventions: Capmatinib; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021
U.S. locations: 1
States / cities: East Hanover, New Jersey

Conditions: Duchenne Muscular Dystrophy
Interventions: Viltolarsen; Drug
Lead sponsor: NS Pharma, Inc.; Industry
Eligibility: 8 Years and older · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Richmond, Virginia

Conditions: Duchenne Muscular Dystrophy
Interventions: Amondys 45, Exondys 51, Vyondys 53; Drug
Lead sponsor: Kevin Flanigan; Other
Eligibility: 6 Months and older · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: QR-421a, Sham-procedure (dose cohort 1&2 only); Drug · Other
Lead sponsor: ProQR Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 4
States / cities: Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more

Conditions: Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
Interventions: Zeleciment Rostudirsen (DYNE-251), Placebo; Drug
Lead sponsor: Dyne Therapeutics; Industry
Eligibility: 4 Years to 18 Years · Male only
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Hillsborough, North Carolina

Conditions: Duchenne Muscular Dystrophy (DMD)
Interventions: eteplirsen; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 7 Years to 16 Years · Male only
Enrollment: 109 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 37
States / cities: Phoenix, Arizona • Los Angeles, California • Sacramento, California + 32 more

Conditions: Duchenne Muscular Dystrophy
Interventions: Eteplirsen, Golodirsen, Casimersen; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: Male only
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2033
U.S. locations: 20
States / cities: Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 17 more

Conditions: Muscular Dystrophy, Duchenne
Interventions: Eteplirsen; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 7 Years to 21 Years · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 9
States / cities: Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more

Conditions: Duchenne Muscular Dystrophy
Interventions: WVE-N531; Drug
Lead sponsor: Wave Life Sciences USA, Inc.; Industry
Eligibility: 4 Years to 18 Years · Male only
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 2
States / cities: Little Rock, Arkansas • Atlanta, Georgia

Conditions: Duchenne Muscular Dystrophy
Interventions: Not listed
Lead sponsor: Cooperative International Neuromuscular Research Group; Network
Eligibility: 4 Years and older · Male only
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 8
States / cities: Sacramento, California • Stanford, California • Washington D.C., District of Columbia + 5 more

Conditions: Cancer
Interventions: Not listed
Lead sponsor: EMD Serono Research & Development Institute, Inc.; Industry
Eligibility: Not listed
Enrollment: 700 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 3
States / cities: Fort Lauderdale, Florida • Columbus, Ohio • Nashville, Tennessee

Conditions: Duchene Muscular Dystrophy
Interventions: Not listed
Lead sponsor: ITF Therapeutics LLC; Industry
Eligibility: 6 Years and older
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 9
States / cities: Little Rock, Arkansas • Washington D.C., District of Columbia • Chicago, Illinois + 6 more

Conditions: Muscular Dystrophy, Duchenne
Interventions: SRP-5051; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 7
States / cities: Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more

Conditions: Becker Muscular Dystrophy
Interventions: Not listed
Lead sponsor: Cooperative International Neuromuscular Research Group; Network
Eligibility: 4 Years and older · Male only
Enrollment: 85 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 13
States / cities: Sacramento, California • Washington D.C., District of Columbia • Gainesville, Florida + 10 more

Search by objective public record fields.

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

Duchenne Electronic Health Record Study

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

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Phase 1/2 Study Evaluating MCLA-129, a Human Anti-EGFR, Anti-c-MET Bispecific Antibody, in Advanced NSCLC and Other Solid Tumors, Alone and in Combination

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

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Real-World Assessment of Clinical Outcomes in Metastatic NSCLC Patients With MET Exon 14 Skipping Mutation and Brain Metastases Treated With Capmatinib

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Study of Eteplirsen in DMD Patients

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

Met Non Small Cell Cancer Registry (MOMENT)

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping