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ClinicalTrials.gov public records Last synced Oct 6, 2026, 1:05 AM EDT

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Showing 25–48 of 60 matching trials from the live ClinicalTrials.gov search.
Filters: "exon skipping" Clear all
Completed Phase 1 Interventional Results available

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

NCT02530905
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Oct 6, 2026, 1:05 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Tepotinib Phase II in NSCLC Harboring MET Alterations (VISION)

NCT02864992
View directory record Official record
Conditions
Advanced (Stage IIIB/IV) Non-small Cell Lung Cancer (NSCLC) With MET Exon 14 (METex14) Skipping Alterations or MET Amplification, Lung Adenocarcinoma Stage IIIB/IV
Interventions
Tepotinib
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older
Enrollment
337 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
39
States / cities
Duarte, California • Encinitas, California • Orange, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Exon 44
Interventions
AOC 1044, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 55 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2025 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 2 Interventional Results available

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

NCT02740972
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 7, 2021 · Synced Oct 6, 2026, 1:05 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

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)

NCT05158296
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 12, 2024 · Synced Oct 6, 2026, 1:05 AM EDT
Recruiting No phase listed Observational

Duchenne Electronic Health Record Study

NCT07609394
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 2 Interventional Results available

Study of Oral cMET Inhibitor INC280 in Patients With EGFR Wild-type (wt), Advanced Non-small Cell Lung Cancer (NSCLC) (Geometry Mono-1)

NCT02414139
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung
Interventions
Capmatinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
373 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
13
States / cities
Long Beach, California • Los Angeles, California • Orange, California + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2024 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 3 Interventional Results available

Study of Eteplirsen in DMD Patients

NCT02255552
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 6, 2026, 1:05 AM EDT
Available No phase listed Expanded access

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

NCT07250737
View directory record Official record
Conditions
Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies, Muscular Dystrophy, Duchenne
Interventions
delpacibart zotadirsen
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
2 Years and older · Male only
U.S. locations
21
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Irvine, California + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Terminated Phase 3 Interventional Results available

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

NCT03532542
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Casimersen, Golodirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 23 Years · Male only
Enrollment
171 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 21 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 6, 2026, 1:05 AM EDT
Active, not recruiting Phase 1 Interventional

Study of TPX-0022 in Patients With Advanced NSCLC, Gastric Cancer or Solid Tumors Harboring Genetic Alterations in MET

NCT03993873
View directory record Official record
Conditions
Advanced Solid Tumor, Metastatic Solid Tumors, MET Gene Alterations
Interventions
elzovantinib (TPX-0022)
Drug
Lead sponsor
Turning Point Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Denver, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 6, 2026, 1:05 AM EDT
Recruiting No phase listed Observational

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

NCT07127978
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 3 Interventional

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT02500381
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, SRP-4053, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
6 Years to 13 Years · Male only
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
23
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Oct 6, 2026, 1:05 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

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

NCT04868877
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Withdrawn Phase 2 Interventional

QUILT-3.036: AMG 337 in Subjects With Advanced or Metastatic Solid Tumors

NCT03147976
View directory record Official record
Conditions
Solid Tumor
Interventions
AMG 337
Drug
Lead sponsor
NantPharma, LLC
Industry
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
El Segundo, California
Source: ClinicalTrials.gov public record
Updated Aug 28, 2019 · Synced Oct 6, 2026, 1:05 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

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

NCT03675126
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 2 Interventional Results available

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01462292
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
GSK2402968 3mg/kg/week, GSK2402968 6 mg/kg/week, Placebo to match GSK2402968 3 mg/kg/week, Placebo to match GSK2402968 6 mg/kg/week
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
5 Years and older · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
14
States / cities
Sacramento, California • Stanford, California • Gulf Breeze, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2017 · Synced Oct 6, 2026, 1:05 AM EDT
Recruiting No phase listed Observational

Met Non Small Cell Cancer Registry (MOMENT)

NCT05376891
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

NCT05176717
View directory record Official record
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
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
12 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
2
States / cities
Dallas, Texas • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 7, 2024 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 2 Interventional Results available

A Study of Capmatinib (INC280) in NSCLC Patients With MET Exon 14 Alterations Who Have Received Prior MET Inhibitor

NCT02750215
View directory record Official record
Conditions
Malignant Non-small Cell Neoplasm of Lung Stage IV
Interventions
Capmatinib (INC280)
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 14, 2022 · Synced Oct 6, 2026, 1:05 AM EDT
Recruiting No phase listed Observational

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

NCT07092540
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
0 Days to 3 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Rochester, New York • Stony Brook, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NCT04060199
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 6, 2026, 1:05 AM EDT
Completed Phase 2 Interventional Results available

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

NCT04956289
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 6, 2026, 1:05 AM EDT