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ClinicalTrials.gov public records Last synced Oct 3, 2026, 11:34 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 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 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM EDT
Not listed No phase listed Observational

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

NCT01539772
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 15, 2018 · Synced Oct 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Platform Study in Patients With Advanced Non-Small Lung Cancer Who Progressed on First-Line Osimertinib Therapy (ORCHARD)

NCT03944772
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
Osimertinib, Savolitinib, Gefitinib, Necitumumab, Durvalumab, Carboplatin, Pemetrexed, Alectinib, Selpercatinib, Selumetinib, Etoposide, Cisplatin, Datopotamab deruxtecan
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
247 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
16
States / cities
Duarte, California • Los Angeles, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2025 · Synced Oct 3, 2026, 11:34 PM EDT
Active, not recruiting Phase 2 Interventional

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

NCT06627179
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Oct 3, 2026, 11:34 PM EDT
Recruiting Phase 2 Interventional

APL-101 Study of Subjects With NSCLC With c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors

NCT03175224
View directory record Official record
Conditions
Solid Tumors, Advanced Cancer, Renal Cancer, Gastric Cancer, Gastroesophageal Junction Adenocarcinoma, NSCLC, Lung Cancer, Brain Tumor, Glioblastoma Multiforme, EGFR Gene Mutation, MET Amplification, HGF, Thyroid Cancer, Pancreatic Cancer, Colon Cancer, MET Alteration, MET Fusion, Exon 14 Skipping
Interventions
APL-101 Oral Capsules
Drug
Lead sponsor
Apollomics Inc.
Industry
Eligibility
18 Years and older
Enrollment
497 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
6
States / cities
Los Angeles, California • Tampa, Florida • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2025 · Synced Oct 3, 2026, 11:34 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Study to Evaluate ANS014004 in Combination With EGFR-TKI in Non-Small Cell Lung Cancer

NCT07285148
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, EGFR Mutation, Metastatic Lung Cancer, MET Alteration
Interventions
ANS014004 + PLB1004, ANS014004 + Osimertinib
Drug
Lead sponsor
Beijing Pearl Biotechnology Limited Liability Company
Industry
Eligibility
18 Years and older
Enrollment
253 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Oct 3, 2026, 11:34 PM EDT
Terminated Phase 2 Interventional Results available

Phase II of Neoadjuvant and Adjuvant Capmatinib in NSCLC

NCT04926831
View directory record Official record
Conditions
Non-small Cell Lung Cancer
Interventions
capmatinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
6
States / cities
La Jolla, California • Sacramento, California • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 3, 2026, 11:34 PM EDT
Terminated Phase 2 Interventional

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

NCT04004065
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vesleteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
11
States / cities
Sacramento, California • Farmington, Connecticut • Gulf Breeze, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Oct 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM EDT
Active, not recruiting Phase 2 Interventional

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT05996003
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Exon 44, DMD
Interventions
NS-089/NCNP-02
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 14 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
11
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 3, 2026, 11:34 PM EDT
Completed No phase listed Observational

Treatment Patterns and Real-World Clinical Outcomes in Patients With Advanced NSCLC and MET Exon 14 Skipping Mutation in the United States

NCT06161051
View directory record Official record
Conditions
Advanced Non-small Cell Lung Cancer and MET Exon 14 Skipping Mutation
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
287 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Dec 7, 2023 · Synced Oct 3, 2026, 11:34 PM EDT
Completed Phase 2 Interventional Results available

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.

NCT04179409
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 26, 2023 · Synced Oct 3, 2026, 11:34 PM EDT
Active, not recruiting Phase 2 Interventional

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

NCT06244082
View directory record Official record
Conditions
DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
Interventions
AOC 1044
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 27 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 3, 2026, 11:34 PM EDT
Terminated Phase 2 Interventional Results available

Study of Capmatinib and Spartalizumab/Placebo in Advanced NSCLC Patients With MET Exon 14 Skipping Mutations

NCT04323436
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung
Interventions
Spartalizumab, Capmatinib, spartalizumab placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Oct 3, 2026, 11:34 PM EDT
Completed Phase 1Phase 2 Interventional

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

NCT03780257
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 (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
Source: ClinicalTrials.gov public record
Updated Apr 20, 2022 · Synced Oct 3, 2026, 11:34 PM EDT
Completed Phase 1Phase 2 Interventional Results available

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

NCT04240314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
scAAV9.U7.ACCA
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
6 Months to 13 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 3, 2026, 11:34 PM 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 3, 2026, 11:34 PM EDT
Completed No phase listed Observational

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

NCT05675683
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 9, 2023 · Synced Oct 3, 2026, 11:34 PM EDT