Search clinical trials for: exon skipping

Total 220 results

PepGen Inc

Recruiting

A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)

Duchenne Muscular Dystrophy

Canada
SWOG Cancer Research Network
National Cancer Institute (NCI)

Not yet recruiting

Targeted Treatment for Advanced Non-Small Cell Lung Cancer That Has a MET Exon 14 Skipping Gene Change (An Expanded Lung-MAP Treatment Trial)

Stage IV Lung Cancer AJCC v8 | Recurrent Lung Non-Small Cell Carcinoma
Guangdong Provincial People's Hospital
Shanghai Junshi Bioscience Co., Ltd.

Recruiting

Neoadjuvant Immunotherapy in Rare Mutations Localized NSCLC

Non-Small Cell Lung Cancer | BRAF V600 Mutation | MET Amplification | MET Exon 14 Skipping Mutation | RET Driver Mutation | Erb-B2 Receptor Tyrosine Kinase Exon 20 Mutation

China
Sqy Therapeutics
Biotrial

Recruiting

Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy (AVANCE1)

Duchenne Muscular Dystrophy

France
Novartis Pharmaceuticals

Recruiting

A Post Approval Commitment Study on Tabrecta® (Capmatinib) in South Korea

Non-Small-Cell Lung Carcinoma

Korea, Republic of
Avidity Biosciences, Inc.

Recruiting

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44)

Duchenne Muscular Dystrophy | Exon 44

United States
Dyne Therapeutics

Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)

Duchenne Muscular Dystrophy (DMD)

United States, Belgium, Canada, United Kingdom, Australia, Spain, Italy
Collin Blakely
Novartis

Recruiting

Capmatinib Plus Trametinib for the Treatment of Metastatic Non-small Cell Lung Cancer With MET Exon 14 Skipping Mutation

Metastatic Lung Non-Small Cell Carcinoma | Stage IV Lung Cancer

United States
Novartis Pharmaceuticals

Recruiting

Study of Capmatinib in Indian Patients With MET Exon 14 Skipping Mutation Positive Advanced NSCLC.

Non-Small Cell Lung Carcinoma

India
Sunshine Lake Pharma Co., Ltd.

Not yet recruiting

Ningetinib in Advanced NSCLC Skipping Mutations With MET Exon 14 Skipping Mutations

Advanced NSCLC
Chungbuk National University Hospital
Merck KGaA, Darmstadt, Germany

Recruiting

Tepotinib in Solid Tumors Harboring MET Alterations

Solid Tumor | MET Amplification | MET Exon 14 Skipping Mutation

Korea, Republic of
Novartis Pharmaceuticals

Recruiting

Special Drug Use-results Surveillance of Tabrecta Tablets

Non-small Cell Lung Cancer

Japan
Hunan Province Tumor Hospital

Recruiting

Crizotinb or Standard Chemotherapy in Met Exon 14 Skipping Advanced NSCLC

Non-small Cell Lung Cancer

China
Apollomics Inc.

Recruiting

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

Gastric Cancer | Pancreatic Cancer | NSCLC | Lung Cancer | Brain Tumor | Glioblastoma Multiforme | Advanced Cancer | Colon Cancer | Solid Tumors | Thyroid Cancer | Renal Cancer | Gastroesophageal Junction Adenocarcinoma | EGFR Gene Mutation | MET Amplification | HGF | MET Alteration | MET Fusion | Exon 14 Skipping

United States, Spain, France, United Kingdom, Taiwan, Australia, Finland, Italy, Singapore, Hungary, Canada, Puerto Rico, Russian Federation
Novartis Pharmaceuticals

Active, not recruiting

Study of Capmatinib in Chinese Adult Patients With Advanced Non-small Cell Lung Cancer Harboring MET Exon 14 Skipping Mutation (GeoMETry-C)

Non-Small Cell Lung Cancer (NSCLC)

China
AstraZeneca

Active, not recruiting

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

Non-Small Cell Lung Cancer

Denmark, Italy, Spain, United States, Korea, Republic of, Japan, Netherlands, Norway, Sweden
Sarepta Therapeutics, Inc.

Active, not recruiting

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

Muscular Dystrophy, Duchenne

United States, Korea, Republic of, India, Slovenia, Turkey, United Kingdom, Germany, Switzerland, Spain, Czechia, France, Serbia, Taiwan, Netherlands, Mexico, Colombia, Denmark, New Zealand, Norway, Greece, Hungary, Ireland, Italy, Jo... and more
Sarepta Therapeutics, Inc.

Active, not recruiting

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

Duchenne Muscular Dystrophy

United States, Korea, Republic of, Italy, United Kingdom, Canada, Poland, Spain, France, Germany, Belgium, Australia, Czechia, Serbia, Greece, Argentina, Bulgaria, Denmark, Hungary, India, Ireland, Israel, Mexico, Russian Federation, ...
Novartis Pharmaceuticals

Completed

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

Carcinoma, Non-Small-Cell Lung

Italy, Spain, Germany, Japan, Taiwan, Belgium, Netherlands, Singapore, Argentina, France, Israel, Sweden, Korea, Republic of, Lebanon, United Kingdom, United States, Austria, Mexico, Norway, Russian Federation
Daiichi Sankyo Co., Ltd.
Orphan Disease Treatment Institute Co., Ltd.

Completed

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Japan
Novartis Pharmaceuticals

Available

Treatment Plan CINC280A02001M to Provide Access to Capmatinib, for MET Exon 14 Skipping Non-Small Cell Lung Cancer (NSCLC)

Non-Small Cell Lung Cancer | Nonsmall Cell Lung Cancer | Carcinoma, Non-Small Cell Lung | Non-Small-Cell Lung Carcinoma
Avidity Biosciences, Inc.

Enrolling by invitation

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping (EXPLORE44OLE)

Duchenne Muscular Dystrophy | DMD | Exon 44 | Duchenne

United States
Novartis Pharmaceuticals

Terminated

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

Carcinoma, Non-Small-Cell Lung

Belgium, Germany, United States, Canada, France, Italy, Japan, Korea, Republic of, Spain
University College, London
Radboud University Medical Center; Groupe Hospitalier Pitie-Salpetriere; Leiden... and other collaborators

Completed

Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study

Duchenne Muscular Dystrophy

Netherlands, France, United Kingdom
Novartis Pharmaceuticals

Active, not recruiting

Phase II of Neoadjuvant and Adjuvant Capmatinib in NSCLC (Geometry-N)

Non-small Cell Lung Cancer

United States
Novartis Pharmaceuticals

Completed

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

Advanced Non-small Cell Lung Cancer and MET Exon 14 Skipping Mutation

United States
Sarepta Therapeutics, Inc.

Active, not recruiting

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

Duchenne Muscular Dystrophy

United States, Belgium, Spain, United Kingdom, Netherlands, Germany, Canada, Italy
Novartis Pharmaceuticals

Completed

Study of Capmatinib Efficacy in Comparison With Docetaxel in Previously Treated Participants With Non-small Cell Lung Cancer Harboring MET Exon 14 Skipping Mutation (GeoMETry-III)

Carcinoma, Non-Small-Cell Lung

Belgium, Germany, Italy, France, Netherlands, Spain, Korea, Republic of, India, Brazil, Portugal
Turning Point Therapeutics, Inc.

Active, not recruiting

Study of TPX-0022 in Patients With Advanced NSCLC, Gastric Cancer or Solid Tumors Harboring Genetic Alterations in MET (SHIELD-1)

Advanced Solid Tumor | Metastatic Solid Tumors | MET Gene Alterations

United States, France, Korea, Democratic People's Republic of, Korea, Republic of, Spain
Kevin Flanigan
Sarepta Therapeutics, Inc.

Completed

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.

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.

Terminated

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

Muscular Dystrophy, Duchenne

United States, Canada
Sarepta Therapeutics, Inc.

Terminated

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

Duchenne Muscular Dystrophy

United States, Belgium, Canada, Spain, United Kingdom, Czechia, Germany, Israel, Bulgaria, France, Italy, Poland, Sweden
Sarepta Therapeutics, Inc.

Terminated

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

Duchenne Muscular Dystrophy

Belgium, France, Italy, United Kingdom
Daiichi Sankyo Co., Ltd.

Active, not recruiting

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Japan
EMD Serono Research & Development Institute, Inc.
Merck KGaA, Darmstadt, Germany

Active, not recruiting

Tepotinib Phase II in NSCLC Harboring MET Alterations (VISION)

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

Spain, Taiwan, United States, Korea, Republic of, Netherlands, Israel, Belgium, Italy, China, France, Germany, Japan, Poland, Austria, Switzerland
Pfizer

Completed

A Study Of Oral PF-02341066, A C-Met/Hepatocyte Growth Factor Tyrosine Kinase Inhibitor, In Patients With Advanced Cancer (PROFILE 1001)

Systemic Anaplastic Large-Cell Lymphoma | Non-Small Cell Lung Cancer ALK-positive | Non-Small Cell Lung Cancer c-Met Dependent | Non-Small Cell Lung Cancer ROS Marker Positive | Advanced Malignancies Except Leukemia

United States, Korea, Republic of, Australia, Japan
ProQR Therapeutics

Terminated

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)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, United Kingdom
Novartis Pharmaceuticals

Completed

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

Metastatic Non-Small Cell Lung Cancer

United States
ProQR Therapeutics

Active, not recruiting

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)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Netherlands, United Kingdom, Germany
Sarepta Therapeutics, Inc.

Completed

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

Muscular Dystrophy, Duchenne

United States, Canada
ProQR Therapeutics

Completed

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

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Canada, France
Massachusetts General Hospital
Novartis

Completed

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

Malignant Non-small Cell Neoplasm of Lung Stage IV

United States
Sarepta Therapeutics, Inc.

Completed

Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

Duchenne Muscular Dystrophy

Belgium, France, Italy, United Kingdom
Phoenicis Therapeutics

Terminated

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

Epidermolysis Bullosa Dystrophica, Recessive | Epidermolysis Bullosa Dystrophica, Dominant

United States, Spain, France
Sarepta Therapeutics, Inc.

Completed

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

Duchenne Muscular Dystrophy

United States
Novartis Pharmaceuticals

Withdrawn

A Phase II, Open-Label, Multicenter Study of Capmatinib in Subjects With MET Exon 14 Skipping Mutation Positive, Advanced, NSCLC With Brain Metastases

Non-small Cell Lung Carcinoma (NSCLC)
Sarepta Therapeutics, Inc.
University College, London; Catholic University of the Sacred Heart; University... and other collaborators

Completed

Phase I/II Study of SRP-4053 in DMD Patients

Duchenne Muscular Dystrophy

United Kingdom, United States, France, Italy
National Center of Neurology and Psychiatry, Japan
Nippon Shinyaku Co., Ltd.

Completed

Exploratory Study of NS-065/NCNP-01 in DMD

Duchenne Muscular Dystrophy

Japan
Asan Medical Center

Unknown

Capmatinib in Patients With Non-small Cell Lung Cancer Harboring cMET exon14 Skipping Mutation

Cancer | Lung Cancer Metastatic | MET Gene Mutation

Korea, Republic of
Novartis Pharmaceuticals

Withdrawn

Study of Oral cMET Inhibitor INC280 in Chinese Patients With EGFR Wild-type Advanced Non-small Cell Lung Cancer (NSCLC)

Carcinoma | Non-Small-Cell Lung Cancer

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