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Clinical trials for Exon skipping

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44335   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    41 result(s) found for: Exon skipping. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2015-002069-52 Sponsor Protocol Number: 4045-301 Start Date*: 2017-01-23
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Trial now transitioned) SE (Completed) DE (Completed) FR (Ongoing) ES (Trial now transitioned) CZ (Trial now transitioned) NL (Ongoing) IT (Trial now transitioned) BG (Trial now transitioned) PL (Trial now transitioned) IE (Trial now transitioned) DK (Trial now transitioned) GR (Completed) NO (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-001762-42 Sponsor Protocol Number: 4658-402 Start Date*: 2020-04-03
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular ...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: IE (Completed) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) ES (Ongoing) SE (Prematurely Ended) DE (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) NO (Trial now transitioned) HU (Trial now transitioned) CZ (Trial now transitioned) SI (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-005696-24 Sponsor Protocol Number: MS200095-0022 Start Date*: 2016-07-04
    Sponsor Name:Merck KGaA
    Full Title: A Phase II, single-arm trial to investigate tepotinib in advanced (locally advanced or metastatic) non-small cell lung cancer with MET exon 14 (METex14) skipping alterations or MET amplification (V...
    Medical condition: Advanced (stage IIIB/IV) non-small cell lung cancer with MET exon 14 (METex14) skipping alterations
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) DE (Trial now transitioned) ES (Trial now transitioned) FR (Trial now transitioned) AT (Completed) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-005478-24 Sponsor Protocol Number: DYNE251DMD201 Start Date*: Information not available in EudraCT
    Sponsor Name:Dyne Therapeutics, Inc
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participan...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: IE (Trial now transitioned) BE (Trial now transitioned) ES (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-000601-77 Sponsor Protocol Number: 5051-201 Start Date*: 2019-12-11
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IE (Prematurely Ended) ES (Temporarily Halted) GB (GB - no longer in EU/EEA) BE (Trial now transitioned) NL (Restarted) DE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-000951-29 Sponsor Protocol Number: 4658-102 Start Date*: 2017-06-22
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Medical condition: Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Completed) DE (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-002008-25 Sponsor Protocol Number: 4053-101 Start Date*: 2014-09-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) o...
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed) IT (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2017-004806-17 Sponsor Protocol Number: PQ-313-002 Start Date*: 2018-05-18
    Sponsor Name:Wings Therapeutics Inc.
    Full Title: A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subj...
    Medical condition: Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10014989 Epidermolysis bullosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Prematurely Ended) DE (Prematurely Ended) CZ (Completed) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-005000-26 Sponsor Protocol Number: 4658-us-201 Start Date*: 2017-03-16
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability, and Pharmacokinetics Study of AVI-4658 (Eteplirsen), a Phosphorodiamidate Morpholino Oligomer, Administ...
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-005024-28 Sponsor Protocol Number: 4658-204 Start Date*: 2017-03-28
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-004625-32 Sponsor Protocol Number: 4045-302 Start Date*: 2019-02-05
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: FR (Completed) DE (Completed) SE (Completed) BE (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA) CZ (Completed) PL (Completed) BG (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-001825-28 Sponsor Protocol Number: R119513 Start Date*: 2019-09-24
    Sponsor Name: The University of Manchester
    Full Title: Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscul...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-001757-54 Sponsor Protocol Number: APL-101-01 Start Date*: 2020-11-27
    Sponsor Name:Apollomics, Inc.
    Full Title: Phase 1 / 2 Multicenter Study of the Safety, Pharmacokinetics, and Preliminary Efficacy of APL-101 in Subjects with Non-Small Cell Lung Cancer with c-Met EXON 14 Skip Mutations and c-Met Dysregulat...
    Medical condition: Non-Small Cell Lung Cancer with c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065252 Solid tumor LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) GB (GB - no longer in EU/EEA) FI (Completed) HU (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002288-69 Sponsor Protocol Number: CINC280A2203 Start Date*: 2020-09-03
    Sponsor Name:Novartis Pharma AG
    Full Title: A Phase II, Open-Label, Multicenter Study of Capmatinib in Participants with MET exon 14 skipping mutation positive, advanced, Non Small-Cell Lung Cancer that has metastasized to the Brain
    Medical condition: cMET mutated NSCLC
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed)
    Trial results: View results
    EudraCT Number: 2015-001955-54 Sponsor Protocol Number: BMN-051-302 Start Date*: 2015-12-08
    Sponsor Name:BioMarin Nederland BV
    Full Title: An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: NL (Prematurely Ended) DE (Completed) BE (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2020-004803-15 Sponsor Protocol Number: 5051-102 Start Date*: 2023-09-18
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label Extension Study for Patients with Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-000485-18 Sponsor Protocol Number: 61186372PANSC2001 Start Date*: 2023-05-16
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Phase 1/2 Study Evaluating the Safety and Efficacy of Amivantamab and Capmatinib Combination Therapy in Unresectable Metastatic Non-small Cell Lung Cancer
    Medical condition: Unresectable Metastatic Non-small Cell Lung Cancer
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Ongoing) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-005023-92 Sponsor Protocol Number: 4658-203 Start Date*: 2017-03-28
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
    Medical condition: Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-005002-19 Sponsor Protocol Number: 4658-301 Start Date*: 2017-03-28
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-005001-39 Sponsor Protocol Number: 4658-us-202 Start Date*: 2017-03-16
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
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