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Clinical trials for Nonsense mutations

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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    14 result(s) found for: Nonsense mutations. Displaying page 1 of 1.
    EudraCT Number: 2020-002337-15 Sponsor Protocol Number: 99625945 Start Date*: 2020-09-16
    Sponsor Name:Oslo University Hospital
    Full Title: TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY)
    Medical condition: Epidermolysis bullosa caused by nonsense mutations or splice site mutations
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NO (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-000966-12 Sponsor Protocol Number: EL-004 Start Date*: 2019-11-18
    Sponsor Name:Eloxx Pharmaceuticals Inc
    Full Title: A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis...
    Medical condition: Cystic fibrosis (CF)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2015-003031-35 Sponsor Protocol Number: R04049 Start Date*: 2015-11-20
    Sponsor Name:Central Manchester University Hospitals NHS Foundation Trust
    Full Title: A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitigating the Alloimmune Response to Intravenous Laronidase in Infants With Severe Mucopolysaccharido...
    Medical condition: Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10028094 Mucopolysaccharidosis IH LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-003159-12 Sponsor Protocol Number: J4B-MC-OKAA Start Date*: 2021-12-24
    Sponsor Name:Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company
    Full Title: A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)
    Medical condition: Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) ES (Temporarily Halted) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-020117-87 Sponsor Protocol Number: REACT-ON Terapia Start Date*: 2010-05-03
    Sponsor Name:FONDAZIONE SALVATORE MAUGERI - CLINICA DEL LAVORO E DELLA RIABILITAZIONE
    Full Title: REACT ON (REscuing ACTivity Of Na-channels) STUDY Study and correction of abnormalities of nonsense mutations in Brugada Syndrome.
    Medical condition: Brugada syndrome
    Disease: Version SOC Term Classification Code Term Level
    9.1 10007541 SOC
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2011-000410-18 Sponsor Protocol Number: 061002 Start Date*: 2011-05-05
    Sponsor Name:Baxter Innovations GmbH
    Full Title: A PHASE 3b CLINICAL STUDY TO ASSESS WHETHER REGULAR ADMINISTRATION OF ADVATE IN THE ABSENCE OF IMMUNOLOGICAL DANGER SIGNALS REDUCES THE INCIDENCE RATE OF INHIBITORS IN PREVIOUSLY UNTREATED PATIENTS...
    Medical condition: Prevention of inhibitor formation, and immune tolerance induction in patients with severe and moderately severe hemophilia A by early and low-dose prophylactic ADVATE therapy
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male
    Trial protocol: AT (Prematurely Ended) DE (Completed) SK (Prematurely Ended) LT (Prematurely Ended) GB (Prematurely Ended) HU (Prematurely Ended) CZ (Prematurely Ended) SE (Prematurely Ended) BE (Prematurely Ended) BG (Completed) ES (Prematurely Ended) NL (Prematurely Ended) GR (Prematurely Ended) PL (Prematurely Ended) PT (Completed)
    Trial results: View results
    EudraCT Number: 2020-003200-14 Sponsor Protocol Number: JR-141-GS31 Start Date*: 2022-05-16
    Sponsor Name:JCR Pharmaceuticals Co., Ltd.
    Full Title: A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients.
    Medical condition: Mucopolysaccharidosis type II
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-001163-30 Sponsor Protocol Number: ALID02307 Start Date*: 2015-05-11
    Sponsor Name:Genzyme, a Sanofi Company
    Full Title: A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase)
    Medical condition: Mucopolysaccharidosis I
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-003407-15 Sponsor Protocol Number: SRP-9001-104 Start Date*: 2023-08-08
    Sponsor Name:Sarepta Therapeutics, Inc
    Full Title: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in association with imlifidase in Subjects with Duchenne Muscular Dystrophy with pre-exis...
    Medical condition: Duchenne muscular dystrophy
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: ES (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    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
    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: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-003374-91 Sponsor Protocol Number: SRP-9001-301 Start Date*: 2022-09-12
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA...
    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
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: FR (Completed) BE (Completed) ES (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-002372-13 Sponsor Protocol Number: SRP-9001-303 Start Date*: 2023-01-19
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects...
    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
    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 (Trial now transitioned) SE (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-004390-41 Sponsor Protocol Number: BLU-667-1101 Start Date*: 2018-03-13
    Sponsor Name:Blueprint Medicines
    Full Title: A Phase 1/2 Study of the Highly-selective RET Inhibitor, BLU-667, in Patients with Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors
    Medical condition: Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10066914 Thyroid tumor LLT
    20.0 100000004864 10025044 Lung cancer LLT
    21.1 100000004864 10065252 Solid tumor LLT
    21.1 100000004864 10029514 Non-small cell lung cancer NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) NL (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2017-000800-59 Sponsor Protocol Number: LOXO-RET-17001 Start Date*: 2019-02-22
    Sponsor Name:Loxo Oncology, Inc. a wholly owned subsidiary of Eli Lilly and Company
    Full Title: A Phase 1/2 Study of Oral LOXO-292 in Patients with Advanced Solid Tumors, Including RET Fusion-Positive Solid Tumors, Medullary Thyroid Cancer, and Other Tumors with RET Activation (LIBRETTO-001)
    Medical condition: Male or female patients age 12 years or older with a locally advanced or metastatic solid tumor with evidence of a RET gene alteration in tumor and/or blood
    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) 10027105 Medullary thyroid cancer PT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) NL (Ongoing) DK (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
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