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Clinical trials for Splicing

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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    17 result(s) found for: Splicing. Displaying page 1 of 1.
    EudraCT Number: 2017-000327-27 Sponsor Protocol Number: ISIS396443-CS2 Start Date*: 2017-01-26
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-002246-41 Sponsor Protocol Number: BP29420 Start Date*: 2014-10-23
    Sponsor Name:F. Hoffmann-La Roche Ltd.
    Full Title: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF T...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) ES (Completed)
    Trial results: View results
    EudraCT Number: 2017-000621-12 Sponsor Protocol Number: ISIS396443-CS3A Start Date*: 2017-02-15
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-000673-37 Sponsor Protocol Number: VX12-770-113 Start Date*: 2017-03-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted
    Medical condition: cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-004596-72 Sponsor Protocol Number: EXCALIBUR Start Date*: 2016-04-06
    Sponsor Name:FONDAZIONE IRCCS "ISTITUTO NAZIONALE DEI TUMORI"
    Full Title: A phase II study evaluating the efficacy of enzalutamide and the role of ARv7 in metastatic castration resistant prostate cancer (mCRPC) patients with visceral disease
    Medical condition: metastatic prostate cancer resistant to castration with visceral disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10007453 Carcinoma of the prostate metastatic LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-000692-32 Sponsor Protocol Number: FARMPre-Proposalcode:TRS-2016-00 Start Date*: 2019-07-17
    Sponsor Name:DIP. MEDICINA DEI SISTEMI UNIVERSITà DEGLI STUDI DI ROMA TOR VERGATA
    Full Title: EFFICACY OF METFORMIN ON MOTILITY AND STRENGTH IN MYOTONIC DYSTROPHY TYPE 1. A randomized, double blind, placebo-controlled, multicenter clinical trial.
    Medical condition: Myotonic Distrophy type 1, confirmed by genetic testing, with a CTG expansion size >100
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013987 Dystrophia myotonica LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-002714-23 Sponsor Protocol Number: LLC1114 Start Date*: 2015-01-07
    Sponsor Name:Fondazione G.I.M.EM.A. Onlus Gruppo Italiano Malattie Ematologiche dell'Adulto
    Full Title: A phase 2 multicenter study to assess the activity and the safety of front-line Ibrutinib plus Rituximab (IR) in unfit patients with Chronic Lymphocytic Leukemia (CLL).
    Medical condition: Chronic Lymphocytic Leukemia
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004864 10024340 Leukemia lymphocytic chronic LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-001947-18 Sponsor Protocol Number: ISIS396443-CS4 Start Date*: 2015-07-07
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscula...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) SE (Completed) ES (Completed) IT (Completed) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2013-004422-29 Sponsor Protocol Number: ISIS396443-CS3B Start Date*: 2014-11-13
    Sponsor Name:Isis Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Mus...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) GB (Completed) ES (Completed) SE (Completed) DE (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2011-003821-93 Sponsor Protocol Number: VX11-661-101 Start Date*: 2013-03-13
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 2, Multicenter, Double Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subject...
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-004184-39 Sponsor Protocol Number: BP39054 Start Date*: 2017-09-05
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Trial now transitioned) DE (Completed) FR (Trial now transitioned) NL (Trial now transitioned) BE (Trial now transitioned) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-001870-16 Sponsor Protocol Number: ISIS396443-CS11 Start Date*: 2016-03-08
    Sponsor Name:Biogen Idec Research Limited
    Full Title: An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) SE (Completed) ES (Ongoing) FR (Completed) BE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2022-000889-18 Sponsor Protocol Number: DYNE101-DM1-201 Start Date*: 2023-02-08
    Sponsor Name:Dyne Therapeutics
    Full Title: A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myoton...
    Medical condition: Myotonic Dystrophy Type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-006574-23 Sponsor Protocol Number: AR101-PREVEnt Start Date*: 2022-08-30
    Sponsor Name:Aytu BioPharma, Inc.
    Full Title: A multicenter, randomized, double-blind, placebo-controlled study of enzastaurin for the prevention of arterial events in patients with vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 ...
    Medical condition: vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 mutations
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10014316 Ehlers-Danlos syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Ongoing) BE (Prematurely Ended) FR (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-002098-12 Sponsor Protocol Number: 232SM201 Start Date*: 2015-05-12
    Sponsor Name:Biogen Idec Research Limited
    Full Title: An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptom...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) GB (GB - no longer in EU/EEA) DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-002534-23 Sponsor Protocol Number: 3124001 Start Date*: 2018-02-08
    Sponsor Name:Orion Corporation Orion Pharma
    Full Title: SAFETY AND PHARMACOKINETICS OF ODM-208 IN PATIENTS WITH METASTATIC CASTRATION-RESISTANT PROSTATE CANCER
    Medical condition: Metastatic castration-resistant prostate cancer
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10076506 Castration-resistant prostate cancer LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: FI (Trial now transitioned) FR (Trial now transitioned) GB (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2014-003657-33 Sponsor Protocol Number: 232SM202 Start Date*: 2015-09-21
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects w...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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