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Clinical trials for Missense 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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    10 result(s) found for: Missense mutations. Displaying page 1 of 1.
    EudraCT Number: 2018-001437-40 Sponsor Protocol Number: KO-TIP-007 Start Date*: 2019-03-08
    Sponsor Name:Kura Oncology, Inc.
    Full Title: The AIM-HN and SEQ-HN Study: A 2 Cohort, Non-comparative, Pivotal Study Evaluating the Efficacy of Tipifarnib in Patients with Head and Neck Squamous Cell Carcinoma (HNSCC) with HRAS Mutations (AIM...
    Medical condition: Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma (HNSCC) with HRAS Mutations
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10063569 Metastatic squamous cell carcinoma PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) DE (Completed) GB (GB - no longer in EU/EEA) NO (Completed) BE (Completed) AT (Completed) NL (Completed) GR (Completed) DK (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-000070-29 Sponsor Protocol Number: 74668 Start Date*: 2022-09-15
    Sponsor Name:VU University Medical Center
    Full Title: A Proof-of-Concept Study to Explore the Potential Efficacy of Deferiprone in Patients With Pelizaeus-Merzbacher disease (PMD)
    Medical condition: Pelizaeus-Merzbacher disease
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-004822-13 Sponsor Protocol Number: GECP17/04 Start Date*: 2018-05-23
    Sponsor Name:Spanish Lung Cancer Group (SLCG/GECP)
    Full Title: An Open Label Phase II Study of Tipifarnib in Advanced Squamous Non-small Cell Lung Cancer with HRAS mutations
    Medical condition: squamous non-small cell lung cancer (SQ-NSCLC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10025044 Lung cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    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: 2015-003418-25 Sponsor Protocol Number: CTU/2014/120 Start Date*: 2016-03-29
    Sponsor Name:MeiraGTx UK II Ltd
    Full Title: An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with D...
    Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-003967-21 Sponsor Protocol Number: MGT009 Start Date*: 2017-06-09
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigme...
    Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-000925-38 Sponsor Protocol Number: UCL13/0076 Start Date*: 2017-01-04
    Sponsor Name:University College London (UCL)
    Full Title: GO-8: Gene therapy for haemophilia A using a novel serotype 8 capsid pseudotyped adeno-associated viral vector encoding Factor VIII-V3
    Medical condition: Severe Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    20.0 100000004850 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    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: 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: 2015-004535-12 Sponsor Protocol Number: KO-TIP-001 Start Date*: 2016-02-25
    Sponsor Name:Kura Oncology, Inc.
    Full Title: An Open Label Phase II Study of Tipifarnib in Advanced Non-Hematological Malignancies with HRAS Mutations
    Medical condition: Advanced Non-Hematological Malignancies with HRAS mutations
    Disease: Version SOC Term Classification Code Term Level
    18.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10048683 Advanced cancer LLT
    18.1 10018065 - General disorders and administration site conditions 10071971 H-ras gene mutation PT
    18.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10066474 Thyroid cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) BE (Completed) NL (Completed) GB (GB - no longer in EU/EEA) IT (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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