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Clinical trials for neurofibromatosis type 1

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    The EU Clinical Trials Register currently displays   43850   clinical trials with a EudraCT protocol, of which   7282   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).

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    19 result(s) found for: neurofibromatosis type 1. Displaying page 1 of 1.
    EudraCT Number: 2011-001789-16 Sponsor Protocol Number: PenCTU/2011/CTIMP-005 Start Date*: 2011-08-26
    Sponsor Name:Plymouth Hospital NHS Trust
    Full Title: Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas
    Medical condition: Neurofibromatosis, type 2 (acoustic neurofibromatosis)
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10029271 Neurofibromatosis, type 2 (acoustic neurofibromatosis) LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-003405-26 Sponsor Protocol Number: NF1-EXCEL Start Date*: 2014-09-03
    Sponsor Name:Erasmus MC
    Full Title: The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL)
    Medical condition: Neurofibromatosis type 1
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing) BE (Ongoing) DK (Prematurely Ended) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-005770-26 Sponsor Protocol Number: KRONF2 Start Date*: 2023-10-20
    Sponsor Name:Medical University of Warsaw
    Full Title: Phase 2a non-commercial and non-randomized intervention study evaluating the efficacy of crizotinib in the treatment of children with severe type 2 neurofibromatosis, in particular those excluded f...
    Medical condition: Neurofibromatosis type 2 is a genetically determined primary malignancy resulting from a mutation that disables the function of the cell division control gene and leads to neoplasia such as benign ...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000523 Acoustic neuroma PT
    27.0 10010331 - Congenital, familial and genetic disorders 10029271 Neurofibromatosis, type 2 (acoustic neurofibromatosis) LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: PL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-005742-38 Sponsor Protocol Number: SANTA2012 Start Date*: 2013-05-02
    Sponsor Name:Central Manchester University Hospitals NHS Foundation Trust
    Full Title: Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1
    Medical condition: Neurofibromatosis Type 1
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2020-005648-52 Sponsor Protocol Number: D1346C00015 Start Date*: 2021-05-31
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase I, Single-Arm, Sequential Study to Evaluate the Effect of Food on the Gastrointestinal Toxicity and Pharmacokinetics of Selumetinib after Multiple Doses in Adolescent Children with Neurofib...
    Medical condition: Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: PL (Completed) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-005608-20 Sponsor Protocol Number: D1346C00004 Start Date*: 2021-11-24
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase I/II, Single-Arm, Open label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥ 1 to < 7 Years with Neurofibr...
    Medical condition: Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Ongoing) ES (Ongoing) NL (Prematurely Ended) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005607-39 Sponsor Protocol Number: D134BC00001 Start Date*: 2021-10-05
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase III, Multicentre, International Study with a Parallel, Randomised, Double blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with...
    Medical condition: Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Restarted) PL (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-005022-10 Sponsor Protocol Number: SYN-1748-MAL-0030-I Start Date*: 2018-02-13
    Sponsor Name:Technische Universität München, Fakultät für Medizin
    Full Title: Improvement of synaptic plasticity and cognitive function in RAS pathway disorders
    Medical condition: Noonan Syndrom and Neurofibromatosis Type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029748 Noonan syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10029268 Neurofibromatosis PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2021-001572-42 Sponsor Protocol Number: FCN-159-002 Start Date*: 2021-12-21
    Sponsor Name:Shanghai Fosun Pharmaceutical Industrial Development Co.,Ltd.
    Full Title: A multi-center, open-label, single-arm Phase I dose-escalation and Phase II dose-expansion study to evaluate the safety, tolerability, PK characteristics and anti-tumor activity of FCN-159 in adult...
    Medical condition: Participants diagnosed with Neurofibromatosis NF1-related plexiform neurofibromas (PN) and symptomatic with requirement of systematic therapy per investigator’s judgment. A PN is defined as a neuro...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) IT (Ongoing) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-001563-36 Sponsor Protocol Number: CRAD001MIL04T Start Date*: 2016-05-12
    Sponsor Name:Novartis Pharmaceuticals
    Full Title: A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)
    Medical condition: Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
    Disease:
    Population Age: Children, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2018-001846-32 Sponsor Protocol Number: BUS2018-1 Start Date*: 2018-09-18
    Sponsor Name:VO Barnmedicin, Skånes University Hospital
    Full Title: Treatment of NF1-related plexiform neurofibroma with trametinib; a single arm, open-label trial with the goals of volumetric partial remission and pain relief
    Medical condition: NF1-related plexiform neurofibroma
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-000156-35 Sponsor Protocol Number: NF2PET Start Date*: 2021-03-18
    Sponsor Name:Leiden University Medical Center
    Full Title: 89Zr-Bevacizumab PET/CT imaging of vestibular schwannomas for the prediction of bevacizumab treatment effect in patients with symptomatic neurofibromatosis type 2.
    Medical condition: Neurofibromatosis type 2
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-016922-15 Sponsor Protocol Number: HSJD-IMANF1 Start Date*: 2010-02-04
    Sponsor Name:HOSPITAL SANT JOAN DE DÉU
    Full Title: ENSAYO PILOTO CON IMATINIB PARA PACIENTES CON NEUROFIBROMA PLEXIFORME ASOCIADO A LA NEUROFIBROMATOSIS TIPO I. PILOT STUDY WITH IMATINIB FOR PLEXIFORM NEUROFIBROMA IN NEUROFIBROMATOSIS TYPE I PATIENTS
    Medical condition: Tratamiento de los neurofibromas plexiformes de alto riesgo no abordables quirúrgicamente en pacientes con neurofibromatosis tipo I
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-001317-16 Sponsor Protocol Number: TRAIN_2019 Start Date*: 2019-10-30
    Sponsor Name:ErasmusMC
    Full Title: The TRAIN study: TRAmetinib In Neurofibromatosis type 1 related symptomatic plexiform neurofibromas
    Medical condition: Adult patients (age >17 years) with (mosaic) NF1 with inoperable symptomatic plexiform neurofibromas
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029267 Neurofibroma PT
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-002635-41 Sponsor Protocol Number: 15HI53 Start Date*: 2018-09-26
    Sponsor Name:Great Ormond Street Hospital
    Full Title: A paediatric phase I/II study of intermittent dosing of the MEK-1 inhibitor selumetinib in children with neurofibromatosis type-1 and inoperable plexiform neurofibroma and/or progressive optic path...
    Medical condition: Neurofibromatosis type 1 associated plexiform neurofibromas Neurofibromatosis type 1 associated progressive or relapsed optic pathway glioma
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    20.0 100000004864 10065866 Plexiform neurofibroma LLT
    20.0 100000004864 10030935 Optic nerve glioma LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-000847-16 Sponsor Protocol Number: D1532C00057 Start Date*: 2019-01-29
    Sponsor Name:National Cancer Institute, Cancer Therapy Evaluation Program
    Full Title: A Phase 1/2 Study of the Mitogen Activated Protein Kinase Kinase(MEK) 1 Inhibitor Selumetinib (AZD6244, ARRY-142886, Hydrogen Sulfate) in Children With Neurofibromatosis Type 1 (NF1) and Inoperable...
    Medical condition: Neurofibromatosis type 1 (NF1)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029267 Neurofibroma PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2005-005377-29 Sponsor Protocol Number: CNS 2004 03 Start Date*: 2006-05-03
    Sponsor Name:Dept of Pediatrics, University Hospital of North Norway
    Full Title: SIOP-LGG 2004 Cooperative multicenter Study for Children and Adolescents with Low Grade Gliome
    Medical condition: Low Grade Glioma
    Disease:
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NO (Ongoing) IE (Completed) GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-001982-26 Sponsor Protocol Number: OZM-063 Start Date*: 2018-04-26
    Sponsor Name:The Hospital for Sick Children
    Full Title: A phase II, open-labeled, multi-center, randomized controlled trial of Vinblastine +/- Bevacizumab for the treatment of chemotherapy-naïve children with unresectable or progressive low grade glioma...
    Medical condition: Unresectable or progressive low grade glioma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10018338 Glioma PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2012-003005-10 Sponsor Protocol Number: IGR2012/1883 Start Date*: 2013-06-06
    Sponsor Name:Gustave Roussy
    Full Title: PHASE I-II STUDY OF VINBLASTINE IN COMBINATION WITH NILOTINIB IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH REFRACTORY OR RECURRENT LOW-GRADE GLIOMA
    Medical condition: Children, adolescents and young adults with refractory or recurrent low-grade gliomas, and children, adolescents and young adults with neurofibromatosis type 1 and previously untreated low-grade g...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065443 Malignant glioma PT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10038111 Recurrent cancer PT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10070308 Refractory cancer PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Completed) FR (Ongoing) NL (Completed) ES (Ongoing)
    Trial results: (No results available)
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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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