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 34. Neurofibromatosis
 [ 98 clinical trials,    130 drugs(DrugBank: 60 drugs),    84 target genes / 186 target pathways

Searched query = "Neurofibromatosis", "von Recklinghausen disease"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
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1JPRN-JapicCTI-19499915/10/20195 November 2019BeatNF2 trialA Randomized Double-blind Multicenter trial to Assess the Efficacy and Safety of Bevacizumab for Neurofibromatosis Type 2Neurofibromatosis type 2Intervention name : Bevacizumab (Genitical Recombination)
INN of the intervention : Bevacizumab (Genitical Recombination)
Dosage And administration of the intervention : 5mg/kg in a total volume of 100ml, div, every 2 week
Control intervention name : Isotonic sodium chloride solution
INN of the control intervention : -
Dosage And administration of the control intervention : 100ml, div
Kiyoshi SaitoMasazumi Fujii, Masao Kobayakawa, Akihiro Inano, Jun Sakuma, Taku Sato, Akio Morita, Mitsuhiro Hasegawa, Kaoru Kurisu, Takashi Tamiya, Takeo Goro, Shigeru Yamaguchi, Hirofumi NakatomiNot Recruiting1864BOTH60Phase 2Japan
2NCT03871257October 4, 20197 October 2019A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade GliomaA Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)Low Grade Glioma;Neurofibromatosis Type 1;Visual Pathway GliomaDrug: Carboplatin;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Selumetinib;Drug: Selumetinib Sulfate;Drug: Vincristine;Drug: Vincristine SulfateNational Cancer Institute (NCI)Not recruiting2 Years21 YearsAll290Phase 3
3NCT03433183October 2, 201922 October 2019SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath TumorsSARC031: A Phase 2 Trial of the MEK Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Combination With the mTOR Inhibitor Sirolimus for Patients With Unresectable or Metastatic Malignant Peripheral Nerve Sheath TumorsMalignant Peripheral Nerve Sheath Tumors;Neurofibromatosis 1Drug: Selumetinib;Drug: SirolimusSarcoma Alliance for Research through CollaborationUnited States Department of Defense;AstraZenecaRecruiting12 YearsN/AAll21Phase 2United States
4NCT03326388September 26, 20194 November 2019Intermittent Dosing Of Selumetinib In Childhood NF1 Associated TumoursA 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 Pathway GliomaNeurofibromatosis Type 1;Plexiform Neurofibroma;Optic Nerve GliomaDrug: SelumetinibGreat Ormond Street Hospital for Children NHS Foundation TrustAstraZenecaRecruiting3 Years18 YearsAll30Phase 1/Phase 2United Kingdom
5NCT03962543September 6, 20194 November 2019MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasA Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant MorbidityPlexiform Neurofibroma;Neurofibromatosis Type 1 (NF1)Drug: PD-0325901 oral capsuleSpringWorks Therapeutics, Inc.Recruiting2 YearsN/AAll100Phase 2United States
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6NCT04085159September 1, 201930 September 2019Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or SchwannomatosisImmunotherapy Targeting Neurofibromatosis or SchwannomatosisCancerBiological: Antigen-specific T cells CART/CTL and DCvacShenzhen Geno-Immune Medical InstituteRecruiting1 Year80 YearsAll100Phase 1/Phase 2China
7NCT03975829August 27, 201917 June 2019Pediatric Long-Term Follow-up and Rollover StudyAn Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)Diffuse Astrocytoma;Anaplastic Astrocytoma;Astrocytoma;Oligodendroglioma, Childhood;Anaplastic Oligodendroglioma;Glioblastoma;Pilocytic Astrocytoma;Giant Cell Astrocytoma;Pleomorphic Xanthoastrocytoma;Anaplastic Pleomorphic Xanthoastrocytoma;Angiocentric Glioma;Chordoid Glioma of Third Ventricle;Gangliocytoma;Ganglioglioma;Anaplastic Ganglioglioma;Dysplastic Gangliocytoma of Cerebrellum;Desmoplastic Infantile Astrocytoma and Ganglioglioma;Papillary Glioneuronal Tumor;Rosette-forming Glioneurona Tumor;Central Neurocytoma;Extraventricular Neurocytoma;Cerebellar Liponeurocytoma;Neurofibromatosis Type 1Drug: dabrafenib;Drug: trametinibNovartis PharmaceuticalsNot recruiting1 YearN/AAll250Phase 4
8NCT03741101June 10, 201930 September 2019Treatment of NF1-related Plexiform Neurofibroma With TrametinibTreatment of NF1-related Plexiform Neurofibroma With Trametinib; a Single Arm, Open-label Trial With the Goals of Volumetric Partial Remission and Pain ReliefNeurofibromatosis 1;Child;Neurofibroma, PlexiformDrug: TrametinibRegion SkaneNovartisRecruiting1 Year17 YearsAll15Phase 2Sweden
9NCT03826940February 19, 201929 April 2019From Molecules to Cognition: Inhibitory Mechanisms in ASD and NF1Linking Inhibition From Molecular to Systems and Cognitive Levels: a Preclinical and Clinical Approach in Autism Spectrum Disorders and Neurofibromatosis.Autism Spectrum Disorder;Neurofibromatosis 1Drug: Lovastatin 60 MG;Drug: PlacebosUniversity of CoimbraRecruiting16 Years65 YearsAll30N/APortugal
10EUCTR2018-001846-32-SE18/09/201824 September 2018Treatment of symptomatic plexiform neurofibromas, a benign tumour associated with the disorder Neurofibromatosis type 1, in children with the drug trametinibTreatment of NF1-related plexiform neurofibroma with trametinib; a single arm, open-label trial with the goals of volumetric partial remission and pain relief - plexifpcNF1-related plexiform neurofibroma;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Mekinist
Product Name: Mekinist
Pharmaceutical Form: Oral drops
VO Barnmedicin, Skånes University HospitalAuthorisedFemale: yes
Male: yes
15Phase 2Sweden
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11NCT03649165September 5, 201819 November 2018A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male ParticipantsA Phase I, Open-label, Single-center Relative Bioavailability and Food Effect Randomized Crossover Study of New Granule and Capsule Formulations of Selumetinib (AZD6244) in Healthy Male SubjectsNeurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs);Healthy ParticipantsDrug: Treatment A;Drug: Treatment B;Drug: Treatment C;Drug: Treatment D;Drug: AcetaminophenAstraZenecaNot recruiting18 Years45 YearsMale24Phase 1United States
12NCT03688568September 1, 20181 October 2018Study of Imatinib in Children With Neurofibromatosis and Airway TumorsPhase 2 Study of Imatinib in Children With Neurofibromatosis and Airway TumorsNeurofibroma, PlexiformDrug: Imatinib MesylateIndiana UniversityFood and Drug Administration (FDA)Recruiting6 Months12 YearsAll20Phase 2United States
13NCT03079999June 11, 201826 August 2019Study of Aspirin in Patients With Vestibular SchwannomaProspective, Randomized, Placebo-Controlled Phase II Trial of Aspirin for Vestibular SchwannomasVestibular Schwannoma;Acoustic Neuroma;Neurofibromatosis 2Drug: Aspirin;Drug: PlaceboMassachusetts Eye and Ear InfirmaryUnited States Department of Defense;Massachusetts General HospitalRecruiting12 YearsN/AAll300Phase 2United States
14ChiCTR18000160402018-05-0914 May 2018Clinical efficacy of Bevacizumab in the treatment of Type II NeurofibromatosisClinical efficacy of Bevacizumab in the treatment of Type II NeurofibromatosisType II NeurofibromatosisCase series:Bevacizumab therapy;Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University, School of MedicineRecruiting6BothCase series:30;OtherChina
15NCT03513757March 4, 201811 June 2018Dexmedetomidine and Propofol for Pediatric MRI SedationAn Observer-blinded Randomized Study of Propofol Infusion vs Bolus Dexmedetomidine and Propofol Sedation for Pediatric Magnetic Resonance ImagingHeadache;Tumor;Seizure Disorder;Neurofibromatoses;Hydrocephalus;Abdominal Neoplasm;Spine DeformityDrug: propofol;Drug: DexmedetomidineMedical College of WisconsinRecruiting12 Months60 MonthsAll70Phase 4United States
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16EUCTR2016-005022-10-DE13/02/201823 July 2018Improvement of synaptic plasticity and cognitive function in RAS pathway disordersImprovement of synaptic plasticity and cognitive function in RAS pathway disordersNoonan Syndrom and Neurofibromatosis Type 1
MedDRA version: 20.0 Level: PT Classification code 10029748 Term: Noonan syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: PT Classification code 10029268 Term: Neurofibromatosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Not possible to specify
Product Name: Lovastatin
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Lovastatin
CAS Number: 75330-75-5
Other descriptive name: LOVASTATIN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
Product Name: Lamotrigin
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Lamotrigin
CAS Number: 84057-84-1
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
Technische Universität München Fakultät für MedizinAuthorisedFemale: yes
Male: yes
28Phase 2Germany
17NCT03232892February 13, 201829 July 2019Trametinib in Patients With Advanced Neurofibromatosis Type 1 (NF1)-Mutant Non-small Cell Lung CancerPhase II Trial to Evaluate Trametinib in Patients With Advanced NF1-mutant Non-small Cell Lung CancerNon-small Cell Lung CancerDrug: TrametinibUniversity of California, San FranciscoNovartis Pharmaceuticals;American Cancer Society, Inc.Recruiting18 YearsN/AAll27Phase 2United States
18NCT03259633February 9, 201811 November 2019An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)NF type1 With Inoperable Plexiform NeurofibromasDrug: SelumetinibAstraZenecaNot recruiting2 Years130 YearsAllPhase 3United States
19NCT02700230December 8, 201715 April 2019Vaccine Therapy in Treating Patients With Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by SurgeryPhase I Trial of Intratumoral Administration of a NIS-Expressing Derivative Manufactured From a Genetically Engineered Strain of Measles Virus in Patients With Unresectable or Recurrent Malignant Peripheral Nerve Sheath TumorMetastatic Malignant Peripheral Nerve Sheath Tumor;Neurofibromatosis Type 1;Recurrent Malignant Peripheral Nerve Sheath TumorProcedure: Computed Tomography;Other: Laboratory Biomarker Analysis;Biological: Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter;Other: Quality-of-Life Assessment;Procedure: Single Photon Emission Computed TomographyMayo ClinicNational Cancer Institute (NCI)Recruiting18 YearsN/AAll30Phase 1United States
20NCT03231306November 28, 201723 April 2019Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform NeurofibromasA Phase II Study of Binimetinib in Children and Adults With NF1 Associated Plexiform Neurofibromas (PNOC010)Neurofibromatosis Type 1;Plexiform NeurofibromaDrug: BinimetinibUniversity of Alabama at BirminghamArray BioPharma;Pacific Pediatric Neuro-Oncology ConsortiumRecruiting1 YearN/AAll40Phase 2United States
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21NCT03190915October 6, 20174 November 2019Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic LeukemiaA Phase 2 Study of the MEK Inhibitor Trametinib (NSC# 763093) in Children With Relapsed or Refractory Juvenile Myelomonocytic LeukemiaJuvenile Myelomonocytic Leukemia;Neurofibromatosis Type 1Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: TrametinibNational Cancer Institute (NCI)Recruiting1 Month21 YearsAll24Phase 2United States
22NCT01968590August 16, 201723 April 2019Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)A Phase II Trial on the Effect of Low-Dose Versus High-Dose Vitamin D Supplementation on Bone Mass in Adults With Neurofibromatosis Type 1 (NF1)Neurofibromatosis Type 1 (NF1)Drug: CholecalciferolUniversity of UtahU.S. Army Medical Research and Materiel Command;Universitätsklinikum Hamburg-Eppendorf;University of British Columbia;Children's Hospital Medical Center, CincinnatiRecruiting25 Years40 YearsAll320Phase 2United States;Canada;Germany
23NCT03095248May 8, 201722 October 2019Trial of Selumetinib in Patients With Neurofibromatosis Type II Related TumorsPhase 2 Trial of Selumetinib in Patients With Neurofibromatosis Type II Related TumorsNeurofibromatosis 2;Vestibular Schwannoma;Meningioma;Ependymoma;GliomaDrug: SelumetinibChildren's Hospital Medical Center, CincinnatiAstraZenecaRecruiting3 Years45 YearsAll34Phase 2United States
24NCT03105258May 1, 201710 April 2017Selumetinib Pilot Study for Cutaneous NeurofibromasPilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (cNF)Neurofibromatosis Type 1;Cutaneous NeurofibromaDrug: SelumetinibUniversity of Alabama at BirminghamNational Cancer Institute (NCI)Not recruiting18 YearsN/AAll24Phase 2United States
25NCT02839720April 11, 201722 October 2019Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous NeurofibromaPilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (CNF)Cutaneous Neurofibroma;Neurofibromatosis Type 1;Optic Nerve GliomaOther: Laboratory Biomarker Analysis;Drug: Selumetinib;Drug: Selumetinib SulfateNational Cancer Institute (NCI)Recruiting18 YearsN/AAll24Phase 2United States
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26NCT03109301April 7, 201715 April 2019Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)A Phase II Trial of the Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Patients With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)Neoplasms, Nerve Tissue;Neurofibromatosis 1;Heredodegenerative Disorders, Nervous System;Peripheral Nervous System DiseasesDrug: Selumetinib (AZD6244 hyd sulfate) 50mg/dose;Drug: Selumetinib (AZD6244 hyd sulfate) 25mg/m2National Cancer Institute (NCI)Not recruiting3 Years99 YearsAll0Phase 2United States
27NCT03090971February 15, 201716 December 2017Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1Clinical Assessment of the Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With Neurofibromatosis Type 1Neurofibromatosis 1;Cutaneous NeurofibromaDrug: Diclofenac Sodium;Drug: Saline SolutionFundação Educacional Serra dos ÓrgãosNot recruiting18 YearsN/AAll7Phase 2Brazil
28NCT02964884November 20164 March 2019Interventions for Reading Disabilities in NF1Neurobiology and Treatment of Reading Disability in NF1Neurofibromatosis Type 1;Learning Disability;Reading Disability;NF1Drug: Lovastatin;Behavioral: reading tutoring intervention;Drug: Placebo Oral Tablet;Behavioral: Other Academic sham tutoringVanderbilt UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Recruiting8 Years20 YearsAll120Phase 2United States
29NCT02831257August 31, 201618 December 2018AZD2014 In NF2 Patients With Progressive or Symptomatic MeningiomasA Single Arm Phase 2 Study of the Dual mTORC1/mTORC2 Inhibitor AZD2014 Provided on an Intermittent Schedule for Neurofibromatosis 2 Patients With Progressive or Symptomatic MeningiomasNeurofibromatosis 2;MeningiomaDrug: AZD2014Massachusetts General HospitalAstraZeneca;United States Department of DefenseNot recruiting18 YearsN/AAll18Phase 2United States
30NCT02728388August 201628 October 2019Photodynamic Therapy for Benign Dermal Neurofibromas- Phase IITopical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase IINEUROFIBROMATOSIS 1Drug: aminolevulinic acidHarry T Whelan, MDRecruiting14 Years30 YearsAll30Phase 2United States
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31NCT02934256July 201616 December 2017Icotinib Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsIcotinib Hydrochloride Tablets Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsVestibular Schwannoma;Neurofibromatosis Type 2Drug: IcotinibLi PengBetta Pharmaceuticals Co.,Ltd.Recruiting16 Years50 YearsAll20Phase 2China
32JPRN-UMIN0000210302016/03/162 April 2019A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1Neurofibromatosis type 10.2% Sirolimus gel twice daily 24 weeks topical application
0.4% Sirolimus gel twice daily 24 weeks topical application
Placebo gel twice daily 24 weeks topical application
Osaka University HospitalNot Recruiting16years-old70years-oldMale and Female18Phase 2Japan
33EUCTR2016-001563-36-Outside-EU/EEA17 May 2016Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)Plexiform Neurofibroma Associated With Neurofibromatosis Type 1;Therapeutic area: Diseases [C] - Cancer [C04]Trade Name: Votubia
Product Name: Everolimus
Product Code: RAD001
Pharmaceutical Form: Tablet
Novartis PharmaceuticalsNot AvailableFemale: yes
Male: yes
26Phase 4Israel
34NCT02407405December 22, 20154 November 2019MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasPhase II Trial of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasNeurofibromatosis 1;Plexiform Neurofibromas (PN)Drug: SelumetinibNational Cancer Institute (NCI)Recruiting18 YearsN/AAll50Phase 2United States
35NCT02644512December 201513 June 2016MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasPhase II Trial of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasNeurofibromatosis 1;Plexiform NeurofibromasDrug: SelumetinibNational Cancer Institute (NCI)Recruiting18 Years99 YearsBoth50Phase 2United States
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36NCT02282917September 20158 April 2019Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and MeningiomaExploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and MeningiomaVestibular Schwannoma;Meningioma;Acoustic Neuroma;Neurofibromatosis Type 2Drug: AR-42Massachusetts Eye and Ear InfirmaryJohns Hopkins University;Mayo Clinic;Stanford University;Ohio State University;Nationwide Children's HospitalNot recruiting18 YearsN/AAll5Early Phase 1United States
37NCT02415153July 14, 201511 November 2019Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System TumorsA Phase I Trial of Pomalidomide for Children With Recurrent, Progressive, or Refractory CNS TumorsNeurofibromatosis Type 1;Recurrent Central Nervous System Neoplasm;Recurrent Childhood Brain Stem Glioma;Recurrent Childhood Visual Pathway Glioma;Refractory Central Nervous System NeoplasmOther: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: PomalidomideNational Cancer Institute (NCI)Not recruiting3 Years20 YearsAll42Phase 1United States
38NCT01345136July 1, 201511 March 2019Study of RAD001 for Treatment of NF2-related Vestibular SchwannomaA Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular SchwannomaNeurofibromatosis Type 2;Neuroma, AcousticDrug: RAD001, everolimusJonsson Comprehensive Cancer CenterNovartis PharmaceuticalsNot recruiting16 Years65 YearsAll4Phase 2United States
39NCT02390752April 29, 201515 July 2019PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)Phase I/II Trial of PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)Neurofibroma, Plexiform;Precursor Cell Lymphoblastic Leukemia-Lymphoma;Leukemia, Prolymphocytic, Acute;SarcomaDrug: PLX3397National Cancer Institute (NCI)Recruiting3 Years35 YearsAll81Phase 1/Phase 2United States
40NCT02332902February 201516 December 2017Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232TEverolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232TNeurofibromatosis 1Drug: EverolimusThe University of Texas Health Science Center, HoustonTexas Neurofibromatosis FoundationNot recruiting18 YearsN/AAll24Phase 2United States
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41NCT02211768December 8, 201430 September 2019Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1: Clinical, Histopathologic, and Genomic AnalysisNeurofibromatosis;MPNSTProcedure: MRI, FDG-PET/CT scans;Drug: [18F]-FLT-PET/CT scansNational Cancer Institute (NCI)Recruiting10 Years99 YearsAll15Phase 1United States
42NCT02256124October 201420 August 2018Effect of Lamotrigine on Cognition in NF1The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial (NF1-EXCEL)Neurofibromatosis Type 1Drug: Lamotrigine;Drug: PlaceboErasmus Medical CenterUniversitaire Ziekenhuizen Leuven;ZonMw: The Netherlands Organisation for Health Research and Development;Hospital Sant Joan de DeuRecruiting12 Years18 YearsAll60Phase 2/Phase 3Belgium;Netherlands;Spain
43JPRN-UMIN0000150812014/09/082 April 2019Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1neurofibromatosis type11) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks.
2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment.
If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks.
3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment.
If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks.
Department of Dermatology Graduate School of Medicine, Osaka UniversityRecruiting20years-oldNot applicableMale and Female3Phase 2,3Japan
44EUCTR2013-003405-26-NL03/09/20147 October 2014Treating learning disabilities in Neurofibromatosis 1 using lamotrigineThe effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL)Neurofibromatosis type 1
MedDRA version: 17.0 Level: LLT Classification code 10029270 Term: Neurofibromatosis, type 1 (von Recklinghausen's disease) System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Lamotrigine dispers
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-
INN or Proposed INN: lamotrigine
Other descriptive name: LAMOTRIGINE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Dispersible tablet
Route of administration of the placebo: Oral use
Erasmus MCAuthorisedFemale: yes
Male: yes
60Phase 2Netherlands
45NCT02096471June 201416 December 2017MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1A Phase 2 Trial of the MEK Inhibitor PD-0325901 in Adolescents and Adults With NF1-Associated Morbid Plexiform NeurofibromasNeurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PNDrug: PD-0325901University of Alabama at BirminghamNot recruiting16 YearsN/AAll19Phase 2United States
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PhaseCountries
46NCT02101736June 201423 April 2019Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and AdultsA Phase II Study of Cabozantinib (XL184) for Plexiform Neurofibromas in Subjects With Neurofibromatosis Type 1 in Children and AdultsNF1;Neurofibromatosis;Plexiform NeurofibromasDrug: CabozantinibUniversity of Alabama at BirminghamRecruiting3 YearsN/AAll48Phase 2United States
47NCT02177825June 201415 April 2019Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform NeurofibromasPhase II Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform NeurofibromasPlexiform NeurofibromasDrug: Imatinib MesylateSt. Justine's HospitalNot recruiting2 Years21 YearsAll5Phase 2Canada
48NCT02129647April 20149 September 2019Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular SchwannomasPhase II Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular SchwannomasNeurofibromatosis Type 2;Vestibular SchwannomasDrug: AxitinibNYU Langone HealthNot recruiting18 YearsN/AAll12Phase 2United States
49EUCTR2012-003005-10-DK06/06/201312 December 2016LOW-GRADE GLIOMAPHASE I-II STUDY OF VINBLASTINE IN COMBINATION WITH NILOTINIB IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH REFRACTORY OR RECURRENT LOW-GRADE GLIOMA - ViniloChildren, 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 gliomas
MedDRA version: 19.0 Level: PT Classification code 10065443 Term: Malignant glioma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 19.0 Level: PT Classification code 10038111 Term: Recurrent cancer System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 19.0 Level: PT Classification code 10070308 Term: Refractory cancer System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) ;Therapeutic area: Diseases [C] - Cancer [C04]
Product Name: Nilotinib
Product Code: AMN107
Pharmaceutical Form: Capsule, hard
Current Sponsor code: AMN107
Other descriptive name: NILOTINIB HYDROCHLORIDE MONOHYDRATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Trade Name: Tasigna 150mg
Product Name: Nilotinib
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: NILOTINIB
CAS Number: 641571-10-0
Trade Name: Tasigna 200mg
Product Name: Nilotinib
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: NILOTINIB
CAS Number: 641571-10-0
Product Name: Vinblastine
Pharmaceutical Form: Powder for solution for injection/infusion
Other descriptive name: VINBLASTINE SULPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Gustave RoussyAuthorisedFemale: yes
Male: yes
160Phase 2Spain;Denmark;Netherlands;Switzerland
50NCT01880749June 201325 March 2019Exploring the Activity of RAD001 in Vestibular Schwannomas and MeningiomasExploring the Activity of RAD001 in Vestibular Schwannomas and MeningiomasVestibular Schwannomas;Meningiomas;Neurofibromatosis Type 2Drug: RAD001New York University School of MedicineNot recruiting18 YearsN/AAll5Early Phase 1United States
No.TrialIDDate_
enrollement
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51NCT01767792May 15, 201315 July 2019Phase 2 Study of Bevacizumab in Children and Young Adults With NF 2 and Progressive Vestibular SchwannomasOpen-label, Phase 2 Study of Bevacizumab in Children and Young Adults With Neurofibromatosis 2 and Progressive Vestibular Schwannomas That Are Poor Candidates for Standard Treatment With Surgery or RadiationNeurofibromatosis Type 2;Progressive Vestibular SchwannomasDrug: BevacizumabUniversity of Alabama at BirminghamGenentech, Inc.Not recruiting6 YearsN/AAll22Phase 2United States
52EUCTR2012-005742-38-GB02/05/201321 August 2017Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1 - SimvAstatin in Neurofibromatosis Type 1-Autism (SANTA)Neurofibromatosis Type 1
MedDRA version: 14.1 Level: LLT Classification code 10029270 Term: Neurofibromatosis, type 1 (von Recklinghausen's disease) System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Trade Name: Simvastatin 20mg/5ml Oral Suspension
Product Name: Simvastatin
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Simvastatin
CAS Number: 79902-63-9
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 4-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Central Manchester University Hospitals NHS Foundation TrustNot RecruitingFemale: yes
Male: yes
0Phase 2United Kingdom
53NCT02584413April 16, 201316 September 2019Hypotonia and Neurofibromatosis Type 1 (NF1) GliomaHypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1Neurofibromatosis Type 1Device: Magnetic resonance imaging;Drug: Gadolinium contrastWashington University School of MedicineSt. Louis Children's HospitalNot recruiting1 Year7 YearsAll29N/AUnited States
54NCT01661283September 201224 October 2016SARC016: Study of Everolimus With Bevacizumab to Treat Refractory Malignant Peripheral Nerve Sheath TumorsPhase 2 Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath TumorsMalignant Peripheral Nerve Sheath Tumors;MPNST;SarcomaDrug: everolimus;Drug: bevacizumabSarcoma Alliance for Research through CollaborationNovartis Pharmaceuticals;Genentech, Inc.;United States Department of Defense;Novartis Pharmaceuticals;Genentech, Inc.;United States Department of DefenseNot recruiting18 YearsN/ABoth25Phase 2United States
55NCT01365468April 201219 October 2017Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)Plexiform Neurofibroma Associated With Neurofibromatosis Type 1Drug: Everolimus (RAD001)Novartis PharmaceuticalsNot recruiting6 YearsN/AAll9Phase 2Israel
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
56NCT01553149March 19, 201228 October 2019Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway GliomaA Phase II Randomized Trial of Lenalidomide (NSC # 703813) in Pediatric Patients With Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway GliomasNeurofibromatosis Type 1;Recurrent Childhood Pilocytic Astrocytoma;Recurrent Childhood Visual Pathway GliomaDrug: Lenalidomide;Other: Pharmacological StudyNational Cancer Institute (NCI)Not recruitingN/A21 YearsAll80Phase 2United States;Australia;Canada;New Zealand
57NCT01552434March 16, 201230 September 2019Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign DiseaseA Phase I Trial of Bevacizumab, Temsirolimus Alone and in Combination With Valproic Acid, or Cetuximab in Patients With Advanced Malignancy and Other IndicationsAdvanced Malignant Neoplasm;Castleman Disease;Digestive System Carcinoma;Erdheim-Chester Disease;Lip and Oral Cavity Carcinoma;Lymphangioleiomyomatosis;Malignant Endocrine Neoplasm;Malignant Female Reproductive System Neoplasm;Malignant Male Reproductive System Neoplasm;Malignant Neoplasm;Malignant Respiratory Tract Neoplasm;Malignant Thoracic Neoplasm;Malignant Urinary System Neoplasm;Mesothelial Neoplasm;Metastatic Malignant Neoplasm;Metastatic Urothelial Carcinoma;Neurofibromatosis Type 2;Recurrent Adult Soft Tissue Sarcoma;Recurrent Breast Carcinoma;Recurrent Childhood Soft Tissue Sarcoma;Recurrent Digestive System Carcinoma;Recurrent Female Reproductive System Carcinoma;Recurrent Male Reproductive System Carcinoma;Recurrent Malignant Neoplasm;Recurrent Pharyngeal Carcinoma;Recurrent Thyroid Gland Carcinoma;Refractory Malignant Neoplasm;Soft Tissue Neoplasm;Stage III Breast Cancer AJCC v7;Stage III Pharyngeal Cancer;Stage IIIA Breast Cancer AJCC v7;Stage IIIB Breast Cancer AJCC v7;Stage IIIC Breast Cancer AJCC v7;Stage IV Breast Cancer AJCC v6 and v7;Stage IV Pharyngeal Cancer;Stage IVA Pharyngeal Cancer;Stage IVB Pharyngeal Cancer;Stage IVC Pharyngeal Cancer;Thyroid Gland NeoplasmBiological: Bevacizumab;Biological: Cetuximab;Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Temsirolimus;Drug: Valproic AcidM.D. Anderson Cancer CenterNational Cancer Institute (NCI)RecruitingN/AN/AAll216Phase 1United States
58NCT01402817March 201216 December 2017Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform NeurofibromasA Pilot Study of Sutent®/Sunitinib (SU11248), an Oral Multi-Targeted Tyrosine Kinase Inhibitor in Subjects With NF-1 Plexiform NeurofibromasNeurofibromatosis;NF1;Plexiform NeurofibromasDrug: Sutent®/SunitinibIndiana UniversityUnited States Department of Defense;PfizerNot recruiting3 Years65 YearsAll19Phase 2United States
59NCT01140360February 201216 December 2017Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform NeurofibromasPilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform NeurofibromasNeurofibromatosis;NeurofibromasDrug: GleevecKent RobertsonNot recruiting3 Years65 YearsAll21Phase 1/Phase 2United States
60NCT01682811November 20114 November 2019Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)Photodynamic Therapy for Benign Dermal Neurofibromas Using Levulan Kerastick For Topical Solution, Plus Illumination With Red LightNeurofibromatosesDrug: Levulan (5-aminolevulinic acid) uptake.;Drug: Levulan (5-aminolevulinic acid) photodynamic therapy.Harry T Whelan, MDNot recruiting18 Years90 YearsAll10Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
61NCT01419639October 201116 December 2017Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2Neurofibromatosis Type IIDrug: Everolimus (RAD001) , Afinitor®New York University School of MedicineNovartis Pharmaceuticals;The Children's Tumor FoundationNot recruiting3 YearsN/AAll10Phase 2United States
62NCT01362803September 21, 201111 November 2019AZD6244 Hydrogen Sulfate for Children With Nervous System TumorsA Phase I/II Study of the Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor Selumetinib (AZD6244; HYD Sulfate) in Children With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)Neurofibromatosis 1;Neurofibromatosis Type 1;NF 1;Neurofibroma, PlexiformDrug: AZD6244National Cancer Institute (NCI)Not recruiting2 Years18 YearsAll99Phase 1/Phase 2United States
63EUCTR2011-001789-16-GB26/08/201119 March 2012Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas - Sorafenib in NF2Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas - Sorafenib in NF2Neurofibromatosis, type 2 (acoustic neurofibromatosis)
MedDRA version: 14.0 Level: LLT Classification code 10029271 Term: Neurofibromatosis, type 2 (acoustic neurofibromatosis) System Organ Class: 10010331 - Congenital, familial and genetic disorders
Trade Name: Nexavar
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: sorafenib (as tosylate)
Other descriptive name: Nexavar
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200mg-
Plymouth Hospital NHS TrustAuthorisedFemale: yes
Male: yes
14United Kingdom
64EUCTR2010-023508-28-GB14/04/201128 October 2013A CLINICAL TRIAL OF THE INTRA-TUMOURAL CONCENTRATION AND ACTIVITY OF NILOTINIB IN INTRA-CUTANEOUS SCHWANNOMAS - PHNT NilotinibNF2A CLINICAL TRIAL OF THE INTRA-TUMOURAL CONCENTRATION AND ACTIVITY OF NILOTINIB IN INTRA-CUTANEOUS SCHWANNOMAS - PHNT NilotinibNF2In vivo investigation of the intra-tumoural concentration and activity of nilotinib in cutaneous schwannomas (CS) in patienst with Neurofibromatosis 2Trade Name: Tasigna
Pharmaceutical Form: Capsule
INN or Proposed INN: NILOTINIB
CAS Number: 641571-10-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Plymouth Hospital NHS TrustAuthorisedFemale: yes
Male: yes
United Kingdom
65NCT01412892April 201119 February 2015Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform NeurofibromasA Single Arm, Multicenter Phase II a Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas That Cannot be Removed by SurgeryNeurofibromatosis Type 1;Plexiform Neurofibroma;NeurofibromatosesDrug: RAD001: EverolimusAssistance Publique - Hôpitaux de ParisNovartisNot recruiting18 Years60 YearsBoth30Phase 2France
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
66EUCTR2010-019759-23-GB12/01/201112 May 2014Evaluation of [11C]-methionine positron emission computerised tomography (PET CT) in diagnosing neurofibromatosis 1(NF1) - malignant peripheral nerve sheath tumours (MPNST) - Methionine PETEvaluation of [11C]-methionine positron emission computerised tomography (PET CT) in diagnosing neurofibromatosis 1(NF1) - malignant peripheral nerve sheath tumours (MPNST) - Methionine PETThe diagnosis of malignant peripheral nerve sheath tumours in patients with neurofibromatosis 1
MedDRA version: 12 Level: LLT Classification code 10029268 Term: Neurofibromatosis 1 associated malignant peripheral nerve sheath tumour
Product Name: [11C]-methionine
Pharmaceutical Form: Intravenous infusion
INN or Proposed INN: 2-amino-4-(methyl[11C]sulfanyl)butanoic acid
CAS Number: 63-68-3
Current Sponsor code: [11C]-L-methionine (MET)
Other descriptive name: Methionine
Concentration unit: mCi/µg millicurie(s)/microgram
Concentration type: up to
Concentration number: 100-
Guys' and St. Thomas' NHS Foundation TrustNot RecruitingFemale: yes
Male: yes
United Kingdom
67NCT01275586January 201116 December 2017Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasPilot Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasNeurofibromatosis;NF1;NeurofibromasDrug: TasignaIndiana UniversityNovartisNot recruiting18 YearsN/AAll6Early Phase 1United States
68EUCTR2011-002228-42-FR2 October 2017N/AN/A - AFINF2MedDRA version: 14.0 Level: PT Classification code 10029268 Term: Neurofibromatosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Cancer [C04]Trade Name: Afinitor
Product Name: Afinitor
Pharmaceutical Form: Tablet
INN or Proposed INN: Evérolimus
Other descriptive name: RAD001
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)Not RecruitingFemale: yes
Male: yes
Phase 2France
69NCT01207687October 201016 December 2017Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)Phase 2 Study of Bevacizumab in Children and Adults With Neurofibromatosis Type 2 and Symptomatic Vestibular SchwannomaVestibular Schwannoma;Neurofibromatosis Type 2Biological: bevacizumab;Other: laboratory biomarker analysis;Procedure: quality-of-life assessmentNational Cancer Institute (NCI)Not recruiting12 YearsN/AAll14Phase 2United States
70NCT01125046July 201011 June 2018Bevacizumab in Treating Patients With Recurrent or Progressive MeningiomasPhase II Trial of Bevacizumab in Patients With Recurrent or Progressive MeningiomasAcoustic Schwannoma;Adult Anaplastic Meningioma;Adult Ependymoma;Adult Grade I Meningioma;Adult Grade II Meningioma;Adult Meningeal Hemangiopericytoma;Adult Papillary Meningioma;Neurofibromatosis Type 1;Neurofibromatosis Type 2;Recurrent Adult Brain TumorBiological: bevacizumabNorthwestern UniversityNot recruiting18 YearsN/AAll50Phase 2United States
No.TrialIDDate_
enrollement
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PhaseCountries
71NCT01089101April 19, 201028 October 2019Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade GliomaA Phase 1 and Phase II and Re-Treatment Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade GliomaLow Grade Glioma;Recurrent Childhood Pilocytic Astrocytoma;Recurrent Neurofibromatosis Type 1;Recurrent Visual Pathway Glioma;Refractory Neurofibromatosis Type 1;Refractory Visual Pathway GliomaOther: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: SelumetinibNational Cancer Institute (NCI)Recruiting3 Years21 YearsAll180Phase 1/Phase 2United States
72EUCTR2009-016922-15-ES04/02/201019 March 2012ENSAYO 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 PATIENTSENSAYO 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 PATIENTSTratamiento de los neurofibromas plexiformes de alto riesgo no abordables quirúrgicamente en pacientes con neurofibromatosis tipo ITrade Name: GLIVEC 100 mg comprimidos recubiertos con película
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IMATINIB
Other descriptive name: IMATINIB
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Trade Name: GLIVEC 400 mg comprimidos recubiertos con película
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IMATINIB
Other descriptive name: IMATINIB
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 400-
HOSPITAL SANT JOAN DE DÉUAuthorisedFemale: yes
Male: yes
Spain
73NCT01031901December 200919 February 2015Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;NeurofibromaDrug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycinThe University of Texas Health Science Center, HoustonSociety for Pediatric DermatologyNot recruiting13 YearsN/ABoth52Phase 1United States
74EUCTR2009-010965-22-NL29/09/200927 January 2014The effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trialThe effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trialNeurofibromatosis 1Product Name: SIMVASTATIN
Product Code: SIMVASTATIN
Pharmaceutical Form: Capsule*
INN or Proposed INN: SIMVASTATIN
CAS Number: 79902639
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 10-40
Pharmaceutical form of the placebo: Capsule*
Route of administration of the placebo: Oral use
Erasmus MC - Department of PediatricsNot RecruitingFemale: yes
Male: yes
106Netherlands
75NCT00352599September 200928 November 2016Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)Neurofibromatosis 1Drug: Lovastatin;Drug: placebo pillUniversity of California, Los AngelesNot recruiting10 Years50 YearsBoth44Phase 1United States
No.TrialIDDate_
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PhaseCountries
76NCT00973739September 200919 October 2017Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsPhase II Study of Lapatinib in Children and Adults With Neurofibromatosis Type 2(NF2) and NF2-related TumorsNeurofibromatosis 2;Vestibular SchwannomaDrug: LapatinibNew York University School of MedicineGlaxoSmithKlineNot recruiting4 Years80 YearsAll21Phase 2United States
77NCT00911248July 31, 200923 April 2019PTC299 for Treatment of Neurofibromatosis Type 2A Phase 2 Study to Assess the Efficacy, Safety, and Pharmacodynamic Activity of PTC299 in Patients With Neurofibromatosis Type 2Neurofibromatosis 2Drug: PTC299PTC TherapeuticsUnited States Department of DefenseNot recruiting18 YearsN/AAll11Phase 2United States
78NCT00853580July 200911 June 2018A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1Neurofibromatosis Type 1Drug: Lovastatin ™;Device: placeboUniversity of Alabama at BirminghamBoston Children’s Hospital;Children's Hospital of Philadelphia;Children's Research Institute;Children's Hospital Medical Center, Cincinnati;National Cancer Institute (NCI);University of Chicago;University of Utah;Washington University School of Medicine;Sydney Children's Hospitals Network;University of Texas Southwestern Medical CenterNot recruiting8 Years15 YearsAll146Phase 2United States;Australia
79NCT00863122June 200919 February 2015Concentration and Activity of Lapatinib in Vestibular SchwannomasExploration and Estimation of Intratumoral Concentration and Activity of Lapatinib in Vivo in Vestibular SchwannomasVestibular Schwannoma;NF2;Neurofibromatosis 2;Acoustic Neuroma;Auditory TumorDrug: lapatinibSidney Kimmel Comprehensive Cancer CenterThe Children's Tumor Foundation;GlaxoSmithKline;New York University;Ohio State University;House Research Institute;Washington University School of Medicine;Weill Medical College of Cornell University;Massachusetts General HospitalRecruiting18 YearsN/ABoth26Phase 0United States
80NCT00865644March 200916 December 2017Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1Neurofibromatosis Type 1;Cutaneous NeurofibromasDrug: Imiquimod 5% CreamMassachusetts General HospitalNot recruiting18 YearsN/AAll11Phase 1United States
No.TrialIDDate_
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PhaseCountries
81NCT00846430October 20081 June 2015Medical Treatment of High-Risk NeurofibromasMedical Treatment of High-Risk Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical TherapiesNeurofibromatosis 1Drug: Peg-Interferon alpha-2b;Drug: Celecoxib (Celebrex);Drug: Temozolomide (temodar);Drug: Vincristine Sulfate (Oncovin)Spectrum Health HospitalsNot recruiting2 Years30 YearsBoth20Phase 2United States
82NCT00727233July 8, 200811 November 2019Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform NeurofibromasPhase I Trial of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib (BAY 43-9006, Nexavar) in Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform NeurofibromasNeurofibromatosis Type I;Plexiform NeurofibromaDrug: Nexavar (BAY 43-9006) (Sorafenib);Drug: Toxicity, Pharmacokinetics;Drug: Pharmacodynamics;Drug: Radiographic Evaluation;Drug: QOL assessment, Neuropsychological;Drug: Bony ToxicityNational Cancer Institute (NCI)Not recruiting3 Years18 YearsAll9Phase 1United States
83NCT00634270April 200816 December 2017A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasA Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasNeurofibromatosis Type 1Drug: SirolimusUniversity of Alabama at BirminghamBoston Children’s Hospital;Children's Hospital of Philadelphia;Children's Research Institute;Children's Hospital Medical Center, Cincinnati;National Cancer Institute (NCI);University of Chicago;University of Utah;Washington University School of MedicineNot recruiting3 Years75 YearsAll58Phase 2United States
84NCT00652990March 200819 February 2015Sirolimus to Treat Plexiform Neurofibromas in Patients With Neurofibromatosis Type IA Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasNeurofibromatosis Type 1;Plexiform Neurofibromas;Paraspinal Plexiform NeurofibromasDrug: SirolimusUniversity of Alabama at BirminghamNational Cancer Institute (NCI)Not recruiting3 YearsN/ABoth18Phase 2United States
85NCT00657202March 200821 July 2016Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1Neurofibromatosis Type 1;Cutaneous NeurofibromasDrug: RanibizumabMassachusetts General HospitalNot recruiting18 YearsN/ABoth11Phase 0United States
No.TrialIDDate_
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PhaseCountries
86NCT00589784October 200719 October 2017Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable MeningiomaPhase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable MeningiomaCNS Cancer;Meningioma;Intracranial Hemangiopericytoma;Hemangioblastoma;NeurofibromatosisDrug: SunitinibMemorial Sloan Kettering Cancer CenterDana-Farber Cancer Institute;University of Virginia;University of Pittsburgh;PfizerNot recruiting18 YearsN/AAll50Phase 2United States
87NCT00901849May 200719 February 2015Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1)Low-grade GliomasDrug: Tarceva and RapamycinRoger PackerChildren's Research InstituteNot recruitingN/A21 YearsBoth21Phase 1United States
88NCT00352495June 200619 February 2015Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade GliomaA Phase I Study of Vinblastine in Combination With Carboplatin for Children With Newly Diagnosed and Recurrent Low-Grade GliomasBrain and Central Nervous System Tumors;Neurofibromatosis Type 1Drug: carboplatin;Drug: vinblastine sulfateChildren's Oncology GroupNational Cancer Institute (NCI)Not recruitingN/A21 YearsBoth26Phase 1United States;Canada
89NCT00326872May 200616 December 2017AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the SpineA Phase II Study of AZD2171 in Adult Patients With Neurofibromatosis Type 1 and Extensive Plexiform and Paraspinal NeurofibromasNeurofibromatosis Type 1;Plexiform Neurofibroma;Spinal Cord NeurofibromaDrug: Cediranib MaleateNational Cancer Institute (NCI)Not recruiting18 YearsN/AAll26Phase 2United States
90NCT01673009May 200619 October 2017Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasPhase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasNeurofibromatosisDrug: GleevecIndiana UniversityNot recruiting3 Years65 YearsAll36Phase 2United States
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91NCT00304083December 200524 September 2018Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath TumorsPhase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated High Grade Malignant Peripheral Nerve Sheath TumorsNeurofibromatosis Type 1;SarcomaBiological: filgrastim;Drug: doxorubicin hydrochloride;Drug: etoposide;Drug: ifosfamide;Procedure: conventional surgery;Radiation: radiation therapySarcoma Alliance for Research through CollaborationNational Cancer Institute (NCI)Not recruitingN/AN/AAll48Phase 2United States;Canada
92NCT00076102July 21, 200411 June 2018Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform NeurofibromasPhase II Trial of Pirfenidone in Children, Adolescents, and Young Adults With Neurofibromatosis Type 1 and Progressive Plexiform NeurofibromasNeurofibromatosis 1;Neurofibroma, PlexiformDrug: PirfenidoneNational Cancer Institute (NCI)Not recruiting3 Years21 YearsAll36Phase 2United States
93NCT00169611January 200419 February 2015NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by MethylphenidateComportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus PlaceboNeurofibromatosis Type 1Drug: methylphenidateHospices Civils de LyonNot recruiting7 Years12 YearsBoth80Phase 4France
94NCT00053937December 200211 May 2015Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform NeurofibromasPhase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform NeurofibromasNeurofibromatosis Type 1;Precancerous ConditionDrug: pirfenidoneNational Cancer Institute (NCI)Not recruiting3 Years21 YearsBothPhase 1United States
95NCT00060008April 200219 October 2017Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform NeurofibromaNovel Imaging Modalities For Plexiform NeurofibromasNeurofibromatosis Type 1;Precancerous ConditionRadiation: fludeoxyglucose F 18;Radiation: gadopentetate dimeglumineChildren's Hospital of PhiladelphiaNot recruitingN/A25 YearsAll18N/AUnited States
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96NCT00021541July 17, 200111 June 2018R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform NeurofibromasA Phase II Randomized, Cross-Over, Double-Blinded, Placebo-Controlled Trial of the Farnesyltransferase Inhibitor R115777 in Pediatric Patients With Neurofibromatosis Type I and Progressive Plexiform NeurofibromasNeurofibroma, Plexiform;Neurofibromatosis Type IDrug: tipifarnib;Other: placeboNational Cancer Institute (NCI)Not recruiting3 Years25 YearsAll62Phase 2United States;Germany
97NCT00030264February 200113 June 2016Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform NeurofibromasVinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II StudyNeurofibromatosis Type 1;Precancerous ConditionDrug: Methotrexate;Drug: VinblastineChildren's Hospital of PhiladelphiaNot recruitingN/A25 YearsBoth25Phase 2United States
98NCT00754780September 200019 February 2015Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type INeurofibromatosisDrug: PirfenidoneMayo ClinicNot recruiting18 Years70 YearsBoth24Phase 2

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