34. 神経線維腫症
[臨床試験数:120,薬物数:182(DrugBank:72),標的遺伝子数:84,標的パスウェイ数:194

Searched query = "Neurofibromatosis", "NF1", "von Recklinghausen disease", "NF2"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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agemax
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PhaseCountries
1NCT04544007
(ClinicalTrials.gov)
February 15, 20212/9/2020A Phase II Trial of Poly-ICLC for Low-Grade GliomasA Phase II Trial of Poly-ICLC for Progressive, Previously Treated Low-Grade Gliomas in Children and Young Adults With Neurofibromatosis Type 1NF1;Low-grade GliomaDrug: Poly ICLCUniversity of Alabama at BirminghamChildren's Healthcare of Atlanta;Children's Hospital Los AngelesNot yet recruitingN/A22 YearsAll20Phase 2United States
2NCT04590235
(ClinicalTrials.gov)
November 12, 202028/7/2020A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)Neurofibromatosis 1;Neurofibroma PlexiformDrug: SelumetinibAstraZenecaMerck Sharp & Dohme Corp.Not yet recruiting3 Years99 YearsAll32Phase 1China
3NCT04481048
(ClinicalTrials.gov)
October 1, 202015/5/2020Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1Antioxidant Therapy With N-acetylcysteine for Motor Behavior and/or Learning in Children With Neurofibromatosis Type 1Neurofibromatosis 1Drug: N-Acetyl cysteine;Other: PlaceboChildren's Hospital Medical Center, CincinnatiUnited States Department of DefenseNot yet recruiting8 Years16 YearsAll58Phase 2NULL
4ChiCTR2000036770
2020-10-012020-08-25Establishment of prediction model of selumetinib therapy in the treatment of plexiform neurofibroma based on exosomes test from liquid biopsyEstablishment of prediction model of selumetinib therapy in the treatment of plexiform neurofibroma based on exosomes test from liquid biopsy Plexiform Neurofibromatosis type 1Gold Standard:Clinical outcomes;Index test:Prediction model of selumetinib therapy in the treatment of plexiform neurofibroma based on exosomes test from liquid biopsy;Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of MedicineNULLPending265BothTarget condition:32;Difficult condition:0China
5JPRN-JapicCTI-205422
01/9/202020/08/2020A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Japanese Paediatric Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable and Symptomatic Plexiform Neurofibromas (PN)A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Japanese Paediatric Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable and Symptomatic Plexiform Neurofibromas (PN) Neurofibromatosis Type 1 and Inoperable and Symptomatic Plexiform NeurofibromaIntervention name : Selumetinib
INN of the intervention : -
Dosage And administration of the intervention : Selumetinib 25 mg/m2 BID
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -
AstraZeneca KKNULLrecruiting318BOTH9Phase 1Japan, North America
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
6NCT04495127
(ClinicalTrials.gov)
August 31, 202029/7/2020Selumetinib Paediatric NF1 Japan StudyA Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Japanese Paediatric Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable and Symptomatic Plexiform Neurofibromas (PN)Neurofibromatosis Type 1Drug: SelumetinibAstraZenecaNULLRecruiting3 Years18 YearsAll12Phase 1Japan
7NCT04435665
(ClinicalTrials.gov)
August 21, 20208/6/2020NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2a Study to Determine Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of NFX-179 Gel in Subjects With Cutaneous NeurofibromasNeurofibromatosis 1;Cutaneous NeurofibromaDrug: NFX-179 Gel;Drug: Vehicle GelNFlection Therapeutics, Inc.NULLRecruiting18 YearsN/AAll48Phase 2United States
8NCT04461886
(ClinicalTrials.gov)
July 8, 20203/7/2020A Long-term Study of NPC-12G Gel in Neurofibromatosis Type IA Long-term Study of NPC-12G Gel in Neurofibromatosis Type INeurofibromatosisDrug: NPC-12G gelNobelpharmaNULLRecruiting3 YearsN/AAll100Phase 3Japan
9NCT04374305
(ClinicalTrials.gov)
June 20, 202030/4/2020Innovative Trial for Understanding the Impact of Targeted Therapies in NF2Innovative Trial for Understanding the Impact of Targeted Therapies in NF2 (INTUITT-NF2)Neurofibromatosis Type 2;Vestibular Schwannoma;Non-vestibular Schwannoma;Meningioma;EpendymomaDrug: BrigatinibScott R. Plotkin, MD, PhDTakeda;The Children's Tumor FoundationRecruiting12 YearsN/AAll80Phase 2United States
10NCT04283669
(ClinicalTrials.gov)
February 18, 202022/2/2020Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular SchwannomasOpen-label, Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular SchwannomasNeurofibromatosis 2;Progressive Vestibular Schwannoma (VS)Drug: CrizotinibUniversity of Alabama at BirminghamMemorial Sloan Kettering Cancer CenterRecruiting6 YearsN/AAll19Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
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PhaseCountries
11NCT04166409
(ClinicalTrials.gov)
January 3, 202015/11/2019A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade GliomaA Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)Astrocytoma;Low Grade GliomaDrug: Carboplatin;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Selumetinib Sulfate;Drug: Vincristine SulfateNational Cancer Institute (NCI)NULLRecruiting2 Years21 YearsAll200Phase 3United States;Canada
12NCT04201457
(ClinicalTrials.gov)
November 20, 201912/12/2019A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF AberrationPhase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-associated Recurrent or Progressive Gliomas in Children and Young AdultsLow Grade Glioma (LGG) of Brain With BRAF Aberration;High Grade Glioma (HGG) of the Brain With BRAF Aberration;Low Grade Glioma of Brain With Neurofibromatosis Type 1Drug: Dabrafenib;Drug: Trametinib;Drug: HydroxychloroquinePediatric Brain Tumor ConsortiumNULLRecruiting1 Year30 YearsAll75Phase 1;Phase 2United States
13NCT03975829
(ClinicalTrials.gov)
November 4, 201930/5/2019Pediatric 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 PharmaceuticalsNULLRecruiting1 YearN/AAll250Phase 4United States;France;Spain;United Kingdom
14EUCTR2019-001317-16-NL
(EUCTR)
30/10/201916/10/2019The TRAIN study: TRAmetinib In Neurofibromatosis type 1 related symptomatic plexiform neurofibromas The TRAIN study: TRAmetinib In Neurofibromatosis type 1 related symptomatic plexiform neurofibromas - TRAIN study: Trametinib in NF1-PNF Adult patients (age >17 years) with (mosaic) NF1 with inoperable symptomatic plexiform neurofibromas
MedDRA version: 20.0;Level: PT;Classification code 10029267;Term: Neurofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
ErasmusMCNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
30Phase 2Netherlands
15JPRN-JapicCTI-194999
15/10/201914/10/2019BeatNF2 trialA Randomized Double-blind Multicenter trial to Assess the Efficacy and Safety of Bevacizumab for Neurofibromatosis Type 2 Neurofibromatosis 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, Takafumi Mitsuhara, Takashi Tamiya, Takeo Goro, Shigeru Yamaguchi, Hirofumi Nakatomi, Soichi Oyapending1864BOTH60Phase 2Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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size
PhaseCountries
16NCT03871257
(ClinicalTrials.gov)
October 4, 201911/3/2019A 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 Sulfate;Drug: Vincristine SulfateNational Cancer Institute (NCI)NULLRecruiting2 Years21 YearsAll290Phase 3United States;Canada
17NCT03433183
(ClinicalTrials.gov)
October 2, 201929/1/2018SARC031: 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
18NCT03326388
(ClinicalTrials.gov)
September 26, 20198/9/2017Intermittent 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
19NCT03962543
(ClinicalTrials.gov)
September 6, 201912/4/2019MEK 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: Mirdametinib (PD-0325901) oral capsule or dispersible tabletSpringWorks Therapeutics, Inc.NULLRecruiting2 YearsN/AAll100Phase 2United States
20NCT04085159
(ClinicalTrials.gov)
September 1, 20199/9/2019Immunotherapy 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 InstituteShenzhen Hospital of Southern Medical University;Shenzhen Children's HospitalRecruiting1 Year80 YearsAll100Phase 1;Phase 2China
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
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size
PhaseCountries
21NCT03741101
(ClinicalTrials.gov)
June 10, 20195/11/2018Treatment 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
22NCT03826940
(ClinicalTrials.gov)
February 19, 201923/1/2019From 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 CoimbraNULLRecruiting16 Years65 YearsAll30N/APortugal
23NCT04481035
(ClinicalTrials.gov)
January 15, 201914/4/2020Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1Neurofibromatosis 1Drug: N-acetylcysteine (NAC);Other: PlaceboChildren's Hospital Medical Center, CincinnatiNULLActive, not recruiting8 Years16 YearsAll5Phase 2United States
24EUCTR2017-002635-41-GB
(EUCTR)
26/09/201819/06/2019 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 PATHWAY GLIOMAA 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 glaiom - Intermittent Selumetinib dosing Phase I/II study Childhood NF1 tumours Neurofibromatosis type 1 associated plexiform neurofibromas Neurofibromatosis type 1 associated progressive or relapsed optic pathway glioma
MedDRA version: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10065866;Term: Plexiform neurofibroma;System Organ Class: 100000004864
MedDRA version: 20.0;Classification code 10030935;Term: Optic nerve glioma;System Organ Class: 100000004864 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Great Ormond Street HospitalNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
38Phase 1;Phase 2United Kingdom
25EUCTR2018-001846-32-SE
(EUCTR)
18/09/201831/07/2018Treatment 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 - plexifpc NF1-related plexiform neurofibroma;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Mekinist
Product Name: Mekinist
VO Barnmedicin, Skånes University HospitalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 2Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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agemin
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PhaseCountries
26NCT03649165
(ClinicalTrials.gov)
September 5, 201824/8/2018A 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: AcetaminophenAstraZenecaNULLCompleted18 Years45 YearsMale24Phase 1United States
27NCT03688568
(ClinicalTrials.gov)
September 1, 201811/9/2018Study of Imatinib in Children With Neurofibromatosis and Airway TumorsPhase 2 Study of Imatinib in Children With Neurofibromatosis and Airway TumorsNeurofibroma, PlexiformDrug: Imatinib MesylateIndiana UniversityNULLRecruiting6 Months12 YearsAll20Phase 2United States
28NCT03079999
(ClinicalTrials.gov)
June 11, 20186/3/2017Study 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
29ChiCTR1800016040
2018-05-092018-05-08Clinical efficacy of Bevacizumab in the treatment of Type II NeurofibromatosisClinical efficacy of Bevacizumab in the treatment of Type II Neurofibromatosis Type II NeurofibromatosisCase series:Bevacizumab therapy;Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University, School of MedicineNULLRecruiting6BothCase series:30;China
30NCT03513757
(ClinicalTrials.gov)
March 4, 20184/2/2018Dexmedetomidine 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: Dexmedetomidine;Drug: Glycopyrrolate;Drug: Lidocaine 1% Injectable Solution;Drug: Nitrous Oxide;Drug: SevofluraneMedical College of WisconsinNULLCompleted12 Months60 MonthsAll40Phase 4United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
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PhaseCountries
31EUCTR2013-003405-26-DK
(EUCTR)
22/02/201824/04/2017Treating 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) - Treating learning disabilities in Neurofibromatosis 1 using lamotrigine Neurofibromatosis type 1
MedDRA version: 20.0;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
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
INN or Proposed INN: lamotrigine
Other descriptive name: LAMOTRIGINE
Erasmus MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Spain;Belgium;Denmark;Netherlands
32NCT03232892
(ClinicalTrials.gov)
February 13, 201814/7/2017Trametinib 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.Terminated18 YearsN/AAll1Phase 2United States
33EUCTR2016-005022-10-DE
(EUCTR)
13/02/201809/11/2017Improvement of synaptic plasticity and cognitive function in RAS pathway disordersImprovement of synaptic plasticity and cognitive function in RAS pathway disorders Noonan 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;Classification code 10029268;Term: Neurofibromatosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Not possible to specify
Product Name: Lovastatin
INN or Proposed INN: Lovastatin
Other descriptive name: LOVASTATIN
Product Name: Lamotrigin beta
INN or Proposed INN: Lamotrigin
Technische Universität München, Fakultät für MedizinNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 2Germany
34NCT02700230
(ClinicalTrials.gov)
December 8, 201725/2/2016Vaccine 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
35NCT03231306
(ClinicalTrials.gov)
November 28, 201721/7/2017Phase 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
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PhaseCountries
36NCT03259633
(ClinicalTrials.gov)
October 30, 201721/8/2017An 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: SelumetinibAstraZenecaNULLApproved for marketing2 Years130 YearsAllUnited States
37EUCTR2013-003405-26-ES
(EUCTR)
16/10/201731/07/2017Treating 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: 20.0;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000012614;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Lamotrigine dispers
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Trade Name: Lamotrigine dispers
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Trade Name: Lamotrigine dispers
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Trade Name: Lamotrigine dispers
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Trade Name: Lamotrigine dispers
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Trade Name: Lamotrigine dispers
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
Erasmus MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Belgium;Spain;Netherlands
38NCT03190915
(ClinicalTrials.gov)
October 6, 201716/6/2017Trametinib 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 1Drug: TrametinibNational Cancer Institute (NCI)Children's Oncology GroupRecruiting1 Month21 YearsAll24Phase 2United States
39NCT01968590
(ClinicalTrials.gov)
August 16, 20177/10/2013Vitamin 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 Development Command;Universitätsklinikum Hamburg-Eppendorf;University of British Columbia;Children's Hospital Medical Center, CincinnatiRecruiting25 Years40 YearsAll320Phase 2United States;Canada;Germany
40NCT03095248
(ClinicalTrials.gov)
May 8, 20178/3/2017Trial 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41NCT03105258
(ClinicalTrials.gov)
May 1, 201723/3/2017Selumetinib 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 yet recruiting18 YearsN/AAll24Phase 2United States
42NCT02839720
(ClinicalTrials.gov)
April 11, 201720/7/2016Selumetinib 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)NULLRecruiting18 YearsN/AAll24Phase 2United States
43NCT03109301
(ClinicalTrials.gov)
April 7, 201711/4/2017Mitogen 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)NULLWithdrawn3 Years99 YearsAll0Phase 2United States
44NCT03090971
(ClinicalTrials.gov)
February 15, 20178/3/2017Use 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 ÓrgãosNULLCompleted18 YearsN/AAll7Phase 2Brazil
45NCT02964884
(ClinicalTrials.gov)
November 20169/11/2016Interventions 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46ChiCTR-DDD-16009551
2016-10-282016-10-23Comparative study of Sanger sequencing and specific panel high-throughput sequencing for detection of NF1 pathogenic mutations in Han ChineseComparative study of Sanger sequencing and specific panel high-throughput sequencing for detection of NF1 pathogenic mutations in Han Chinese neurofibromatosis;M95400/1Gold Standard:;Index test:;Shanghai 9th People's Hospital Affiliated to Shanghai Jiaotong University School of MedicineNULLRecruiting1045BothTarget condition:0;Difficult condition:0China
47NCT02831257
(ClinicalTrials.gov)
August 31, 20167/7/2016AZD2014 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 DefenseActive, not recruiting18 YearsN/AAll18Phase 2United States
48NCT02728388
(ClinicalTrials.gov)
August 201625/3/2016Photodynamic Therapy for Benign Dermal Neurofibromas- Phase IITopical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase IINEUROFIBROMATOSIS 1Drug: aminolevulinic acidHarry T Whelan, MDNULLRecruiting14 Years30 YearsAll30Phase 2United States
49NCT02934256
(ClinicalTrials.gov)
July 201613/10/2016Icotinib 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
50JPRN-UMIN000021030
2016/03/1622/02/2016A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 Neurofibromatosis 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 HospitalNULLComplete: follow-up complete16years-old70years-oldMale and Female18Phase 2Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51NCT02718131
(ClinicalTrials.gov)
March 20167/3/2016A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)A Study of INFUSE Bone Graft (Recombinant Human Bone Morphogenetic Protein-2/Absorbable Collagen Sponge) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis 1 (NF1)NF1;Congenital Pseudarthrosis of TibiaDevice: INFUSE Bone Graft (BMP-2);Procedure: Control GroupUniversity of Alabama at BirminghamThe Children's Tumor Foundation;MedtronicActive, not recruiting2 Years18 YearsAll54N/AUnited States;Australia
52NCT02407405
(ClinicalTrials.gov)
January 7, 20162/4/2015MEK 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 (NF1);Plexiform Neurofibromas (PN)Drug: SelumetinibNational Cancer Institute (NCI)NULLRecruiting18 YearsN/AAll60Phase 2United States
53NCT02644512
(ClinicalTrials.gov)
December 201531/12/2015MEK 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)NULLRecruiting18 Years99 YearsBoth50Phase 2United States
54NCT02282917
(ClinicalTrials.gov)
September 201531/10/2014Exploratory 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 HospitalActive, not recruiting18 YearsN/AAll5Early Phase 1United States
55NCT02415153
(ClinicalTrials.gov)
July 14, 201513/4/2015Pomalidomide 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 Childhood Brain Stem Glioma;Recurrent Childhood Visual Pathway Glioma;Recurrent Primary Central Nervous System Neoplasm;Refractory Primary Central Nervous System NeoplasmOther: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: PomalidomideNational Cancer Institute (NCI)NULLActive, not recruiting3 Years20 YearsAll29Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56NCT01345136
(ClinicalTrials.gov)
July 1, 201521/4/2011Study 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 PharmaceuticalsActive, not recruiting16 Years65 YearsAll4Phase 2United States
57EUCTR2013-003405-26-BE
(EUCTR)
30/04/201530/03/2015Treating 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: 17.1;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 dispersErasmus MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Belgium;Netherlands
58NCT02390752
(ClinicalTrials.gov)
April 29, 201517/3/2015PLX3397 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)NULLRecruiting3 Years35 YearsAll81Phase 1;Phase 2United States
59NCT02332902
(ClinicalTrials.gov)
February 201512/12/2014Everolimus 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 FoundationCompleted18 YearsN/AAll24Phase 2United States
60NCT02211768
(ClinicalTrials.gov)
December 8, 20146/8/2014Transformation 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)NULLCompleted10 YearsN/AAll10Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
61NCT02256124
(ClinicalTrials.gov)
October 201417/9/2014Effect 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 DeuTerminated12 Years18 YearsAll41Phase 2;Phase 3Belgium;Netherlands;Spain
62JPRN-UMIN000015081
2014/09/0807/09/2014Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 neurofibromatosis 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 UniversityNULLRecruiting20years-oldNot applicableMale and Female3Phase 2;Phase 3Japan
63EUCTR2013-003405-26-NL
(EUCTR)
03/09/201420/09/2013Treating 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
INN or Proposed INN: Lamotrigine
Other descriptive name: LAMOTRIGINE
INN or Proposed INN: lamotrigine
Other descriptive name: LAMOTRIGINE
Erasmus MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Netherlands
64NCT02096471
(ClinicalTrials.gov)
June 201412/3/2014MEK 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 BirminghamNULLCompleted16 YearsN/AAll19Phase 2United States
65NCT02101736
(ClinicalTrials.gov)
June 201414/3/2014Cabozantinib 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 BirminghamNULLRecruiting3 YearsN/AAll48Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
66NCT02177825
(ClinicalTrials.gov)
June 201425/6/2014Study 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 HospitalNULLTerminated2 Years21 YearsAll5Phase 2Canada
67NCT02129647
(ClinicalTrials.gov)
April 201429/4/2014Study 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 HealthNULLActive, not recruiting18 YearsN/AAll12Phase 2United States
68NCT02104323
(ClinicalTrials.gov)
January 201426/3/2014Endostatin Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsRecombinant Human Endostatin Injection Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors by Continuous Intravenous PumpingVestibular Schwannoma;Neurofibromatosis Type 2Drug: EndostatinBeijing Tiantan HospitalShandong Simcere-Medgenn Bio-pharmaceutical Co., LtdCompleted16 Years30 YearsAll20Phase 2China
69EUCTR2012-003005-10-DK
(EUCTR)
06/06/201308/05/2013LOW-GRADE GLIOMAPHASE I-II STUDY OF VINBLASTINE IN COMBINATION WITH NILOTINIB IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH REFRACTORY OR RECURRENT LOW-GRADE GLIOMA - Vinilo 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 gliomas
MedDRA version: 20.0;Level: PT;Classification code 10065443;Term: Malignant glioma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0;Classification code 10038111;Term: Recurrent cancer;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
Other descriptive name: NILOTINIB HYDROCHLORIDE MONOHYDRATE
Trade Name: Tasigna 150mg
Product Name: Nilotinib
INN or Proposed INN: NILOTINIB
Trade Name: Tasigna 200mg
Product Name: Nilotinib
INN or Proposed INN: NILOTINIB
Product Name: Vinblastine
Other descriptive name: VINBLASTINE SULPHATE
Trade Name: Tasigna 50 mg
Product Name: Nilotinib
INN or Proposed INN: NILOTINIB
Gustave RoussyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
160Phase 2France;Spain;Denmark;Netherlands;Switzerland
70NCT01880749
(ClinicalTrials.gov)
June 201311/6/2013Exploring the Activity of RAD001 in Vestibular Schwannomas and MeningiomasExploring the Activity of RAD001 in Vestibular Schwannomas and MeningiomasNeurofibromatosis Type 2;Vestibular Schwannomas;MeningiomasDrug: RAD001NYU Langone HealthNULLCompleted18 YearsN/AAll5Early Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
71NCT01767792
(ClinicalTrials.gov)
May 15, 20137/1/2013Phase 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.Active, not recruiting6 YearsN/AAll22Phase 2United States
72EUCTR2012-005742-38-GB
(EUCTR)
02/05/201305/04/2013Early 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
INN or Proposed INN: Simvastatin
Central Manchester University Hospitals NHS Foundation TrustNULLNot RecruitingFemale: yes
Male: yes
0Phase 2United Kingdom
73NCT02584413
(ClinicalTrials.gov)
April 16, 201320/10/2015Hypotonia 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 HospitalTerminated1 Year7 YearsAll29N/AUnited States
74NCT01661283
(ClinicalTrials.gov)
September 20127/8/2012SARC016: 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 DefenseCompleted18 YearsN/AAll25Phase 2United States
75NCT01365468
(ClinicalTrials.gov)
April 201227/5/2011Efficacy 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 PharmaceuticalsNULLTerminated6 YearsN/AAll9Phase 2Israel
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76NCT01553149
(ClinicalTrials.gov)
March 19, 201210/3/2012Low-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)NULLActive, not recruitingN/A21 YearsAll75Phase 2United States;Australia;Canada;New Zealand
77NCT01552434
(ClinicalTrials.gov)
March 16, 20127/3/2012Bevacizumab 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
78NCT01402817
(ClinicalTrials.gov)
March 201225/7/2011Study 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;PfizerTerminated3 Years65 YearsAll19Phase 2United States
79NCT01140360
(ClinicalTrials.gov)
February 20128/6/2010Pilot 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 RobertsonNULLCompleted3 Years65 YearsAll21Phase 1;Phase 2United States
80NCT01490476
(ClinicalTrials.gov)
January 201230/11/2011Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) PatientsA Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular SchwannomaNeurofibromatosis 2Drug: RAD001Assistance Publique - Hôpitaux de ParisNULLCompleted15 YearsN/AAll10Phase 2France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81NCT01682811
(ClinicalTrials.gov)
November 20115/9/2012Phase 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, MDNULLCompleted18 Years90 YearsAll10Phase 1United States
82NCT01419639
(ClinicalTrials.gov)
October 201117/8/2011Phase 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 FoundationCompleted3 YearsN/AAll10Phase 2United States
83NCT01362803
(ClinicalTrials.gov)
September 21, 201127/5/2011AZD6244 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)NULLActive, not recruiting2 Years18 YearsAll99Phase 1;Phase 2United States
84EUCTR2011-001789-16-GB
(EUCTR)
26/08/201117/05/2011Investigation 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 NF2 Neurofibromatosis, 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
Plymouth Hospital NHS TrustNULLNot Recruiting Female: yes
Male: yes
14Phase 4United Kingdom
85EUCTR2010-023508-28-GB
(EUCTR)
14/04/201130/11/2010A 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 NilotinibNF2 In vivo investigation of the intra-tumoural concentration and activity of nilotinib in cutaneous schwannomas (CS) in patienst with Neurofibromatosis 2Plymouth Hospital NHS TrustNULLNot Recruiting Female: yes
Male: yes
5Phase 4United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
86NCT01412892
(ClinicalTrials.gov)
April 20118/8/2011Use 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 ParisNovartisCompleted18 Years60 YearsBoth30Phase 2France
87EUCTR2010-019759-23-GB
(EUCTR)
12/01/201113/12/2010Evaluation 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 PET The 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
INN or Proposed INN: 2-amino-4-(methyl[11C]sulfanyl)butanoic acid
Other descriptive name: Methionine
Guys' and St. Thomas' NHS Foundation TrustNULLNot RecruitingFemale: yes
Male: yes
United Kingdom
88NCT01275586
(ClinicalTrials.gov)
January 201111/1/2011Study 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 UniversityNovartisCompleted18 YearsN/AAll6Early Phase 1United States
89NCT01207687
(ClinicalTrials.gov)
October 20101/9/2010Bevacizumab 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)NULLCompleted12 YearsN/AAll14Phase 2United States
90NCT01125046
(ClinicalTrials.gov)
July 20107/5/2010Bevacizumab 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 UniversityNULLUnknown status18 YearsN/AAll50Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
91NCT01089101
(ClinicalTrials.gov)
April 19, 201017/3/2010Selumetinib 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)NULLRecruiting3 Years21 YearsAll220Phase 1;Phase 2United States
92EUCTR2009-016922-15-ES
(EUCTR)
04/02/201019/11/2009ENSAYO 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 PATIENTS Tratamiento de los neurofibromas plexiformes de alto riesgo no abordables quirúrgicamente en pacientes con neurofibromatosis tipo ITrade Name: GLIVEC 100 mg comprimidos recubiertos con película
INN or Proposed INN: IMATINIB
Other descriptive name: IMATINIB
Trade Name: GLIVEC 400 mg comprimidos recubiertos con película
INN or Proposed INN: IMATINIB
Other descriptive name: IMATINIB
HOSPITAL SANT JOAN DE DÉUNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Spain
93NCT01031901
(ClinicalTrials.gov)
December 200910/12/2009Topical 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 DermatologyCompleted13 YearsN/ABoth52Phase 1United States
94EUCTR2009-010965-22-NL
(EUCTR)
29/09/200921/09/2009The 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 trial Neurofibromatosis 1Product Name: SIMVASTATIN
Product Code: SIMVASTATIN
INN or Proposed INN: SIMVASTATIN
Erasmus MC - Department of PediatricsNULLNot RecruitingFemale: yes
Male: yes
106Netherlands
95NCT00973739
(ClinicalTrials.gov)
September 20093/9/2009Lapatinib 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 MedicineGlaxoSmithKlineCompleted4 Years80 YearsAll21Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
96NCT00352599
(ClinicalTrials.gov)
September 200913/7/2006Trial 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 AngelesNULLCompleted10 Years50 YearsBoth44Phase 1United States
97NCT00911248
(ClinicalTrials.gov)
July 31, 200928/5/2009PTC299 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 DefenseTerminated18 YearsN/AAll11Phase 2United States
98NCT00853580
(ClinicalTrials.gov)
July 200923/2/2009A 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 National 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 CenterCompleted8 Years15 YearsAll146Phase 2United States;Australia
99NCT00863122
(ClinicalTrials.gov)
June 200913/3/2009Concentration 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 Center at Johns HopkinsThe 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 HospitalCompleted18 YearsN/AAll26Early Phase 1United States
100NCT00865644
(ClinicalTrials.gov)
March 200917/3/2009Topical 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 HospitalNULLCompleted18 YearsN/AAll11Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
101NCT00846430
(ClinicalTrials.gov)
October 200816/1/2009Medical 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 HospitalsNULLActive, not recruiting2 Years30 YearsBoth20Phase 2United States
102NCT00727233
(ClinicalTrials.gov)
July 8, 200831/7/2008Sorafenib 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)NULLCompleted3 Years18 YearsAll9Phase 1United States
103NCT00634270
(ClinicalTrials.gov)
April 200820/2/2008A 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 MedicineCompleted3 Years75 YearsAll58Phase 2United States
104NCT00652990
(ClinicalTrials.gov)
March 20083/4/2008Sirolimus 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)Active, not recruiting3 YearsN/ABoth18Phase 2United States
105NCT00657202
(ClinicalTrials.gov)
March 200827/3/2008Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1Neurofibromatosis Type 1;Cutaneous NeurofibromasDrug: RanibizumabMassachusetts General HospitalNULLCompleted18 YearsN/ABoth11Phase 0United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
106NCT00589784
(ClinicalTrials.gov)
October 200726/12/2007Phase 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;PfizerCompleted18 YearsN/AAll50Phase 2United States
107NCT00901849
(ClinicalTrials.gov)
May 200713/5/2009Tarceva/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 InstituteCompletedN/A21 YearsBoth21Phase 1United States
108NCT00352495
(ClinicalTrials.gov)
June 200613/7/2006Vinblastine 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)CompletedN/A21 YearsBoth26Phase 1United States;Canada
109NCT01673009
(ClinicalTrials.gov)
May 200622/8/2012Phase 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 UniversityNULLCompleted3 Years65 YearsAll36Phase 2United States
110NCT00326872
(ClinicalTrials.gov)
May 200616/5/2006AZD2171 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)NULLTerminated18 YearsN/AAll26Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
111NCT00304083
(ClinicalTrials.gov)
December 200515/3/2006Combination 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)CompletedN/AN/AAll48Phase 2United States;Canada
112NCT00076102
(ClinicalTrials.gov)
July 21, 200413/1/2004Pirfenidone 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)NULLCompleted3 Years21 YearsAll36Phase 2United States
113NCT00169611
(ClinicalTrials.gov)
January 20049/9/2005NF1-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 LyonNULLCompleted7 Years12 YearsBoth80Phase 4France
114NCT00053937
(ClinicalTrials.gov)
December 20025/2/2003Pirfenidone 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)NULLCompleted3 Years21 YearsBothPhase 1United States
115NCT00060008
(ClinicalTrials.gov)
April 20026/5/2003Fludeoxyglucose 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 PhiladelphiaNULLTerminatedN/A25 YearsAll18N/AUnited States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
116NCT00021541
(ClinicalTrials.gov)
July 17, 200119/6/2006R115777 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)NULLCompleted3 Years25 YearsAll62Phase 2United States;Germany
117NCT00030264
(ClinicalTrials.gov)
February 200114/2/2002Combination 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 PhiladelphiaNULLCompletedN/A25 YearsAll23Phase 2United States
118NCT00754780
(ClinicalTrials.gov)
September 200016/9/2008Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type INeurofibromatosisDrug: PirfenidoneMayo ClinicNULLCompleted18 Years70 YearsBoth24Phase 2NULL
119EUCTR2016-001563-36-Outside-EU/EEA
(EUCTR)
12/05/2016Efficacy 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
Novartis PharmaceuticalsNULLNAFemale: yes
Male: yes
26Phase 4Israel
120EUCTR2011-002228-42-FR
(EUCTR)
29/08/2011N/AN/A - AFINF2
MedDRA 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
INN or Proposed INN: Evérolimus
Other descriptive name: RAD001
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)NULLNot RecruitingFemale: yes
Male: yes
Phase 2France