Neurofibromatosis 1: all 221 studies

Page 1 of 3. Open to participants first (44), then studies with results posted, then the rest; newest registration first within each.

• Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN)RecruitingNCT07888517
• Cognitive Intervention for Children and Adolescents With NF1Not yet recruitingNCT07869914Not applicable
• Neurocutaneous Syndromes in Children: Prevalence and Clinical CharacteristicsNot yet recruitingNCT07843823
• How Can the Experience of Hospital Outpatient Departments be Improved?Not yet recruitingNCT07818213
• Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath TumorNot yet recruitingNCT07743554Phase 2
• Gait in Young Children With NF1Not yet recruitingNCT07564622
• Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade GliomaNot yet recruitingNCT07521657Phase 2
• Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health LiteracyRecruitingNCT07233408Not applicable
• Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)RecruitingNCT07221331
• Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1RecruitingNCT07102394Phase 1
• iCanCope With NF: Innovating an Efficacious Digital Self-management and Transitional Care Program for Adolescents With NeurofibromatosisNot yet recruitingNCT07077408Not applicable
• Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1Not yet recruitingNCT07024394Phase 1/2
• PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform NeurofibromasRecruitingNCT06961565Phase 1
• Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative StudyRecruitingNCT06880991
• Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNSTRecruitingNCT06735820Phase 1/2
• The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate CapsuleNot yet recruitingNCT06621082Phase 2
• Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate CapsuleNot yet recruitingNCT06620354Phase 2
• A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1RecruitingNCT06541847Phase 2
• Genetic Bases of Neuroendocrine Neoplasms in Mexican PatientsRecruitingNCT06523582
• Neurofibromatosis Type 1 Tumor Early Detection StudyEnrolling by invitationNCT06515860
• A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)RecruitingNCT06507748Not applicable
• Study of Cabozantinib With Selumetinib for Plexiform NeurofibromasRecruitingNCT06502171Phase 1
• A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)RecruitingNCT06379230
• Real-World Treatment Study of Koselugo (Selumetinib)RecruitingNCT06360406
• Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)Enrolling by invitationNCT06300502Phase 1
• Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)RecruitingNCT06222203
• Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1RecruitingNCT06188741Phase 2
• FARD (RaDiCo Cohort) (RaDiCo-FARD)RecruitingNCT05954416
• A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic LeukemiaRecruitingNCT05849662Phase 1/2
• MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme MalignanciesRecruitingNCT05735717Phase 2
• 89Zr-Bevacizumab PET/CT Imaging in NF2 PatientsRecruitingNCT05685836
• Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled TrialRecruitingNCT05361811Not applicable
• HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasRecruitingNCT05331105Phase 2
• Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for SarcomasRecruitingNCT05253131Phase 2
• Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1Enrolling by invitationNCT05238909
• Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1RecruitingNCT05199376Not applicable
• Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- RetestRecruitingNCT05186870
• Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial IntelligenceRecruitingNCT04763109Not applicable
• Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical NeurofibromasRecruitingNCT04750928Phase 1/2
• RASopathy BiorepositoryRecruitingNCT04395495
• The Neurofibromatosis-associated Tumor BiobankNot yet recruitingNCT03359304
• Familial Investigations of Childhood Cancer PredispositionRecruitingNCT03050268
• Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)RecruitingNCT02544022
• The NF Registry Sponsored by the Children's Tumor FoundationRecruitingNCT01885767
• Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1CompletedNCT05912400Results posted
• Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PNActive, not recruitingNCT05309668Phase 1/2Results posted
• HIFU Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and EfficacyCompletedNCT05119582Not applicableResults posted
• NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)CompletedNCT05005845Phase 2Results posted
• Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform NeurofibromasActive, not recruitingNCT04924608Phase 3Results posted
• Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical TrialCompletedNCT04561765Not applicableResults posted
• Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1CompletedNCT04481048Phase 2Results posted
• Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1CompletedNCT04481035Phase 2Results posted
• NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)CompletedNCT04435665Phase 2Results posted
• MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasActive, not recruitingNCT03962543Phase 2Results posted
• Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment TrialsCompletedNCT03531814Not applicableResults posted
• SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath TumorsCompletedNCT03433183Phase 2Results posted
• Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform NeurofibromasCompletedNCT03231306Phase 2Results posted
• Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic LeukemiaActive, not recruitingNCT03190915Phase 2Results posted
• Trial of Selumetinib in Patients With Neurofibromatosis Type II Related TumorsTerminatedNCT03095248Phase 2Results posted
• Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous NeurofibromaCompletedNCT02839720Phase 2Results posted
• AZD2014 In NF2 Patients With Progressive or Symptomatic MeningiomasCompletedNCT02831257Phase 2Results posted
• A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)TerminatedNCT02718131Not applicableResults posted
• Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic PainCompletedNCT02471339Phase 3Results posted
• Feasibility and Clinically Application of Magnetic Resonance FingerprintingTerminatedNCT02387840Not applicableResults posted
• Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232TCompletedNCT02332902Phase 2Results posted
• Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular SchwannomasCompletedNCT02129647Phase 2Results posted
• Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and AdultsCompletedNCT02101736Phase 2Results posted
• MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1CompletedNCT02096471Phase 2Results posted
• Phase 2 Study of Bevacizumab in Children and Young Adults With NF 2 and Progressive Vestibular SchwannomasCompletedNCT01767792Phase 2Results posted
• Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)CompletedNCT01682811Phase 1Results posted
• Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasCompletedNCT01673009Phase 2Results posted
• Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway GliomaCompletedNCT01553149Phase 2Results posted
• Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2CompletedNCT01419639Phase 2Results posted
• Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform NeurofibromasTerminatedNCT01402817Phase 2Results posted
• Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)TerminatedNCT01365468Phase 2Results posted
• Sorafenib in Children and Young Adults With Recurrent or Progressive Low-Grade AstrocytomasTerminatedNCT01338857Phase 2Results posted
• Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasCompletedNCT01275586Early Phase 1Results posted
• Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)CompletedNCT01207687Phase 2Results posted
• Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform NeurofibromasCompletedNCT01140360Phase 1/2Results posted
• Bevacizumab in Treating Patients With Recurrent or Progressive MeningiomasCompletedNCT01125046Phase 2Results posted
• Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsCompletedNCT00973739Phase 2Results posted
• A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1CompletedNCT00853580Phase 2Results posted
• Medical Treatment of "High-Risk" NeurofibromasCompletedNCT00846430Phase 2Results posted
• A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasCompletedNCT00634270Phase 2Results posted
• Neurobiology and Treatment of Reading Disability in NF-1CompletedNCT00624234Not applicableResults posted
• Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable MeningiomaCompletedNCT00589784Phase 2Results posted
• AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the SpineTerminatedNCT00326872Phase 2Results posted
• Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath TumorsCompletedNCT00304083Phase 2Results posted
• Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform NeurofibromasCompletedNCT00076102Phase 2Results posted
• Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform NeurofibromaTerminatedNCT00060008Not applicableResults posted
• Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform NeurofibromasCompletedNCT00030264Phase 2Results posted
• R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform NeurofibromasCompletedNCT00021541Phase 2Results posted
• Precision Medicine for Neurocutaneous Syndromes in Western ChinaCompletedNCT07327164
• Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs)SuspendedNCT06934928Not applicable
• A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1CompletedNCT06262113Not applicable
• Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous NeurofibromasCompletedNCT06132165Phase 1
• Dosing and Tolerability of Deoxycholic Acid Vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous NeurofibromasCompletedNCT06120036Phase 1
• FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform NeurofibromasActive, not recruitingNCT05913037Phase 3
• Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled StudyWithdrawnNCT05825365Phase 2
• Mindful Parenting - A Single Case Experimental Design StudyStatus unknownNCT05779527Not applicable

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