National Institutes of Health: Neurofibromatosis 1 trials

The registry lists 37 Neurofibromatosis 1 studies with National Institutes of Health as lead sponsor. 6 are open to participants now and 9 have results posted.

Early Phase 1 1Phase 1 6Phase 2 12Phase 3 2No phase 19(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative StudyRecruitingNCT06880991
• 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
• Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)RecruitingNCT06222203
• Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled TrialRecruitingNCT05361811Not applicable
• Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical NeurofibromasRecruitingNCT04750928Phase 1/2
• Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)RecruitingNCT02544022
• Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment TrialsCompletedNCT03531814Not applicableResults posted
• Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic LeukemiaActive, not recruitingNCT03190915Phase 2Results posted
• Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous NeurofibromaCompletedNCT02839720Phase 2Results posted
• Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic PainCompletedNCT02471339Phase 3Results posted
• Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway GliomaCompletedNCT01553149Phase 2Results posted
• Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)CompletedNCT01207687Phase 2Results posted
• AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the SpineTerminatedNCT00326872Phase 2Results posted
• Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform NeurofibromasCompletedNCT00076102Phase 2Results posted
• R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform NeurofibromasCompletedNCT00021541Phase 2Results posted
• Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical TrialCompletedNCT04879160
• Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)CompletedNCT04774289
• A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade GliomaActive, not recruitingNCT03871257Phase 3
• Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)WithdrawnNCT03109301Phase 2
• Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System TumorsCompletedNCT02415153Phase 1
• MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasActive, not recruitingNCT02407405Phase 2
• Internet Support Group for Parents of a Child With Neurofibromatosis Type 1CompletedNCT02153931
• Relationship Stressors in Parents of Children With Cancer or Neurofibromatosis Type 1 (NF1)CompletedNCT01702922
• Validating Pain Scales in Children and Young AdultsCompletedNCT01639950
• Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic PainCompletedNCT01633008Early Phase 1
• Analysis of Data Collected From Individuals Administered Neurobehavioral AssessmentsCompletedNCT01445314
• AZD6244 Hydrogen Sulfate for Children With Nervous System TumorsActive, not recruitingNCT01362803Phase 1/2
• Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II PatientsCompletedNCT01222728
• Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade GliomaActive, not recruitingNCT01089101Phase 1/2
• Natural History Study of Patients With Neurofibromatosis Type IActive, not recruitingNCT00924196
• Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform NeurofibromasCompletedNCT00727233Phase 1
• Adaptation and Quality of Life Among Adults With Neurofibromatosis Type ICompletedNCT00684398
• Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1CompletedNCT00314119
• Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)CompletedNCT00111384
• Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform NeurofibromasCompletedNCT00053937Phase 1
• Eligibility Screening for a NCI Pediatric Oncology Branch Research StudyCompletedNCT00026780
• Study of Plexiform Neurofibromas in Neurofibromatosis Type 1CompletedNCT00006435