| Not Yet Recruiting | Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas NCT06502171 | Girish Dhall, MD | Phase 1 |
| Not Yet Recruiting | iCanCope With NF: Innovating an Efficacious Digital Self-management and Transitional Care Program for Adolesce NCT07077408 | The Hospital for Sick Children | N/A |
| Recruiting | Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health NCT07233408 | Massachusetts General Hospital | N/A |
| Recruiting | Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 NCT06188741 | University of Alabama at Birmingham | Phase 2 |
| Not Yet Recruiting | Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosi NCT07024394 | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | Phase 1 / Phase 2 |
| Enrolling By Invitation | Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibroma NCT06300502 | Massachusetts General Hospital | Phase 1 |
| Recruiting | A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis NCT06262113 | Massachusetts General Hospital | N/A |
| Recruiting | A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leu NCT05849662 | Therapeutic Advances in Childhood Leukemia Consortium | Phase 1 / Phase 2 |
| Recruiting | Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tu NCT06222203 | National Cancer Institute (NCI) | — |
| Not Yet Recruiting | Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule NCT06620354 | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | Phase 2 |
| Not Yet Recruiting | The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsu NCT06621082 | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | Phase 2 |
| Recruiting | Neurofibromatosis Type 1 Tumor Early Detection Study NCT06515860 | David Miller | — |
| Recruiting | Real-World Treatment Study of Koselugo (Selumetinib) NCT06360406 | AstraZeneca | — |
| Completed | Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 NCT06132165 | Massachusetts General Hospital | Phase 1 |
| Recruiting | Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility S NCT05361811 | National Cancer Institute (NCI) | N/A |
| Completed | Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1 NCT05912400 | University of Arkansas | — |
| Active Not Recruiting | FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibroma NCT05913037 | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | Phase 3 |
| Recruiting | MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies NCT05735717 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Completed | Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutan NCT06120036 | Massachusetts General Hospital | Phase 1 |
| Recruiting | Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients NCT06523582 | Universidad Nacional Autonoma de Mexico | — |
| Active Not Recruiting | NF-1, Nutraceutical Intervention NCT05363267 | Masonic Cancer Center, University of Minnesota | Phase 1 |
| Completed | Clinical Hypnosis and Home Blood Pressure Monitoring in Children With Neurofibromatosis Type 1 NCT05196854 | Murdoch Childrens Research Institute | N/A |
| Completed | Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosi NCT05377008 | Virginia Commonwealth University | N/A |
| Enrolling By Invitation | Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1 NCT05238909 | Ann & Robert H Lurie Children's Hospital of Chicago | — |
| Recruiting | Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neu NCT04750928 | National Cancer Institute (NCI) | Phase 1 / Phase 2 |
| Active Not Recruiting | Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas NCT04924608 | AstraZeneca | Phase 3 |
| Recruiting | HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas NCT05331105 | Shanghai Kechow Pharma, Inc. | Phase 2 |
| Completed | NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) NCT05005845 | NFlection Therapeutics, Inc. | Phase 2 |
| Active Not Recruiting | Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type NCT05677594 | Johns Hopkins University | — |
| Active Not Recruiting | Natural History Study of Cutaneous Neurofibromas in People With NF1 NCT05581511 | Johns Hopkins University | — |
| Completed | Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects NCT04879160 | National Cancer Institute (NCI) | — |
| Completed | Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1 NCT04941027 | Stanford University | — |
| Active Not Recruiting | Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and NCT04954001 | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | Phase 1 / Phase 2 |
| Completed | Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Appli NCT04561765 | Yale University | N/A |
| Completed | Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosi NCT04774289 | National Cancer Institute (NCI) | — |
| Unknown | Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1 NCT04860362 | University of Wisconsin, Milwaukee | N/A |
| Active Not Recruiting | A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoper NCT04590235 | AstraZeneca | Phase 1 |
| Active Not Recruiting | Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1 NCT04481048 | Children's Hospital Medical Center, Cincinnati | Phase 2 |
| Unknown | Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1 NCT04856514 | University of Wisconsin, Milwaukee | N/A |
| Completed | NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) NCT04435665 | NFlection Therapeutics, Inc. | Phase 2 |
| Completed | SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients Wit NCT03433183 | Sarcoma Alliance for Research through Collaboration | Phase 2 |
| Unknown | Comparison of Gastrointestinal Motility in Healthy Children and Children With Constipation NCT03981510 | University of Aarhus | N/A |
| Active Not Recruiting | Treatment of NF1-related Plexiform Neurofibroma With Trametinib NCT03741101 | Region Skane | Phase 2 |
| Completed | Mechanism of Action of Transcranial Direct Current Stimulation in Neurofibromatosis Type 1 NCT04991428 | University of Manchester | N/A |
| Completed | From Molecules to Cognition: Inhibitory Mechanisms in ASD and NF1 NCT03826940 | University of Coimbra | N/A |
| Completed | Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis T NCT04481035 | Children's Hospital Medical Center, Cincinnati | Phase 2 |
| Recruiting | A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE) NCT06379230 | Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University | — |
| Completed | Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatm NCT03531814 | National Cancer Institute (NCI) | N/A |
| Active Not Recruiting | Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1 NCT03820778 | Children's National Research Institute | N/A |
| Completed | Non-invasive Stimulation in Neurofibromatosis Type 1 NCT03310996 | University of Manchester | N/A |
| Completed | Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing NCT03406208 | Massachusetts General Hospital | N/A |
| Recruiting | RASopathy Biorepository NCT04395495 | Children's Hospital Medical Center, Cincinnati | — |
| Withdrawn | Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People Wi NCT03109301 | National Cancer Institute (NCI) | Phase 2 |
| Completed | Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1 NCT03090971 | Fundação Educacional Serra dos Órgãos | Phase 2 |
| Recruiting | Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II NCT02728388 | Donald Basel | Phase 2 |
| Completed | Study About Annoucement of the Diagnosis of Neurofibromatosis 1 in de Novo Forms NCT03298438 | University Hospital, Brest | — |
| Unknown | Analysis of Plasma for Diagnosis and Follow-up of Neurofibromatosis Type 1 NCT02680431 | Juha Peltonen | — |
| Recruiting | Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 NCT02544022 | National Cancer Institute (NCI) | — |
| Completed | Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T NCT02332902 | The University of Texas Health Science Center, Houston | Phase 2 |
| Completed | Internet Support Group for Parents of a Child With Neurofibromatosis Type 1 NCT02153931 | National Cancer Institute (NCI) | — |
| Completed | PET/MRI in CNS and Extra-CNS Tumors of Patients With Neurofibromatosis-1 (NF1) NCT01800032 | UNC Lineberger Comprehensive Cancer Center | — |
| Recruiting | Neurofibromatosis (NF) Registry Portal NCT01885767 | The Children's Tumor Foundation | — |
| Unknown | Modifying Genes in Neurofibromatosis 1 NCT01650142 | Assistance Publique - Hôpitaux de Paris | — |
| Active Not Recruiting | AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors NCT01362803 | National Cancer Institute (NCI) | Phase 1 / Phase 2 |
| Unknown | Whole Body Magnetic Resonance Imaging With Diffusion Weighted Imaging : Potential Role in Neurofibromatosis NCT01777451 | Universitaire Ziekenhuizen KU Leuven | — |
| Completed | Genetic Evaluation of NF1 and Scoliosis Patients NCT01776125 | University of Minnesota | — |
| Completed | Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1) NCT00352599 | University of California, Los Angeles | Phase 1 |
| Completed | Medical Treatment of "High-Risk" Neurofibromas NCT00846430 | Corewell Health West | Phase 2 |
| Completed | Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas NCT00076102 | National Cancer Institute (NCI) | Phase 2 |