117 completed, 34 recruiting, 24 active not recruiting, 23 unknown. 85 Phase 2, 41 Not applicable, 41 Phase 1, 10 Phase 3, 6 Early Phase 1.
Follow Neurofibromatoses: new trials by email Literature Review of Neurofibromatoses trials Research Report on Neurofibromatoses
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs) GentleMax Pro (with skin cooling), GentleMax Pro (without skin cooling) - Neurofibromatosis 1 (NF1), Neurofibromatosis Type I | Not applicable | Suspended | Massachusetts General Hospital | 10 | 2027-01-01 | 2026-06-04 |
| Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma mirdametinib with radiation, mirdametinib - Neurofibromatosis 1 (NF1) | Phase 2 | Not yet recruiting | University of Alabama at Birmingham | 55 | 2027-01-01 | 2026-10-05 |
| Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor avutometinib capsule + defactinib tablet - MPNST (Malignant Peripheral Nerve Sheath Tumor) | Phase 2 | Not yet recruiting | Girish Dhall, MD | 25 | 2027-01-01 | 2026-10-28 |
| Selumetinib for NF2-Related Schwannomatosis Selumetinib - Neurofibromatosis Type 2, NF2 | Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 20 | 2026-12-01 | 2026-10-28 |
| Cognitive Intervention for Children and Adolescents With NF1 Unstuck and On Target! - Neurofibromatosis 1 (NF1), Executive Function (Cognition) | Not applicable | Not yet recruiting | The Hospital for Sick Children | 60 | 2026-09-28 | 2026-10-13 |
| 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) Neurofibromatosis Type 1 (NF1) | Recruiting | AstraZeneca | 800 | 2026-09-28 | 2026-10-22 | |
| Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics Sturge-Weber Syndrome, Neurocutaneous Syndromes | Not yet recruiting | Assiut University | 100 | 2026-09-20 | 2026-09-28 | |
| How Can the Experience of Hospital Outpatient Departments be Improved? Qualitative data collection activities - Neurofibromatosis Type 1 (NF1), NF2-related Schwannomatosis | Not yet recruiting | King's College London | 30 | 2026-09-01 | 2026-09-14 | |
| Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas Selumetinib + ZEN-3694 ± Durvalumab - MPNST, NF1 | Phase 2 | Recruiting | University of Alabama at Birmingham | 41 | 2026-08-04 | 2026-08-24 |
| Gait in Young Children With NF1 Neurofibromatosis Type 1 | Not yet recruiting | NYU Langone Health | 56 | 2026-08-01 | 2026-05-08 | |
| Luvometinib in Combination With Serplulimab for NF2-Related Tumors Luvometinib, Serplulimab - NF2-related Schwannomatosis, NF2 | Phase 1 / Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 30 | 2026-07-31 | 2026-07-16 |
| Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2) Selumetinib 25mg/m2, Luvometinib, Serplulimab - Neurofibromatosis Type 2, Vestibular Schwannoma | Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 200 | 2026-07-18 | 2026-07-20 |
| Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II Trineumin - Neurofibromatosis Type II, NF2 | Phase 1 / Phase 2 | Not yet recruiting | PRG Science & Technology Co., Ltd. | 25 | 2026-07-01 | 2026-01-23 |
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas Cabozantinib Oral Tablet, Selumetinib Oral Capsule - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 | Recruiting | Girish Dhall, MD | 30 | 2026-06-09 | 2026-10-02 |
| Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST APG-115, Selumetinib - Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1) | Phase 1 / Phase 2 | Recruiting | AeRang Kim | 45 | 2026-06-01 | 2026-07-27 |
| iCanCope With NF: Innovating an Efficacious Digital Self-management and Transitional Care Program for Adolescents With Neurofibromatosis iCanCope-NF Youth app, iCanCope-NF Youth app + CM - Neurofibromatosis 1 | Not applicable | Not yet recruiting | The Hospital for Sick Children | 60 | 2026-03-01 | 2025-07-22 |
| Assessment of Patient Experience With Auto-Captioning Glasses in NF2-Related-Schwannomatosis Auto-captioning Glasses - NF2-related Schwannomatosis | Not applicable | Completed | Massachusetts General Hospital | 18 | 2026-02-19 | 2026-08-25 |
| 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) AlgometRx Nociometer(R) - Neurofibromatosis Type 1 | Not applicable | Recruiting | National Cancer Institute (NCI) | 70 | 2026-02-18 | 2026-10-16 |
| Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1 Imlygic - NF1, Neurofibromatosis | Phase 1 | Recruiting | Johns Hopkins University | 10 | 2026-02-01 | 2026-05-08 |
| Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy NF1 Educational Videos, Peer Navigator, Letters about NF1 Care - Neurofibromatosis 1 | Not applicable | Recruiting | Massachusetts General Hospital | 50 | 2025-12-28 | 2026-07-15 |
Showing 20 of 245. Sign in to see all of them.
Neurofibromatosis 1 (174) · Neurofibroma, Plexiform (48) · Neurofibromatosis 2 (47) · Neuroma, Acoustic (24) · Neurofibroma (22) · Neurofibrosarcoma (16) · Meningioma (13) · Ependymoma (7) · Glioma (7) · Tuberous Sclerosis (7) · Brain Neoplasms (6) · Neoplasms (6) · Optic Nerve Glioma (6) · Noonan Syndrome (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (245) · Neoplasms by Histologic Type (245) · Neoplastic Syndromes, Hereditary (245) · Neurodegenerative Diseases (245) · Heredodegenerative Disorders, Nervous System (244) · Neoplasms, Nerve Tissue (244) · Nervous System Diseases (244) · Genetic Diseases, Inborn (243) · Neurocutaneous Syndromes (243) · Nerve Sheath Neoplasms (242) · Neoplasms (239) · Neurofibroma (223) · Neuromuscular Diseases (186) · Peripheral Nervous System Diseases (183)
AZD 6244 (22) · Bevacizumab (6) · Everolimus (6) · Lovastatin (5) · Sirolimus (5) · mirdametinib (5) · trametinib (5) · Deoxycholic Acid (4) · Blood Specimen Collection (3) · Imatinib Mesylate (3) · Lasers, Solid-State (3) · Magnetic Resonance Imaging (3) · Magnetic Resonance Spectroscopy (3) · Surveys and Questionnaires (3) · pirfenidone (3)
National Cancer Institute (NCI) (34) · Massachusetts General Hospital (17) · AstraZeneca (12) · University of Alabama at Birmingham (10) · NYU Langone Health (6) · Assistance Publique - Hôpitaux de Paris (5) · Massachusetts Eye and Ear Infirmary (5) · Beijing Tiantan Hospital (4) · Children's Hospital Medical Center, Cincinnati (4) · Novartis Pharmaceuticals (4) · Guy's and St Thomas' NHS Foundation Trust (3) · Indiana University (3) · Johns Hopkins University (3) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (3) · Shriners Hospitals for Children (3)
United States (163) · China (17) · France (17) · United Kingdom (14) · Germany (10) · Canada (7) · Netherlands (7) · Spain (7) · Australia (6) · Belgium (6) · Italy (5) · Japan (5) · Russia (5) · Austria (3) · Brazil (3)
Brigitte C Widemann, M.D. (11) · Staci M Peron, Ph.D. (7) · Ana-Maria Vranceanu, PhD (5) · Girish Dhall, MD (4) · Jason R Fangusaro (3) · Matthias A Karajannis, MD, MS (3) · Pamela L Wolters, Ph.D. (3) · Pinan Liu (3) · Richard R Anderson, MD (3) · Scott Plotkin, MD, PhD (3) · Vanessa Merker, PhD (3) · AeRang Kim, MD, PhD (2) · Brigitte C. Widemann, MD (2) · Carlos Romo, MD (2)
PMID 28029918, PMID 32187457, PMID 15683544, PMID 17105749, PMID 21996156, PMID 22353803, PMID 26851632, PMID 28230061, PMID 28409229, PMID 31010905, PMID 31582003, PMID 33354735, PMID 33863389, PMID 34541874, PMID 35007270, PMID 38908005, PMID 21764821, PMID 22934754, PMID 23099009, PMID 24321536, PMID 24500418, PMID 24753394, PMID 25205361, PMID 26783591, PMID 26943915, PMID 26976425, PMID 27956565, PMID 29718344, PMID 30284154, PMID 30339775, PMID 30355560, PMID 31127422, PMID 31151904, PMID 31881853, PMID 32152628, PMID 32421171, PMID 32562630, PMID 33395032, PMID 33442015, PMID 33470897, PMID 33507822, PMID 33507832, PMID 33536190, PMID 33641042, PMID 33728588, PMID 33817379, PMID 34091540, PMID 34409538, PMID 34570301, PMID 34667843
U10CA180886 (3), UM1CA081457 (2), 2016 (1), 2018 (1), 2023 (1), K23NS146694 (1), N01CM17104 (1), N01CM62205 (1), P30CA006973 (1), P30CA013148 (1), P30CA015083 (1), R01HD089474 (1), R01NS049096 (1), R01NS050509 (1), R33NS122094 (1)