80 completed, 26 recruiting, 21 active not recruiting, 18 unknown. 55 Phase 2, 33 Not applicable, 30 Phase 1, 6 Phase 3, 2 Phase 4.
Follow Neurofibromatosis 1: new trials by email Literature Review of Neurofibromatosis 1 trials Research Report on Neurofibromatosis 1
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 |
|---|---|---|---|---|---|---|
| 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 | 23 | 2027-01-01 | 2026-08-04 |
| 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 |
| 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 | |
| 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 |
| 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 |
| Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy) Neurofibromatosis Type 1 | Recruiting | AstraZeneca | 200 | 2025-11-19 | 2026-09-11 | |
| Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 Selumetinib - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 2 | Recruiting | University of Alabama at Birmingham | 200 | 2025-08-27 | 2026-05-29 |
| Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1 Luvometinib Tablets - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 65 | 2025-06-12 | 2025-06-17 |
| PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas PAS-004 Tablets - NF1 Mutation, Neurofibroma Plexiform | Phase 1 | Recruiting | Pasithea Therapeutics Corp. | 56 | 2025-05-30 | 2025-12-04 |
| Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study Arm 1 - Neurofibromatosis Type 1, Neurofibroma | Recruiting | National Cancer Institute (NCI) | 110 | 2025-04-29 | 2026-10-16 |
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Neurofibroma, Plexiform (43) · Neurofibromatoses (23) · Neurofibroma (19) · Neurofibrosarcoma (13) · Neurofibromatosis 2 (7) · Optic Nerve Glioma (6) · Brain Neoplasms (5) · Glioma (5) · Tuberous Sclerosis (5) · Astrocytoma (4) · Central Nervous System Neoplasms (4) · Dyslexia (4) · Noonan Syndrome (4) · Learning Disabilities (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (174) · Neoplasms by Histologic Type (174) · Neoplastic Syndromes, Hereditary (174) · Nervous System Diseases (174) · Neurodegenerative Diseases (174) · Neuromuscular Diseases (174) · Genetic Diseases, Inborn (173) · Heredodegenerative Disorders, Nervous System (173) · Neoplasms, Nerve Tissue (173) · Peripheral Nervous System Diseases (173) · Neoplasms (172) · Nerve Sheath Neoplasms (172) · Neurocutaneous Syndromes (172) · Neurofibroma (155) · Neurofibromatoses (151)
AZD 6244 (19) · Lovastatin (5) · trametinib (5) · Deoxycholic Acid (4) · Lasers, Solid-State (3) · mirdametinib (3) · Acetylcysteine (2) · Biopsy (2) · Blood Specimen Collection (2) · Carboplatin (2) · Everolimus (2) · Inhalation (2) · Lamotrigine (2) · Lasers (2) · Magnetic Resonance Imaging (2)
National Cancer Institute (NCI) (30) · AstraZeneca (11) · Massachusetts General Hospital (11) · University of Alabama at Birmingham (7) · Assistance Publique - Hôpitaux de Paris (3) · Children's Hospital Medical Center, Cincinnati (3) · Johns Hopkins University (3) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (3) · Shriners Hospitals for Children (3) · University Hospital, Toulouse (3) · Washington University School of Medicine (3) · Ann & Robert H Lurie Children's Hospital of Chicago (2) · Children's Hospital of Philadelphia (2) · Children's National Research Institute (2) · Corewell Health West (2)
United States (116) · France (14) · China (10) · United Kingdom (10) · Canada (7) · Spain (7) · Australia (6) · Belgium (6) · Germany (6) · Netherlands (5) · Italy (4) · Japan (4) · Russia (4) · Brazil (3) · Israel (3)
Brigitte C Widemann, M.D. (10) · Staci M Peron, Ph.D. (5) · Girish Dhall, MD (3) · Jason R Fangusaro (3) · Pamela L Wolters, Ph.D. (3) · Richard R Anderson, MD (3) · AeRang Kim, MD, PhD (2) · Ana-Maria Vranceanu, PhD (2) · Brigitte C. Widemann, MD (2) · Carlos Romo, MD (2) · Douglas R Stewart, M.D. (2) · Eric Legius, MD PhD (2) · Kimberly J Johnson, PhD (2) · Pierre Wolkenstein, MD, PhD (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 24321536, PMID 24500418, PMID 24753394, PMID 25205361, PMID 27956565, PMID 29718344, PMID 30284154, PMID 30339775, PMID 30355560, PMID 31151904, PMID 31881853, PMID 32152628, PMID 32421171, PMID 33395032, PMID 33507822, PMID 33507832, PMID 33536190, PMID 33641042, PMID 33728588, PMID 33817379, PMID 34091540, PMID 34409538, PMID 34570301, PMID 35064449, PMID 35170228, PMID 35394491, PMID 35410754, PMID 35467749, PMID 35925531, PMID 35970940, PMID 36651958, PMID 36996759, PMID 37115514
U10CA180886 (3), UM1CA081457 (2), 2016 (1), 2018 (1), 2023 (1), K23NS146694 (1), N01CM17104 (1), N01CM62205 (1), P30CA013148 (1), P30CA015083 (1), R01HD089474 (1), R01NS049096 (1), R01NS050509 (1), R33NS122094 (1), R61NS122094 (1)