37 completed, 13 terminated, 11 active not recruiting, 10 not yet recruiting. 85 Phase 2, 14 Phase 1, 4 Phase 3.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| Mirdametinib in Patients With Advanced NF1-mutant Melanoma Mirdametinib - Advanced Unresectable Melanoma, Metastatic Melanoma | Phase 2 | Recruiting | Kevin Kim, MD | 10 | 2025-10-13 | 2025-11-19 |
| 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 |
| A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1 HLX-1502 - Neurofibromatosis Type 1 | Phase 2 | Recruiting | Healx Limited | 25 | 2025-02-10 | 2026-07-29 |
| Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children Selumetinib - Plexiform Neurofibromas (PN) | Phase 2 | Not yet recruiting | West China Hospital | 50 | 2025-01-01 | 2025-01-08 |
| A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia Trametinib, Azacitidine, Fludarabine - Leukemia, Juvenile Myelomonocytic, JMML | Phase 1 / Phase 2 | Recruiting | Therapeutic Advances in Childhood Leukemia Consortium | 58 | 2024-10-11 | 2026-09-04 |
| The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule Selumetinib - Neurofibromatosis 1 | Phase 2 | Not yet recruiting | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | 19 | 2024-09-30 | 2024-10-01 |
| Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule Selumetinib - Neurofibromatosis 1 | Phase 2 | Not yet recruiting | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | 33 | 2024-09-30 | 2024-10-01 |
| Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study Selumetinib, Placebo - Neurofibromatosis Type 1, Plexiform Neurofibromas | Phase 2 | Withdrawn | AstraZeneca | 2023-08-31 | 2023-08-14 | |
| MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies Fludarabine, Busulfan, Melphalan - Hematologic Malignancy, Acute Leukemia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 70 | 2023-05-11 | 2026-04-06 |
| Doxycycline in Cutaneous Schwannoma (NF2) Doxycycline Injection [Doxy] - Neurofibromatosis Type 2 | Phase 1 / Phase 2 | Active not recruiting | Massachusetts Eye and Ear Infirmary | 19 | 2022-09-19 | 2026-10-16 |
| Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas REC-2282, Placebo - Neurofibromatosis Type 2 | Phase 2 / Phase 3 | Terminated | Recursion Pharmaceuticals Inc. | 25 | 2022-06-20 | 2026-08-17 |
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Neurofibromatosis 1 (55) · Neurofibroma, Plexiform (24) · Neurofibromatosis 2 (24) · Neuroma, Acoustic (15) · Meningioma (9) · Neurofibroma (8) · Neurofibrosarcoma (7) · Ependymoma (6) · Brain Neoplasms (3) · Glioma (3) · Leukemia, Myelomonocytic, Juvenile (3) · Optic Nerve Glioma (3) · Astrocytoma (2) · Nerve Sheath Neoplasms (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (85) · Neoplasms (85) · Neoplasms by Histologic Type (85) · Neoplastic Syndromes, Hereditary (85) · Nervous System Diseases (85) · Neurocutaneous Syndromes (85) · Neurodegenerative Diseases (85) · Genetic Diseases, Inborn (84) · Heredodegenerative Disorders, Nervous System (84) · Neoplasms, Nerve Tissue (84) · Nerve Sheath Neoplasms (83) · Neurofibroma (77) · Neuromuscular Diseases (62) · Peripheral Nervous System Diseases (60)
AZD 6244 (17) · Everolimus (5) · Bevacizumab (4) · mirdametinib (4) · trametinib (4) · Imatinib Mesylate (3) · Lovastatin (3) · Sirolimus (3) · Acetylcysteine (2) · Inhalation (2) · Lamotrigine (2) · fludarabine (2) · pirfenidone (2) · Aminolevulinic Acid (1) · Aspirin (1)
National Cancer Institute (NCI) (12) · University of Alabama at Birmingham (10) · Beijing Tiantan Hospital (4) · Children's Hospital Medical Center, Cincinnati (3) · NYU Langone Health (3) · Assistance Publique - Hôpitaux de Paris (2) · AstraZeneca (2) · Indiana University (2) · Massachusetts Eye and Ear Infirmary (2) · NFlection Therapeutics, Inc. (2) · Novartis Pharmaceuticals (2) · Sarcoma Alliance for Research through Collaboration (2) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (2) · Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University (2) · AeRang Kim (1)
United States (57) · China (10) · Germany (4) · Canada (3) · Spain (3) · Australia (2) · France (2) · Netherlands (2) · United Kingdom (2) · Austria (1) · Belgium (1) · Brazil (1) · Israel (1) · Italy (1) · Japan (1)
Brigitte C Widemann, M.D. (5) · Girish Dhall, MD (3) · Matthias A Karajannis, MD, MS (3) · AeRang Kim, MD, PhD (2) · Chie-Schin Shih, MD (2) · D. Bradley Welling, MD, PhD (2) · Fu Zhao (2) · Pinan Liu (2) · Scott Plotkin, MD (2) · Zhiquan Huang, doctoral (2) · Albert S Cornelius, MD (1) · Brian Weiss, MD (1) · Brigitte C. Widemann, MD (1)
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 23099009, PMID 24500418, PMID 24753394, PMID 26976425, PMID 27956565, PMID 29718344, PMID 30284154, PMID 30339775, PMID 31151904, PMID 31881853, PMID 32562630, PMID 33442015, PMID 33507822, PMID 33536190, PMID 33728588, PMID 33817379, PMID 35007270, PMID 35467749, PMID 36996759, PMID 37115514, PMID 37126770, PMID 37280688, PMID 37400844, PMID 38372904, PMID 38867349, PMID 38869947, PMID 38904277, PMID 38907342, PMID 39116355, PMID 39340758, PMID 39514826, PMID 39762421, PMID 40009403, PMID 40121838, PMID 40709016, PMID 41139346
U10CA180886 (2), 2016 (1), 2018 (1), N01CM17104 (1), N01CM62205 (1), P30CA006973 (1), P30CA013148 (1), P30CA015083 (1), R01HD089474 (1), U01CA081457 (1), U10CA098543 (1), U54CA196519 (1), UM1CA081457 (1)