10 completed, 5 active not recruiting, 4 terminated, 3 recruiting. 28 Phase 2, 5 Phase 1.
Follow Neurofibroma, Plexiform: new trials by email Literature Review of Neurofibroma, Plexiform trials Research Report on Neurofibroma, Plexiform
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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Placebo-Controlled Study Selumetinib, Placebo - Neurofibromatosis Type 1, Plexiform Neurofibromas | Phase 2 | Withdrawn | AstraZeneca | 2023-08-31 | 2023-08-14 | |
| HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas HL-085 - Neurofibromatosis 1, Plexiform Neurofibromas | Phase 2 | Recruiting | Shanghai Kechow Pharma, Inc. | 70 | 2021-10-18 | 2023-05-31 |
| Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 FCN-159 - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Active not recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 160 | 2021-03-26 | 2024-11-12 |
| MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas Mirdametinib (PD-0325901) oral capsule or dispersible tablet - Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1) | Phase 2 | Active not recruiting | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | 114 | 2019-09-29 | 2026-10-06 |
| Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours Selumetinib - Neurofibromatosis Type 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Completed | Great Ormond Street Hospital for Children NHS Foundation Trust | 14 | 2019-09-26 | 2024-04-04 |
| Treatment of NF1-related Plexiform Neurofibroma With Trametinib Trametinib - Neurofibromatosis 1, Child | Phase 2 | Unknown | Region Skane | 15 | 2019-06-10 | 2024-04-11 |
| Study of Imatinib in Children With Neurofibromatosis and Airway Tumors Imatinib Mesylate - Neurofibroma, Plexiform | Phase 2 | Withdrawn | Indiana University | 2018-09-01 | 2021-02-12 | |
| Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway. Trametinib - Low-grade Glioma, Plexiform Neurofibroma | Phase 2 | Active not recruiting | St. Justine's Hospital | 114 | 2018-08-16 | 2026-04-24 |
| Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas Binimetinib - Neurofibromatosis Type 1, Plexiform Neurofibroma | Phase 2 | Completed | University of Alabama at Birmingham | 45 | 2017-11-28 | 2025-05-02 |
| Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST) Selumetinib (AZD6244 hyd sulfate) 50mg/dose, Selumetinib (AZD6244 hyd sulfate) 25mg/m2 - Neoplasms, Nerve Tissue, Neurofibromatosis 1 | Phase 2 | Withdrawn | National Cancer Institute (NCI) | 2017-04-07 | 2019-04-05 | |
| MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas Selumetinib - Neurofibromatosis 1 (NF1), Plexiform Neurofibromas (PN) | Phase 2 | Active not recruiting | National Cancer Institute (NCI) | 36 | 2016-01-07 | 2026-09-15 |
| Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations Trametinib, Dabrafenib - Cancer | Phase 1 / Phase 2 | Completed | Novartis Pharmaceuticals | 139 | 2015-01-15 | 2021-07-14 |
| Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults Cabozantinib - NF1, Neurofibromatosis | Phase 2 | Completed | University of Alabama at Birmingham | 45 | 2014-06-01 | 2023-05-24 |
| Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas Imatinib Mesylate - Plexiform Neurofibromas | Phase 2 | Terminated | St. Justine's Hospital | 5 | 2014-06-01 | 2019-04-08 |
| Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1) Everolimus (RAD001) - Plexiform Neurofibroma Associated With Neurofibromatosis Type 1 | Phase 2 | Terminated | Novartis Pharmaceuticals | 9 | 2012-04-01 | 2016-05-12 |
| Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas Sutent®/Sunitinib - Neurofibromatosis, NF1 | Phase 2 | Terminated | Indiana University | 19 | 2012-03-01 | 2018-03-14 |
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Neurofibromatosis 1 (21) · Neurofibromatoses (7) · Neurofibroma (3) · Brain Neoplasms (1) · Glioma (1) · Heredodegenerative Disorders, Nervous System (1) · Histiocytosis, Langerhans-Cell (1) · Neoplasms (1) · Neoplasms, Nerve Tissue (1) · Nerve Sheath Neoplasms (1) · Neuroblastoma (1) · Optic Nerve Glioma (1) · Pain (1) · Peripheral Nervous System Diseases (1)
Neoplasms by Histologic Type (28) · Nervous System Diseases (28) · Nervous System Neoplasms (28) · Neuromuscular Diseases (28) · Neoplasms (27) · Neoplasms, Nerve Tissue (27) · Nerve Sheath Neoplasms (27) · Peripheral Nervous System Diseases (27) · Peripheral Nervous System Neoplasms (27) · Neurofibroma (25) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Genetic Diseases, Inborn (24) · Neoplastic Syndromes, Hereditary (24) · Neurocutaneous Syndromes (24) · Neurodegenerative Diseases (24)
AZD 6244 (7) · trametinib (3) · Imatinib Mesylate (2) · pirfenidone (2) · Everolimus (1) · Methotrexate (1) · Vinblastine (1) · binimetinib (1) · cabozantinib (1) · cediranib (1) · dabrafenib (1) · mirdametinib (1) · peginterferon alfa-2b (1) · tipifarnib (1)
National Cancer Institute (NCI) (6) · University of Alabama at Birmingham (3) · Indiana University (2) · Novartis Pharmaceuticals (2) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (2) · St. Justine's Hospital (2) · Assistance Publique - Hôpitaux de Paris (1) · AstraZeneca (1) · Children's Hospital of Philadelphia (1) · Great Ormond Street Hospital for Children NHS Foundation Trust (1) · Healx Limited (1) · Mayo Clinic (1) · Region Skane (1) · Shanghai Kechow Pharma, Inc. (1) · SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany (1)
United States (16) · China (4) · Canada (3) · France (2) · United Kingdom (2) · Australia (1) · Germany (1) · Israel (1) · Spain (1) · Sweden (1)
Brigitte C Widemann, M.D. (4) · Chie-Schin Shih, MD (2) · Brigitte Widemann, M.D. (1) · Bruce Korf, MD, PhD (1) · Darren Hargrave, MB Bch (1) · Dusica Babovic-Vuksanovic (1) · Dusica Babovic-Vuksanovic, M.D. (1) · Hongqi Tian, Ph.D (1) · Jean B. Belasco, MD (1) · Jean Tersak, M.D. (1) · Kathryn Nevel (1) · Michael Fisher (1)
PMID 24500418, PMID 24753394, PMID 27510726, PMID 28029918, PMID 30339775, PMID 31881853, PMID 32187457, PMID 32562630, PMID 33442015, PMID 33536190, PMID 34331515, PMID 35007270, PMID 35467749, PMID 36375115, PMID 36884302, PMID 37115514, PMID 37400844, PMID 38869947, PMID 39116355, PMID 39514826, PMID 39762421, PMID 40709016, PMID 41139346, PMID 42540436
2018 (1), N01CM17104 (1), N01CM62205 (1), P30CA015083 (1)