3 active not recruiting, 2 not yet recruiting, 2 recruiting, 2 terminated. 10 Phase 2, 4 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Active not recruiting 3, Not yet recruiting 2, Recruiting 2, Terminated 2, Completed 1 |
| Phase 1 | 4 | Active not recruiting 2, Not yet recruiting 1, Recruiting 1 |
Follow NF1: new trials by email Literature Review of NF1 trials Research Report on NF1
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 |
|---|---|---|---|---|---|---|
| Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults Mirdametinib - LGCMN, Large and Giant Congenital Melanocytic Nevi | Phase 2 | Not yet recruiting | National Cancer Institute (NCI) | 15 | 2027-01-04 | 2026-10-29 |
| 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 |
| 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 |
| Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF). Mirdametinib - NF1, Cutaneous Neurofibroma | Phase 1 / Phase 2 | Recruiting | Johns Hopkins University | 24 | 2024-02-12 | 2026-04-13 |
| A Phase II Trial of Poly-ICLC for Low-Grade Gliomas Poly ICLC - NF1, Low-grade Glioma | Phase 2 | Terminated | University of Alabama at Birmingham | 3 | 2022-02-08 | 2025-03-21 |
| 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 |
| Interventions for Reading Disabilities in NF1 Lovastatin, reading tutoring intervention, Placebo Oral Tablet - Neurofibromatosis Type 1, Learning Disability | Phase 2 | Active not recruiting | Vanderbilt University | 120 | 2016-11-01 | 2026-07-01 |
| 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 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 |
| AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors AZD6244 - Neurofibromatosis 1, Neurofibromatosis Type 1 | Phase 1 / Phase 2 | Active not recruiting | National Cancer Institute (NCI) | 99 | 2011-09-21 | 2026-10-27 |
Neurofibroma, Plexiform (5) · Neurofibromatosis 1 (5) · Neurofibromatoses (2) · Arachnoid Cysts (1) · Astrocytoma (1) · Dyslexia (1) · Ependymoma (1) · Learning Disabilities (1) · Melanoma (1) · Neurocutaneous melanosis (1) · Neurofibrosarcoma (1) · Pain (1) · Sarcoma (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Neoplasms (8) · Neoplasms by Histologic Type (8) · Neoplasms, Nerve Tissue (8) · Nervous System Diseases (8) · Genetic Diseases, Inborn (7) · Heredodegenerative Disorders, Nervous System (7) · Neoplastic Syndromes, Hereditary (7) · Nerve Sheath Neoplasms (7) · Nervous System Neoplasms (7) · Neurocutaneous Syndromes (7) · Neurodegenerative Diseases (7) · Neurofibroma (7) · Neuromuscular Diseases (7) · Peripheral Nervous System Diseases (7)
AZD 6244 (2) · mirdametinib (2) · Lovastatin (1) · cabozantinib (1) · poly ICLC (1)
University of Alabama at Birmingham (3) · National Cancer Institute (NCI) (2) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (2) · Indiana University (1) · Johns Hopkins University (1) · Vanderbilt University (1)
United States (9) · China (2) · Spain (1)
Chie-Schin Shih, MD (2) · Brigitte C Widemann, M.D. (1) · Carlos Romo, MD (1) · Juliette Southworth, BS, CCRP (1) · Laurie Cutting, PhD (1) · Marielle E Yohe, M.D. (1) · Sheryl L. Rimrodt-Frierson, MD (1)
PMID 28029918, PMID 32187457, PMID 32562630, PMID 33442015, PMID 35467749, PMID 37115514, PMID 37400844, PMID 38869947, PMID 40709016, PMID 41139346, PMID 42540436
R01HD089474 (1)