5 active not recruiting, 4 completed, 3 recruiting, 3 terminated. 10 Phase 2, 6 Phase 1, 1 Early Phase 1, 1 Not applicable, 1 Phase 3.
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 | 2026-10-23 |
| 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 |
| 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 |
| 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 Study of Avutometinib for People With Solid Tumor Cancers Avutometinib - Refractory Cancer, CNS Tumors | Phase 1 | Active not recruiting | Memorial Sloan Kettering Cancer Center | 3 | 2023-10-20 | 2026-06-15 |
| FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas Test group (Group A): FCN-159 8 mg, orally, once daily;, Control group (Group B): Placebo, orally, once daily; - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 3 | Active not recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 167 | 2023-06-20 | 2025-03-06 |
| Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1 Neurofibromatosis 1, NF1 | Enrolling by invitation | Ann & Robert H Lurie Children's Hospital of Chicago | 200 | 2022-03-04 | 2026-09-09 | |
| 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 |
| A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1) INFUSE Bone Graft (BMP-2), Control Group - NF1, Congenital Pseudarthrosis of Tibia | Not applicable | Terminated | University of Alabama at Birmingham | 5 | 2016-03-01 | 2021-10-27 |
| 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 |
| Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas Tasigna - Neurofibromatosis, NF1 | Early Phase 1 | Completed | Indiana University | 6 | 2011-01-01 | 2017-04-12 |
| Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I Neurofibromatosis Type 1, Von Recklinghausen Disease | Completed | National Human Genome Research Institute (NHGRI) | 645 | 2008-05-15 | 2019-12-16 | |
| Study of Plexiform Neurofibromas in Neurofibromatosis Type 1 Plexiform Neurofibromas, NF1 | Completed | National Cancer Institute (NCI) | 24 | 2001-05-18 | 2020-05-04 |
Neurofibroma, Plexiform (10) · Neurofibromatosis 1 (9) · Neurofibromatoses (4) · Neurofibroma (3) · Arachnoid Cysts (1) · Astrocytoma (1) · Brain Neoplasms (1) · Central Nervous System Neoplasms (1) · Dyslexia (1) · Ependymoma (1) · Learning Disabilities (1) · Melanoma (1) · Neoplasms (1) · Neuroblastoma (1) · Neurocutaneous melanosis (1)
Neoplasms by Histologic Type (15) · Neoplasms, Nerve Tissue (15) · Nervous System Diseases (15) · Neoplasms (14) · Nerve Sheath Neoplasms (14) · Neuromuscular Diseases (14) · Peripheral Nervous System Diseases (14) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Nervous System Neoplasms (13) · Genetic Diseases, Inborn (12) · Heredodegenerative Disorders, Nervous System (12) · Neoplastic Syndromes, Hereditary (12) · Neurocutaneous Syndromes (12) · Neurodegenerative Diseases (12) · Peripheral Nervous System Neoplasms (12)
AZD 6244 (2) · Control Groups (2) · mirdametinib (2) · Lovastatin (1) · cabozantinib (1) · nilotinib (1) · poly ICLC (1) · talimogene laherparepvec (1)
University of Alabama at Birmingham (4) · National Cancer Institute (NCI) (3) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (3) · Indiana University (2) · Johns Hopkins University (2) · Ann & Robert H Lurie Children's Hospital of Chicago (1) · Memorial Sloan Kettering Cancer Center (1) · National Human Genome Research Institute (NHGRI) (1) · Vanderbilt University (1)
United States (16) · China (3) · Australia (1) · Spain (1)
Brigitte C Widemann, M.D. (2) · Carlos Romo, MD (2) · Chie-Schin Shih, MD (2) · Barbara B Biesecker (1) · Bruce R. Korf, MD, PhD (1) · Carlos Prada, MD (1) · Juliette Southworth, BS, CCRP (1) · Laurie Cutting, PhD (1) · Marielle E Yohe, M.D. (1) · Melissa Markel, MD (1) · Sameer Farouk Sait, MD (1) · Sheryl L. Rimrodt-Frierson, MD (1) · Wenbin Li, 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), R33NS122094 (1), R61NS122094 (1)