78 completed, 19 recruiting, 19 terminated, 16 active not recruiting. 157 Phase 2, 30 Phase 1, 9 Phase 3.
Follow Neurocutaneous Syndromes: new trials by email Literature Review of Neurocutaneous Syndromes trials Research Report on Neurocutaneous Syndromes
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 |
| Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1) AV078, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Not yet recruiting | Aeovian Pharmaceuticals, Inc. | 42 | 2026-09-01 | 2026-09-29 |
| 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 |
| Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) Antisense oligonucleotide targeting the ATM gene - Ataxia Telangiectasia | Phase 1 / Phase 2 | Recruiting | Timothy Yu | 10 | 2026-08-01 | 2026-05-28 |
| 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 |
| Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) SVG103 - Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC) | Phase 1 / Phase 2 | Recruiting | Sovargen | 12 | 2026-07-14 | 2026-08-10 |
| 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 |
| CAT-VHL Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-Hippel Lindau Disease investigating the role of CAIX as target in VHL disease and in VHL-/- tumors. - VHL - Von Hippel-Lindau Syndrome | Phase 2 | Recruiting | IRCCS Ospedale San Raffaele | 38 | 2026-02-25 | 2026-03-09 |
| An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg - Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer) | Phase 1 / Phase 2 | Recruiting | Beijing Tide Pharmaceutical Co., Ltd | 266 | 2025-12-03 | 2026-01-28 |
| 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 |
Showing 20 of 157. Sign in to see all of them.
Neurofibromatosis 1 (55) · Tuberous Sclerosis (35) · Neurofibroma, Plexiform (24) · Neurofibromatosis 2 (24) · von Hippel-Lindau Disease (18) · Neurofibromatoses (15) · Neuroma, Acoustic (15) · Epilepsy (11) · Ataxia Telangiectasia (10) · Meningioma (9) · Neurofibroma (8) · Neurofibrosarcoma (7) · Ependymoma (6) · Sturge-Weber Syndrome (6) · Hemangioblastoma (5)
Nervous System Diseases (156) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (153) · Genetic Diseases, Inborn (147) · Neoplasms (136) · Neoplastic Syndromes, Hereditary (121) · Neurodegenerative Diseases (121) · Heredodegenerative Disorders, Nervous System (120) · Neoplasms by Histologic Type (116) · Neoplasms, Nerve Tissue (91) · Nerve Sheath Neoplasms (83) · Neurofibroma (77) · Neurofibromatoses (70) · Neuromuscular Diseases (66) · Peripheral Nervous System Diseases (64)
Everolimus (18) · AZD 6244 (17) · Sirolimus (16) · Bevacizumab (4) · Sunitinib (4) · mirdametinib (4) · trametinib (4) · Imatinib Mesylate (3) · Lovastatin (3) · belzutifan (3) · Acetylcysteine (2) · Aspirin (2) · Cannabidiol (2) · Inhalation (2) · Lamotrigine (2)
National Cancer Institute (NCI) (15) · University of Alabama at Birmingham (10) · Children's Hospital Medical Center, Cincinnati (9) · Beijing Tiantan Hospital (4) · NYU Langone Health (4) · Novartis Pharmaceuticals (4) · Anne Comi, MD (3) · M.D. Anderson Cancer Center (3) · National Eye Institute (NEI) (3) · Assistance Publique - Hôpitaux de Paris (2) · AstraZeneca (2) · Cardiff University (2) · Dana-Farber Cancer Institute (2) · Erasmus Medical Center (2) · Indiana University (2)
United States (106) · China (16) · Australia (11) · Spain (10) · United Kingdom (9) · Canada (8) · France (8) · Germany (7) · Italy (6) · Netherlands (6) · Israel (4) · Poland (4) · Belgium (3) · Japan (3) · Turkey (Türkiye) (3)
Brigitte C Widemann, M.D. (5) · Girish Dhall, MD (3) · Matthias A Karajannis, MD, MS (3) · AeRang Kim, MD, PhD (2) · Anne M Comi, MD (2) · Chie-Schin Shih, MD (2) · D. Bradley Welling, MD, PhD (2) · Dennis J Dlugos, MD (2) · Eric Jonasch, MD (2) · Fu Zhao (2) · John Bissler, MD (2) · Martina Bebin, MD, MPA (2) · Mary Kay Koenig, MD (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 34783716, PMID 16763896, PMID 16934683, PMID 18184959, PMID 19841031, PMID 21047224, PMID 21482669, PMID 21525172, PMID 21915260, PMID 22105611, PMID 22144182, PMID 22262746, PMID 22875082, PMID 22927201, PMID 23099009, PMID 23325902, PMID 23437092, PMID 23798472, PMID 24236946, PMID 24500418, PMID 24753394, PMID 25656498, PMID 26381530, PMID 26433381, PMID 26547562, PMID 26851119, PMID 26976425, PMID 27956565, PMID 28871440, PMID 28969699, PMID 29023494, PMID 29110179, PMID 29718344, PMID 29800048, PMID 30236511, PMID 30284154
2018 (4), 2023 (3), U10CA180886 (2), 2016 (1), 2025 (1), N01CM17104 (1), N01CM62205 (1), P30CA006516 (1), P30CA006973 (1), P30CA013148 (1), P30CA015083 (1), R01CA107164 (1), R01HD089474 (1), R21CA103486 (1), U01CA081457 (1)