219 completed, 70 recruiting, 51 unknown, 43 active not recruiting. 157 Phase 2, 72 Phase 1, 70 Not applicable, 36 Phase 3, 13 Phase 4.
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 |
|---|---|---|---|---|---|---|
| Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs) GentleMax Pro (with skin cooling), GentleMax Pro (without skin cooling) - Neurofibromatosis 1 (NF1), Neurofibromatosis Type I | Not applicable | Suspended | Massachusetts General Hospital | 10 | 2027-01-01 | 2026-06-04 |
| 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 |
| A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above. sirolimus topical gel 0.2% - Epidermal Nevi | Early Phase 1 | Not yet recruiting | Northwestern University | 14 | 2026-10-01 | 2026-09-10 |
| Cognitive Intervention for Children and Adolescents With NF1 Unstuck and On Target! - Neurofibromatosis 1 (NF1), Executive Function (Cognition) | Not applicable | Not yet recruiting | The Hospital for Sick Children | 60 | 2026-09-28 | 2026-10-13 |
| Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN) Neurofibromatosis Type 1 (NF1) | Recruiting | AstraZeneca | 800 | 2026-09-28 | 2026-10-22 | |
| Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics Sturge-Weber Syndrome, Neurocutaneous Syndromes | Not yet recruiting | Assiut University | 100 | 2026-09-20 | 2026-09-28 | |
| 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 |
| How Can the Experience of Hospital Outpatient Departments be Improved? Qualitative data collection activities - Neurofibromatosis Type 1 (NF1), NF2-related Schwannomatosis | Not yet recruiting | King's College London | 30 | 2026-09-01 | 2026-09-14 | |
| Biomarkers for Babies and Young Children With Ataxia Telangiectasia Ataxia Telangiectasia | Not yet recruiting | University of Nottingham | 56 | 2026-09-01 | 2026-07-16 | |
| 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 |
| Gait in Young Children With NF1 Neurofibromatosis Type 1 | Not yet recruiting | NYU Langone Health | 56 | 2026-08-01 | 2026-05-08 | |
| 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 |
| Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas Von Hippel-Lindau Disease, Central Nervous System Hemangioblastoma | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 25 | 2026-07-15 | 2026-07-15 | |
| 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 |
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas Cabozantinib Oral Tablet, Selumetinib Oral Capsule - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 | Recruiting | Girish Dhall, MD | 30 | 2026-06-09 | 2026-10-02 |
Showing 20 of 468. Sign in to see all of them.
Neurofibromatosis 1 (174) · Tuberous Sclerosis (114) · Neurofibromatoses (57) · von Hippel-Lindau Disease (55) · Neurofibroma, Plexiform (48) · Neurofibromatosis 2 (47) · Ataxia Telangiectasia (42) · Epilepsy (26) · Neuroma, Acoustic (24) · Neurofibroma (22) · Lymphangioleiomyomatosis (17) · Sturge-Weber Syndrome (17) · Neurofibrosarcoma (16) · Carcinoma, Renal Cell (15) · Meningioma (14)
Nervous System Diseases (465) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (458) · Genetic Diseases, Inborn (443) · Neoplasms (399) · Neoplastic Syndromes, Hereditary (359) · Neurodegenerative Diseases (358) · Heredodegenerative Disorders, Nervous System (357) · Neoplasms by Histologic Type (330) · Neoplasms, Nerve Tissue (261) · Nerve Sheath Neoplasms (242) · Neurofibroma (223) · Neuromuscular Diseases (196) · Peripheral Nervous System Diseases (192) · Congenital Abnormalities (190)
Everolimus (26) · AZD 6244 (22) · Sirolimus (21) · Blood Specimen Collection (8) · Cannabidiol (8) · Bevacizumab (7) · Lovastatin (5) · belzutifan (5) · mirdametinib (5) · trametinib (5) · Deoxycholic Acid (4) · Magnetic Resonance Imaging (4) · Magnetic Resonance Spectroscopy (4) · Ranibizumab (4) · Sunitinib (4)
National Cancer Institute (NCI) (39) · Massachusetts General Hospital (18) · AstraZeneca (12) · Novartis Pharmaceuticals (12) · University of Alabama at Birmingham (12) · Children's Hospital Medical Center, Cincinnati (11) · NYU Langone Health (8) · Assistance Publique - Hôpitaux de Paris (7) · M.D. Anderson Cancer Center (7) · National Eye Institute (NEI) (7) · Jazz Pharmaceuticals (6) · Johann Wolfgang Goethe University Hospital (6) · Johns Hopkins University (6) · University of California, Los Angeles (6) · Massachusetts Eye and Ear Infirmary (5)
United States (289) · United Kingdom (43) · France (39) · China (33) · Australia (28) · Italy (28) · Spain (28) · Germany (27) · Canada (22) · Netherlands (21) · Poland (17) · Belgium (15) · Israel (14) · Japan (14) · Russia (12)
Brigitte C Widemann, M.D. (11) · Staci M Peron, Ph.D. (7) · Ana-Maria Vranceanu, PhD (5) · Stefan Zielen, Prof. Dr. (5) · Girish Dhall, MD (4) · Anne M Comi, M.D. (3) · Darcy Krueger, MD, PhD (3) · Eric Jonasch, MD (3) · Jason R Fangusaro (3) · Joel Moss, M.D. (3) · Mary Kay Koenig, MD (3) · Matthias A Karajannis, MD, MS (3) · Naris Nilubol, M.D. (3)
PMID 28029918, PMID 29023494, PMID 32187457, PMID 34783716, PMID 15683544, PMID 17105749, PMID 21996156, PMID 22353803, PMID 26851632, PMID 28230061, PMID 28409229, PMID 30053159, PMID 30291925, PMID 31010905, PMID 31582003, PMID 31912454, PMID 33354735, PMID 33863389, PMID 34477998, PMID 34541874, PMID 34957550, PMID 35007270, PMID 38908005, PMID 38996393, PMID 40739200, PMID 16763896, PMID 16934683, PMID 17804777, PMID 18184959, PMID 19141364, PMID 19841031, PMID 21047224, PMID 21354816, PMID 21482669, PMID 21525172, PMID 21764821, PMID 21915260, PMID 22105611, PMID 22113127, PMID 22144182, PMID 22262746, PMID 22875082, PMID 22927201, PMID 22934754, PMID 23099009, PMID 23158522, PMID 23325902, PMID 23437092, PMID 23732715, PMID 23733802
2018 (6), 2023 (5), U10CA180886 (3), U54NS065705 (3), 2015 (2), 2025 (2), UM1CA081457 (2), 2008 (1), 2016 (1), K23NS146694 (1), N01CM17104 (1), N01CM62205 (1), P30CA006516 (1), P30CA006973 (1), P30CA013148 (1)