15 completed, 9 terminated, 5 active not recruiting, 4 recruiting. 36 Phase 3, 9 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 36 | Completed 15, Terminated 9, Active not recruiting 5, Recruiting 4, Unknown 2, Withdrawn 1 |
| Phase 2 | 9 | Terminated 3, Completed 2, Unknown 2, Recruiting 1, Withdrawn 1 |
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 |
|---|---|---|---|---|---|---|
| Extension Study for Participants in Studies That Include Belzutifan (MK-6482-043/LITESPARK-043) Belzutifan, Nivolumab, Lenvatinib - Von Hippel-Lindau Disease, Malignant Neoplasms | Phase 3 | Recruiting | Merck Sharp & Dohme LLC | 450 | 2026-03-23 | 2026-10-21 |
| A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) N-Acetyl-L-Leucine, Placebo - Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T) | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2025-03-18 | 2025-07-03 |
| Open-Label Extension of EryDex Study IEDAT-04-2022 Dexamethasone sodium phosphate - Ataxia Telangiectasia | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 101 | 2024-12-11 | 2026-05-05 |
| Evaluate the Neurological Effects of EryDex on Subjects With A-T Dexamethasone sodium phosphate, Placebo - Ataxia Telangiectasia | Phase 3 | Completed | Quince Therapeutics S.p.A. | 105 | 2024-06-24 | 2026-04-30 |
| Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC) YCJ-01 Full Spectrum cannbis Extract, Placebo - Tuberous Sclerosis Complex (TSC) | Phase 3 | Recruiting | Oils4Cure | 84 | 2024-06-01 | 2026-02-11 |
| 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 |
| Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 3 | Recruiting | Katarzyna Kotulska | 200 | 2023-01-23 | 2026-10-22 |
| Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas REC-2282, Placebo - Neurofibromatosis Type 2 | Phase 2 / Phase 3 | Terminated | Recursion Pharmaceuticals Inc. | 25 | 2022-06-20 | 2026-08-17 |
| Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC NPC-12Y gel, NPC-12Y placebo gel - Tuberous Sclerosis Complex | Phase 3 | Completed | Nobelpharma | 43 | 2022-06-01 | 2024-11-22 |
| Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy Ganaxolone - Tuberous Sclerosis Complex | Phase 3 | Terminated | Marinus Pharmaceuticals | 117 | 2022-05-16 | 2025-10-01 |
| Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Ganaxolone, Placebo - Tuberous Sclerosis Complex | Phase 3 | Completed | Marinus Pharmaceuticals | 129 | 2022-04-01 | 2025-07-11 |
| Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas Selumetinib, Placebo - Neurofibromatosis 1, Plexiform Neurofibroma (PN) | Phase 3 | Active not recruiting | AstraZeneca | 145 | 2021-11-19 | 2026-03-23 |
| Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures GWP42003-P - Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome | Phase 3 | Terminated | Jazz Pharmaceuticals | 3 | 2021-05-19 | 2025-10-31 |
| Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants Vigabatrin, Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 2 / Phase 3 | Recruiting | Katarzyna Kotulska | 60 | 2021-05-07 | 2026-10-22 |
| Topical Everolimus in Patients With Tuberous Sclerosis Complex Everolimus, Placebo - Facial Angiofibromas | Phase 2 / Phase 3 | Withdrawn | Hospices Civils de Lyon | 2020-12-01 | 2021-05-03 | |
| A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I NPC-12G gel - Neurofibromatosis | Phase 3 | Terminated | Nobelpharma | 100 | 2020-07-08 | 2022-05-24 |
| A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma Carboplatin, Magnetic Resonance Imaging, Quality-of-Life Assessment - Low Grade Glioma, Neurofibromatosis Type 1 | Phase 3 | Active not recruiting | National Cancer Institute (NCI) | 165 | 2020-01-15 | 2026-08-13 |
| Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex rapamycin, placebo - Facial Angiofibroma, Tuberous Sclerosis | Phase 2 / Phase 3 | Completed | Dermatology Specialties Limited Partnership | 107 | 2019-07-28 | 2023-09-08 |
| Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections Brolucizumab, Aflibercept - Age-Related Macular Degeneration | Phase 3 | Terminated | Novartis Pharmaceuticals | 535 | 2018-10-30 | 2023-01-30 |
| Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study EryDex System - Ataxia Telangiectasia, Genetic Syndrome | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 104 | 2018-06-12 | 2024-10-08 |
Showing 20 of 36. Sign in to see all of them.
Tuberous Sclerosis (19) · Neurofibromatosis 1 (6) · Ataxia Telangiectasia (5) · Seizures (5) · Epilepsy (3) · Neurofibroma, Plexiform (3) · Angiofibroma (2) · Astrocytoma (2) · Neoplasms (2) · Neurofibromatoses (2) · Neurofibromatosis 2 (2) · Plaque, Amyloid (2) · Tuberous Sclerosis 2 (2) · Angelman Syndrome (1) · Angiomyolipoma (1)
Nervous System Diseases (36) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (35) · Genetic Diseases, Inborn (34) · Heredodegenerative Disorders, Nervous System (29) · Neoplasms (29) · Neoplastic Syndromes, Hereditary (29) · Neurodegenerative Diseases (29) · Congenital Abnormalities (20) · Hamartoma (19) · Malformations of Cortical Development (19) · Malformations of Cortical Development, Group I (19) · Neoplasms, Multiple Primary (19) · Nervous System Malformations (19) · Neoplasms by Histologic Type (15)
Everolimus (5) · Sirolimus (5) · Cannabidiol (3) · dexamethasone 21-phosphate (2) · ganaxolone (2) · AZD 6244 (1) · Acceptance and Commitment Therapy (1) · Calcitriol (1) · Carboplatin (1) · Control Groups (1) · Fluid Therapy (1) · HDAC-42 (1) · Heptavalent Pneumococcal Conjugate Vaccine (1) · IB1001 (1) · Imatinib Mesylate (1)
Novartis Pharmaceuticals (5) · Nobelpharma (4) · Jazz Pharmaceuticals (3) · Quince Therapeutics S.p.A. (3) · Erasmus Medical Center (2) · Katarzyna Kotulska (2) · Marinus Pharmaceuticals (2) · National Cancer Institute (NCI) (2) · Anne Comi, MD (1) · AstraZeneca (1) · Dermatology Specialties Limited Partnership (1) · Hospices Civils de Lyon (1) · Institute of Child Health (1) · IntraBio Inc (1) · Medical University of Vienna (1)
United States (21) · Spain (15) · United Kingdom (15) · Poland (11) · Germany (10) · Italy (10) · Australia (9) · Canada (8) · France (8) · Japan (8) · Netherlands (7) · Belgium (5) · Russia (5) · China (4) · Denmark (3)
Dirk Thye, MD (2) · Alice P. Chen, MD (1) · Anne M Comi, M.D. (1) · Guenter R Janhofer, MD, PhD (1) · Henriette A Moll, MD, PhD (1) · Ioana Stanescu (1) · Jason R Fangusaro (1) · Katarzyna Kotulska-Jozwiak (1) · Mari Wataya-Kaneda, MD, PhD (1) · Mari Wataya-Kaneda,, MD, PhD (1)
PMID 29023494, PMID 30053159, PMID 34957550, PMID 23158522, PMID 23733802, PMID 24729041, PMID 25456370, PMID 25682485, PMID 26156073, PMID 26858193, PMID 27351628, PMID 29096777, PMID 29800026, PMID 30192751, PMID 31217257, PMID 31982581, PMID 32385845, PMID 33346789, PMID 33395032, PMID 35175622, PMID 37463422, PMID 38441854, PMID 39340758, PMID 39917433, PMID 40121838, PMID 40473450, PMID 42309084, PMID 42600624
2015 (2), 2018 (2), 2008 (1), 2025 (1), U10CA180886 (1), U54NS065705 (1)