37 completed, 11 recruiting, 10 active not recruiting, 7 terminated. 72 Phase 1, 30 Phase 2.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| Sapu003 in Advanced mTOR-sensitive Solid Tumors Sapu003, Exemestane 25 MG - Breast Cancer Metastatic, Renal Cell Carcinoma (RCC) | Phase 1 | Recruiting | SAPU NANO (US) LLC | 27 | 2025-12-15 | 2026-01-27 |
| 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 |
| 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 |
| PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas PAS-004 Tablets, PAS-004 Capsule - NF1 Mutation, Neurofibroma Plexiform | Phase 1 | Recruiting | Pasithea Therapeutics Corp. | 56 | 2025-05-30 | 2026-10-28 |
| Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs. Radiprodil + co-administered drugs - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Completed | GRIN Therapeutics, Inc. | 18 | 2025-05-20 | 2025-11-18 |
| Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults Radiprodil with Carbamazepine, Radiprodil with Itraconazole - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Terminated | GRIN Therapeutics, Inc. | 30 | 2025-05-08 | 2025-11-21 |
| Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs) Kybella - Neurofibromas, Cutaneous, Neurofibromatosis 1 | Phase 1 | Enrolling by invitation | Massachusetts General Hospital | 15 | 2025-04-08 | 2026-04-22 |
| 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 |
| A Study of HS-10516 in Patients With VHL Syndrome Associated Tumors Oral HS-10516 - Von Hippel Lindau-Deficient Clear Cell Renal Cell Carcinoma | Phase 1 | Recruiting | Jiangsu Hansoh Pharmaceutical Co., Ltd. | 60 | 2024-07-19 | 2024-08-14 |
| Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II Radiprodil - Tuberous Sclerosis Complex, Focal Cortical Dysplasia | Phase 1 / Phase 2 | Active not recruiting | GRIN Therapeutics, Inc. | 30 | 2024-07-10 | 2026-04-01 |
| Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas Deoxycholic Acid, Polidocanol, 1064nm Nd:YAG laser - Neurofibromatosis 1 | Phase 1 | Completed | Massachusetts General Hospital | 13 | 2024-03-01 | 2025-11-21 |
| A Study of BPI-452080 in Subjects With Solid Tumors BPI-452080 - Solid Tumor, Renal Cell Carcinoma | Phase 1 | Unknown | Betta Pharmaceuticals Co., Ltd. | 87 | 2023-04-28 | 2023-05-06 |
| Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas Kybella, Asclera - Neurofibromatosis 1 | Phase 1 | Completed | Massachusetts General Hospital | 20 | 2022-12-06 | 2025-02-21 |
Showing 20 of 72. Sign in to see all of them.
Neurofibromatosis 1 (30) · Tuberous Sclerosis (16) · Neurofibroma, Plexiform (9) · Neurofibromatoses (9) · von Hippel-Lindau Disease (9) · Hereditary Sensory and Autonomic Neuropathies (5) · Neurofibromatosis 2 (5) · Ataxia Telangiectasia (4) · Epilepsy (4) · Focal Cortical Dysplasia (4) · Lymphangioleiomyomatosis (4) · Neurofibroma (4) · Neurofibrosarcoma (4) · Astrocytoma (3) · Carcinoma, Renal Cell (3)
Nervous System Diseases (72) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (69) · Genetic Diseases, Inborn (69) · Neoplasms (64) · Heredodegenerative Disorders, Nervous System (57) · Neurodegenerative Diseases (57) · Neoplasms by Histologic Type (56) · Neoplastic Syndromes, Hereditary (56) · Neoplasms, Nerve Tissue (46) · Nerve Sheath Neoplasms (41) · Neuromuscular Diseases (38) · Peripheral Nervous System Diseases (38) · Neurofibroma (37) · Neurofibromatoses (32)
AZD 6244 (9) · Everolimus (7) · Sirolimus (5) · Deoxycholic Acid (4) · Ranibizumab (3) · radiprodil (3) · Cetuximab (2) · Injections (2) · Lasers, Solid-State (2) · Magnetic Resonance Spectroscopy (2) · Polidocanol (2) · Specimen Handling (2) · Acetaminophen (1) · Aminolevulinic Acid (1) · Azacitidine (1)
National Cancer Institute (NCI) (7) · AstraZeneca (5) · Children's Hospital Medical Center, Cincinnati (5) · Massachusetts General Hospital (5) · National Eye Institute (NEI) (4) · GRIN Therapeutics, Inc. (3) · Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (2) · Johns Hopkins University (2) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (2) · AeRang Kim (1) · Anne Comi, MD (1) · Bayer (1) · Beijing Tiantan Hospital (1) · Beijing Tide Pharmaceutical Co., Ltd (1) · Betta Pharmaceuticals Co., Ltd. (1)
United States (54) · China (9) · Australia (7) · Spain (6) · Canada (3) · Italy (3) · Japan (3) · Netherlands (3) · United Kingdom (3) · Germany (2) · Poland (2) · Russia (2) · Belgium (1) · Czechia (1) · France (1)
Brigitte C Widemann, M.D. (4) · Richard R Anderson, MD (3) · Jason R Fangusaro (2) · AeRang Kim, MD, PhD (1) · Alex V Levin, MD, MHSc (1) · Anne M Comi, MD (1) · Brigitte C. Widemann, MD (1) · Carlos Romo, MD (1) · Carmen P Priolo, MD PhD (1) · Carrie E Bearden, PhD (1) · Catherine Meyerle, MD (1) · Christelle Achkar, MD (1) · Clay Hoerig, MD (1) · D. Bradley Welling, MD, PhD (1)
PMID 21047224, PMID 21482669, PMID 21764821, PMID 22262746, PMID 22934754, PMID 23325902, PMID 23798472, PMID 26381530, PMID 28029918, PMID 29023494, PMID 30424962, PMID 31151904, PMID 32187457, PMID 33470897, PMID 33641042, PMID 34549489, PMID 35170228, PMID 35410754, PMID 35467749, PMID 37018064, PMID 37115514, PMID 37311055, PMID 37400844, PMID 38721358, PMID 38869947, PMID 39331837, PMID 40709016, PMID 41139346, PMID 41389341, PMID 41592259, PMID 42394972, PMID 42540436
UM1CA081457 (2), 2018 (1), 2023 (1), U01CA081457 (1)