neurofibromatosis type 1
neurofibromatosis type 1
Explore linked clinical trials. Standard reference: MONDO:001897561 results ยท Page 1 of 3
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Active, not recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT01362803 | |
| Natural History Study of Patients With Neurofibromatosis Type I | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, connective tissue neoplasm, cranial nerve neoplasm, cranial nerve neuropathy, epithelial neoplasm, eye disorder, fibrosarcoma, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, optic nerve disorder, optic nerve glioma, optic nerve neoplasm, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma, primitive neuroectodermal tumor, sarcoma | Active, not recruiting | Not stated | 2026-10-08 | NCT00924196 | |
| MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Active, not recruiting | Phase 2 | 2026-10-06 | NCT03962543 | |
| Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioblastoma, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, primitive neuroectodermal tumor | Not yet recruiting | Phase 2 | 2026-10-05 | NCT07521657 | |
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Recruiting | Phase 1 | 2026-10-02 | NCT06502171 | |
| Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Recruiting | Not stated | 2026-10-02 | NCT02544022 | |
| Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics | Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Malformations of Cortical Development, Group I, Neoplasms by Histologic Type, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Skin Abnormalities, Skin and Connective Tissue Diseases, Sturge-Weber syndrome, angiomatosis, cardiovascular disorder, central nervous system malformation, cerebral cortical dysplasia, ectodermal dysplasia syndrome, hamartoma, hemangioma, hereditary disease, hereditary neoplastic syndrome, hereditary skin disorder, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, skin disorder, tuberous sclerosis, vascular cancer, vascular disorder | Not yet recruiting | Not stated | 2026-09-28 | NCT07843823 | |
| NF-1, Nutraceutical Intervention | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Active, not recruiting | Phase 1 | 2026-09-23 | NCT05363267 | |
| Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, low grade glioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, primitive neuroectodermal tumor | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-21 | NCT01089101 | |
| Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-09-16 | NCT04750928 | |
| MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Active, not recruiting | Phase 2 | 2026-09-15 | NCT02407405 | |
| How Can the Experience of Hospital Outpatient Departments be Improved? | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ear Diseases, NF2-related schwannomatosis, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, communication disorder, eye disorder, hearing disorder, hearing loss disorder, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, learning disability, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurodevelopmental disorder, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, otorhinolaryngologic disease, peripheral nervous system disorder, psychiatric disorder, vision disorder | Not yet recruiting | Not stated | 2026-09-14 | NCT07818213 | |
| Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Recruiting | Not stated | 2026-09-11 | NCT07221331 | |
| Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Enrolling by invitation | Not stated | 2026-09-09 | NCT05238909 | |
| A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia | CBL-related disorder, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, bone marrow disorder, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, juvenile myelomonocytic leukemia, leukemia, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-09-04 | NCT05849662 | |
| Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, connective tissue neoplasm, fibrosarcoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma, sarcoma | Recruiting | Not stated | 2026-09-02 | NCT06222203 | |
| Neurofibromatosis Type 1 Tumor Early Detection Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Malignant Peripheral Nerve Sheath Tumors, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, atypical neurofibroma, connective tissue neoplasm, fibrosarcoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma, sarcoma | Enrolling by invitation | Not stated | 2026-08-31 | NCT06515860 | |
| Pediatric Long-Term Follow-up and Rollover Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Oligodendroglioma, Childhood, anaplastic astrocytoma, anaplastic ganglioglioma, anaplastic oligodendroglioma, anaplastic pleomorphic xanthoastrocytoma, angiocentric glioma, astrocytoma (excluding glioblastoma), brain disorder, central nervous system cancer, central nervous system disorder, central neurocytoma, cerebellar liponeurocytoma, chordoid glioma of the third ventricle, diffuse astrocytoma, epithelial neoplasm, extraventricular neurocytoma, gangliocytoma, ganglioglioma, glioblastoma, glioma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, inherited neurodegenerative disorder, interstitial lung disease, lung disorder, lymphatic system disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neuroblastoma, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, oligodendroglioma, papillary glioneuronal tumor, peripheral nervous system disorder, peripheral nervous system neoplasm, pilocytic astrocytoma, plexiform neurofibroma, primitive neuroectodermal tumor, respiratory system disorder, rosette-forming glioneuronal tumor of fourth ventricule | Active, not recruiting | Phase 4 | 2026-08-27 | NCT03975829 | |
| Real-World Treatment Study of Koselugo (Selumetinib) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Recruiting | Not stated | 2026-08-25 | NCT06360406 | |
| PASS of Paediatric Patients Initiating Selumetinib | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Active, not recruiting | Not stated | 2026-08-24 | NCT05388370 | |
| Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial | Abnormalities, Multiple, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Costello syndrome, Craniofacial Abnormalities, Legius syndrome, Musculoskeletal Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Noonan syndrome, Skin and Connective Tissue Diseases, cardiofaciocutaneous syndrome, cardiovascular disorder, congenital heart disease, connective tissue disorder, heart disorder, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, musculoskeletal system disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Recruiting | Not applicable | 2026-08-24 | NCT05361811 | |
| Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, connective tissue neoplasm, fibrosarcoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, sarcoma | Recruiting | Phase 2 | 2026-08-24 | NCT05253131 | |
| Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Active, not recruiting | Phase 1 | 2026-08-20 | NCT05101148 | |
| A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Active, not recruiting | Phase 1 | 2026-08-18 | NCT04590235 | |
| Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm | Recruiting | Not stated | 2026-08-14 | NCT06880991 |