41 completed, 10 active not recruiting, 8 unknown, 7 recruiting. 18 Phase 2, 13 Not applicable, 10 Phase 1, 3 Phase 3, 2 Phase 4.
Follow Neurofibromatosis Type 1: new trials by email Literature Review of Neurofibromatosis Type 1 trials Research Report on Neurofibromatosis Type 1
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 |
|---|---|---|---|---|---|---|
| Gait in Young Children With NF1 Neurofibromatosis Type 1 | Not yet recruiting | NYU Langone Health | 56 | 2026-08-01 | 2026-05-08 | |
| A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1) AlgometRx Nociometer(R) - Neurofibromatosis Type 1 | Not applicable | Recruiting | National Cancer Institute (NCI) | 70 | 2026-02-18 | 2026-10-16 |
| Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy) Neurofibromatosis Type 1 | Recruiting | AstraZeneca | 200 | 2025-11-19 | 2026-09-11 | |
| Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study Arm 1 - Neurofibromatosis Type 1, Neurofibroma | Recruiting | National Cancer Institute (NCI) | 110 | 2025-04-29 | 2026-10-16 | |
| 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 |
| Neurofibromatosis Type 1 Tumor Early Detection Study Liquid biopsy for MPNST development - Neurofibromatosis Type 1, Neurofibromatosis 1 | Enrolling by invitation | David Miller | 1000 | 2024-08-07 | 2026-08-31 | |
| Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study Selumetinib, Placebo - Neurofibromatosis Type 1, Plexiform Neurofibromas | Phase 2 | Withdrawn | AstraZeneca | 2023-08-31 | 2023-08-14 | |
| Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence Whole-body Magnetic Resonance Imaging - Neurofibromatosis Type 1 | Not applicable | Recruiting | Nicole Baca | 15 | 2022-07-08 | 2026-05-07 |
| PASS of Paediatric Patients Initiating Selumetinib Neurofibromatosis Type 1 | Active not recruiting | AstraZeneca | 124 | 2022-05-23 | 2026-08-24 | |
| Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1 Neurofibromatosis 1, NF1 | Enrolling by invitation | Ann & Robert H Lurie Children's Hospital of Chicago | 200 | 2022-03-04 | 2026-09-09 | |
| Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN Selumetinib granule formulation, Selumetinib capsule formulation - Neurofibromatosis Type 1 | Phase 1 / Phase 2 | Active not recruiting | AstraZeneca | 36 | 2022-01-21 | 2026-08-13 |
| Molecular Aspects of Preimplantation Genetic Diagnosis for NF1 Neurofibromatosis Type 1 | Completed | University Hospital, Strasbourg, France | 60 | 2021-09-15 | 2024-10-16 | |
| 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 Selumetinib - Neurofibromatosis Type 1 | Phase 1 | Active not recruiting | AstraZeneca | 24 | 2021-07-21 | 2026-08-20 |
| Natural History Study of Cutaneous Neurofibromas in People With NF1 Evaluation of the natural history of cutaneous neurofibromas - Neurofibromatosis Type 1, Neurofibromatosis 1 | Active not recruiting | Johns Hopkins University | 500 | 2021-06-28 | 2026-10-23 | |
| Selumetinib Paediatric NF1 Japan Study Selumetinib - Neurofibromatosis Type 1 | Phase 1 | Completed | AstraZeneca | 12 | 2020-08-31 | 2023-04-26 |
| Function of the Pigment Epithelium in Patients With Type 1 Neurofibromatosis Electro-oculogram, Full-field electroretinogram - Neurofibromatosis Type 1 | Completed | Assistance Publique - Hôpitaux de Paris | 30 | 2020-05-11 | 2025-09-12 | |
| 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 |
| Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- Retest Outcome measures - Neurofibromatosis Type 1 | Unknown | Guy's and St Thomas' NHS Foundation Trust | 20 | 2019-11-30 | 2023-03-03 | |
| Pediatric Long-Term Follow-up and Rollover Study dabrafenib, trametinib - Diffuse Astrocytoma, Anaplastic Astrocytoma | Phase 4 | Active not recruiting | Novartis Pharmaceuticals | 163 | 2019-11-04 | 2026-08-27 |
| Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours Selumetinib - Neurofibromatosis Type 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Completed | Great Ormond Street Hospital for Children NHS Foundation Trust | 14 | 2019-09-26 | 2024-04-04 |
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Neurofibromatosis 1 (77) · Neurofibroma, Plexiform (18) · Neurofibroma (9) · Neurofibromatoses (7) · Neurofibrosarcoma (6) · Optic Nerve Glioma (6) · Dyslexia (4) · Astrocytoma (3) · Brain Neoplasms (3) · Central Nervous System Neoplasms (3) · Glioma (3) · Neuroblastoma (3) · Precancerous Conditions (3) · Learning Disabilities (2) · Neoplasms (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (77) · Genetic Diseases, Inborn (77) · Heredodegenerative Disorders, Nervous System (77) · Neoplasms by Histologic Type (77) · Neoplasms, Nerve Tissue (77) · Neoplastic Syndromes, Hereditary (77) · Nerve Sheath Neoplasms (77) · Nervous System Diseases (77) · Neurocutaneous Syndromes (77) · Neurodegenerative Diseases (77) · Neuromuscular Diseases (77) · Peripheral Nervous System Diseases (77) · Neoplasms (75) · Neurofibromatoses (70) · Neurofibroma (68)
AZD 6244 (6) · Biopsy (2) · Carboplatin (2) · Lovastatin (2) · Magnetic Resonance Imaging (2) · Magnetic Resonance Spectroscopy (2) · Vinblastine (2) · trametinib (2) · Acceptance and Commitment Therapy (1) · Bevacizumab (1) · Dosage Forms (1) · Doxorubicin (1) · Etoposide (1) · Filgrastim (1) · Fluorodeoxyglucose F18 (1)
National Cancer Institute (NCI) (16) · AstraZeneca (6) · Shriners Hospitals for Children (3) · University Hospital, Toulouse (3) · University of Alabama at Birmingham (3) · Washington University School of Medicine (3) · Ann & Robert H Lurie Children's Hospital of Chicago (2) · Assistance Publique - Hôpitaux de Paris (2) · Children's Hospital of Philadelphia (2) · KU Leuven (2) · Massachusetts General Hospital (2) · Vanderbilt University (2) · Alexion Pharmaceuticals, Inc. (1) · Case Comprehensive Cancer Center (1) · Children's National Research Institute (1)
United States (54) · France (11) · Belgium (5) · Spain (5) · United Kingdom (5) · Canada (4) · Netherlands (4) · Australia (3) · Germany (3) · Italy (3) · Japan (3) · Russia (3) · Israel (2) · Argentina (1) · Austria (1)
Brigitte C Widemann, M.D. (3) · Brigitte C. Widemann, MD (2) · Douglas R Stewart, M.D. (2) · Eric Legius, MD PhD (2) · Jason R Fangusaro (2) · Kimberly J Johnson, PhD (2) · Pamela L Wolters, Ph.D. (2) · Roger Packer, MD (2) · Staci M Peron, Ph.D. (2) · Barbara B Biesecker (1) · Brian Weiss, MD (1) · Bruce Korf, MD (1) · Bruce Korf, MD, PhD (1) · Bruce R Korf (1)
PMID 21764821, PMID 24321536, PMID 25205361, PMID 27956565, PMID 28029918, PMID 30339775, PMID 32152628, PMID 32187457, PMID 33395032, PMID 33507832, PMID 33641042, PMID 33728588, PMID 35007270, PMID 35394491, PMID 35467749, PMID 37115514, PMID 37126770, PMID 38721358, PMID 38867349, PMID 38869947, PMID 38907342, PMID 39340758, PMID 39418437, PMID 40009403, PMID 40121838, PMID 40709016, PMID 41592259, PMID 42540436
U10CA180886 (3), 2023 (1), K23NS146694 (1), N01CM17104 (1), N01CM62205 (1), P30CA013148 (1), P30CA015083 (1), R01HD089474 (1), R01NS049096 (1), R01NS050509 (1), R33NS122094 (1), R61NS122094 (1), U10CA098543 (1), U54CA196519 (1), UM1CA081457 (1)