Neurofibromatosis 1
68
14
26
27
Key Insights
Highlights
Success Rate
96% trial completion (above average)
Published Results
12 trials with published results (18%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
1.5%
1 terminated out of 68 trials
96.4%
+9.9% vs benchmark
3%
2 trials in Phase 3/4
44%
12 of 27 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 27 completed trials
Clinical Trials (68)
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Neurofibromatosis Type 1 Tumor Early Detection Study
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Real-World Treatment Study of Koselugo (Selumetinib)
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
NF-1, Nutraceutical Intervention
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial