Neurofibromatosis 1
68
17
28
26
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 68 trials
100.0%
+13.5% vs benchmark
3%
2 trials in Phase 3/4
38%
10 of 26 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 26 completed trials
Clinical Trials (68)
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Real-World Treatment Study of Koselugo (Selumetinib)
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
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
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
NF-1, Nutraceutical Intervention
Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1
Neurofibromatosis Type 1 Tumor Early Detection Study
Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing
RASopathy Biorepository
Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1