Neurofibromatosis Type 1
77
9
18
41
Key Insights
Highlights
Success Rate
87% trial completion (above average)
Published Results
15 trials with published results (19%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
7.8%
6 terminated out of 77 trials
87.2%
+0.7% vs benchmark
5%
4 trials in Phase 3/4
37%
15 of 41 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 41 completed trials
Clinical Trials (77)
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence
Pediatric Long-Term Follow-up and Rollover Study
Gait in Young Children With NF1
Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)
Natural History Study of Patients With Neurofibromatosis Type I
Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
Familial Investigations of Childhood Cancer Predisposition
Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)
Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
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)
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
PASS of Paediatric Patients Initiating Selumetinib
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
FARD (RaDiCo Cohort) (RaDiCo-FARD)
Neurofibromatosis Type 1 Tumor Early Detection Study
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
Natural History Study of Cutaneous Neurofibromas in People With NF1