Key Insights

Highlights

Success Rate

100% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 50/100

Termination Rate

0.0%

0 terminated out of 68 trials

Success Rate

100.0%

+13.5% vs benchmark

Late-Stage Pipeline

3%

2 trials in Phase 3/4

Results Transparency

38%

10 of 26 completed with results

Key Signals

10 with results100% success

Data Visualizations

Phase Distribution

47Total
Not Applicable (16)
P 1 (12)
P 2 (17)
P 3 (2)

Trial Status

Completed26
Recruiting17
Active Not Recruiting11
Unknown6
Not Yet Recruiting5
Enrolling By Invitation2

Trial Success Rate

100.0%

Benchmark: 86.5%

Based on 26 completed trials

Clinical Trials (68)

Showing 20 of 20 trials
NCT01362803Phase 1Active Not RecruitingPrimary

AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

NCT06360406RecruitingPrimary

Real-World Treatment Study of Koselugo (Selumetinib)

NCT04750928Phase 1RecruitingPrimary

Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas

NCT02544022RecruitingPrimary

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)

NCT06300502Phase 1Enrolling By Invitation

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

NCT04481035Phase 2CompletedPrimary

Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1

NCT05849662Phase 1Recruiting

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

NCT04924608Phase 3Active Not RecruitingPrimary

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

NCT06188741Phase 2RecruitingPrimary

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

NCT06502171Phase 1Not Yet RecruitingPrimary

Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas

NCT07233408Not ApplicableRecruitingPrimary

Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy

NCT05361811Not ApplicableRecruitingPrimary

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

NCT05005845Phase 2Completed

NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

NCT04590235Phase 1Active Not RecruitingPrimary

A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

NCT05363267Phase 1Active Not RecruitingPrimary

NF-1, Nutraceutical Intervention

NCT05677594Active Not RecruitingPrimary

Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1

NCT06515860Recruiting

Neurofibromatosis Type 1 Tumor Early Detection Study

NCT03406208Not ApplicableCompleted

Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing

NCT04395495Recruiting

RASopathy Biorepository

NCT04941027CompletedPrimary

Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1

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