Neurofibromatosis Type 1
Pipeline by Development Stage
Drug Modality Breakdown
On Market (1)
Approved therapies currently available
Competitive Landscape
11 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Showing 15 of 23 trials with date data
Clinical Trials (23)
Total enrollment: 1,998 patients across 23 trials
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
Interventions for Reading Disabilities in NF1
Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
International Trial of Selumetinib in Combination With Dexamethasone for the Treatment of Acute Lymphoblastic Leukaemia
A Study of Selumetinib in Patients With Kaposi's Sarcoma
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 and Olaparib in Solid Tumors
A Study to Assess the Relative Bioavailability of Process Variants of Selumetinib in Healthy Male Volunteers
To Assess the Effect of Itraconazole and Fluconazole on the Pharmacokinetics of Selumetinib in Healthy Male Volunteers
To Assess the Pharmacokinetics, Safety and Tolerability of Selumetinib in Patients With Hepatic Impairment and Healthy Subjects
To Assess the Pharmacokinetics, Safety and Tolerability of Selumetinib in Renal Impaired Subjects and Healthy Subjects
To Assess the Effect of Rifampicin on the Pharmacokinetics of Selumetinib in Healthy Male Volunteers
Dose-escalation Study to Assess Selumetinib Safety, Tolerability and PK
Real-World Treatment Study of Koselugo (Selumetinib)
PASS of Paediatric Patients Initiating Selumetinib
US Selumetinib Registry
Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I
Phase Legend
Key Insights
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