AstraZeneca: Neurofibromatosis 1 trials

The registry lists 12 Neurofibromatosis 1 studies with AstraZeneca as lead sponsor. 3 are open to participants now and 2 have results posted.

Phase 1 5Phase 2 2Phase 3 1No phase 5(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN)RecruitingNCT07888517
• Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)RecruitingNCT07221331
• Real-World Treatment Study of Koselugo (Selumetinib)RecruitingNCT06360406
• Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PNActive, not recruitingNCT05309668Phase 1/2Results posted
• Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform NeurofibromasActive, not recruitingNCT04924608Phase 3Results posted
• Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled StudyWithdrawnNCT05825365Phase 2
• US Selumetinib RegistryTerminatedNCT05683678
• PASS of Paediatric Patients Initiating SelumetinibActive, not recruitingNCT05388370
• 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 NeurofibromasActive, not recruitingNCT05101148Phase 1
• A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)Active, not recruitingNCT04590235Phase 1
• Selumetinib Paediatric NF1 Japan StudyCompletedNCT04495127Phase 1
• A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male ParticipantsCompletedNCT03649165Phase 1