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

Phase 1 Active not recruiting 32 enrolled AstraZeneca Merck Sharp & Dohme LLC · collab

Non-randomizedSingle-groupOpen-labelTreatment

Summary

A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN).

Timeline

Start: 2020-12-16
Primary completion: 2022-08-16
Completion: 2026-08-31

Publications

Zhang X, Sui W, Zheng H, Chen J, Yuan X. Durable responses to long-term selumetinib in Chinese pediatric NF1 patients with inoperable plexiform neurofibromas. Front Pharmacol. 2026 Jun 18;17:1855171. doi: 10.3389/fphar.2026.1855171. eCollection 2026.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Selumetinib	Small molecule	25 mg/m2	Oral

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

Summary

Timeline

Publications

Drugs

Indications