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

NCT04590235 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-05-14

No results posted yet for this study

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).

Conditions

  • Neurofibromatosis 1
  • Neurofibroma Plexiform

Interventions

DRUG

Selumetinib

All eligible subjects will first receive a single oral dose of selumetinib 25 mg/m\^2. After a washout period of 2 days, oral selumetinib 25 mg/m\^2 twice daily will be administered continuously. Subjects will continue to receive selumetinib until disease progression or unacceptable drug-related toxicity, whichever occurs first. 10 mg and 25 mg capsules strengths available.

Sponsors & Collaborators

Principal Investigators

  • Qingfeng Li · Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-16
Primary Completion
2022-08-16
Completion
2026-08-31
FDA Drug
Yes

Countries

  • China

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04590235 on ClinicalTrials.gov