Suspended

Selumetinib Safety and Efficacy in NF1 Patients with Inoperable Plexiform Neurofibromas

Study Aim

This phase 1 study aims to evaluate the safety and efficacy of Selumetinib in Chinese pediatric and adult patients with Neurofibromatosis Type 1 (NF1) and inoperable Plexiform Neurofibromas, focusing on the drug's side effects and its concentration levels in the body after single and multiple doses.

What is being tested

Selumetinib

Drug

Who is being recruted

Congenital, Hereditary, and Neonatal Diseases and Abnormalities+16

+ Neoplasms

+ Neoplasms by Histologic Type

From 3 to 99 Years

+10 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 1

Interventional

Study Start: December 2020

See protocol details

Summary

Principal SponsorAstraZeneca

Last updated: January 31, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: December 16, 2020

Actual date on which the first participant was enrolled.

This study focuses on a treatment for Chinese pediatric and adult patients with a condition called Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN), which are tumors that grow on nerves and cannot be removed by surgery. The goal is to evaluate the safety, tolerability, and effectiveness of a drug named Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor. This drug is designed to help control the growth of these tumors. The study is important as it aims to provide a potential solution for managing this condition, addressing the unmet needs of these patients and possibly improving their quality of life. During the study, approximately 16 pediatric and 16 adult patients will receive Selumetinib orally, twice a day, at a dose of 25 mg/m^2. They will continue this treatment until the disease progresses or they experience unacceptable drug-related toxicity. The study will monitor adverse events, their frequency, and their relationship to the treatment. It will also measure how the drug behaves in the body after single and multiple doses, including its maximum concentration in the plasma and its terminal half-life. After a patient discontinues the treatment, they will be followed for a specified period for safety assessment.

Official TitleA 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)

NCT04590235

Principal SponsorAstraZeneca

Last updated: January 31, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

32 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 3 to 99 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesNeoplasmsNeoplasms by Histologic TypeNeoplasms, Nerve TissueNeoplastic Syndromes, HereditaryNervous System DiseasesNervous System NeoplasmsNeurofibromaNeurofibromatosis 1Neuromuscular DiseasesPeripheral Nervous System DiseasesPeripheral Nervous System NeoplasmsNeurofibromatosesNerve Sheath NeoplasmsNeurofibroma, PlexiformNeurodegenerative DiseasesHeredodegenerative Disorders, Nervous SystemNeurocutaneous SyndromesGenetic Diseases, Inborn

Criteria

6 inclusion criteria required to participate

Chinese subjects ≥3 years and <18 years of age.

Chinese subjects ≥18 years of age at the time of study enrollment.

Subjects must be diagnosed with (i) NF1 as per NIH Consensus Development Conference Statement and（ii) PN is defined as a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches. (iii) inoperable PN.

Subjects must have at least one measurable typical or nodular PN.

Show More Criteria

4 exclusion criteria prevent from participating

Evidence of malignant peripheral nerve sheath tumour.

Clinically significant cardiovascular disease.

Prior malignancy (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the subject had been disease free for ≥2 years or which would not have limited survival to <2 years) or other cancer requiring treatment with chemotherapy or radiation therapy.

Subjects with the following ophthalmological findings/conditions: Current or past history of retinal pigment epithelial detachment/central serous retinopathy or retinal vein occlusion; Intraocular pressure >21 mmHg (or ULN adjusted by age) or uncontrolled glaucoma (irrespective of IOP); Subjects with known glaucoma and increased IOP who do not have meaningful vision (light perception only or no light perception) and are not experiencing pain related to the glaucoma, may be eligible after discussion with the study physician; Any other significant abnormality on ophthalmic examination that would make the subject unsuitable for enrolment into the study, as assessed by the investigator.

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

All eligible subjects will first receive a single oral dose of selumetinib 25 mg/m\^2. Then, selumetinib 25 mg/m\^2 oral twice daily will be administered continuously until disease progression or unacceptable drug-related toxicity, whichever occurs first.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 2 locations

Suspended

Research Site

Shanghai, ChinaOpen Research Site in Google Maps

Suspended

Research Site

Shanghai, China

Suspended2 Study Centers