Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs
PEDIA
Observational Study of the Effectiveness and Safety of the MEK 1/2 Inhibitor (MEKi) Koselugo (Selumetinib) in Pediatric Patients With Symptomatic, Inoperable, Neurofibromatosis Type 1 (NF1)-Associated Plexiform Neurofibromas (PN)
1 other identifier
observational
409
1 country
25
Brief Summary
This is a prospective, multicenter, observational study of Chinese pediatric NF1-PN patients treated with selumetinib. The study will be conducted at approximately 12 centers in China and will include approximately 80-100 patients. Treatment centers that have PN diagnosis and/or selumetinib treatment experience will be targeted for recruitment. Patients/caregivers who are eligible and willing to participate will be enrolled into the study. Patients will start selumetinib treatment after enrollment. The study will have a 16-month enrollment period. Patients will be followed up until the end of a 24-month observation period after first dose of selumetinib, or patient death, lost to follow-up, withdrawal of consent, whichever occurs first. Patients will be followed within a 24-month period (starting after first dose received) in the study even if selumetinib is discontinued. The aims of this study are to expand understanding of disease characteristics and treatment pattern of NF1-PN in China in a real-world setting and to evaluate real-world effectiveness and safety of selumetinib for Chinese pediatric patients with NF1-PN
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Dec 2023
Typical duration for all trials
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 8, 2023
CompletedFirst Posted
Study publicly available on registry
December 19, 2023
CompletedStudy Start
First participant enrolled
December 20, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 30, 2027
February 12, 2026
February 1, 2026
3.4 years
November 8, 2023
February 11, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (5)
NF1-PN patient demographics and disease characteristics at baseline and during selumetinib treatment
Race,ethnicity,Gender,Date of birth,Relationship of caregiver to patient
From baseline to 24 months after first dose of selumetinib
NF1-PN disease characteristics
Date of diagnosis or age at diagnosis of NF1, Date of diagnosis or age at diagnosis of PN, NF1 clinical diagnosis criteria and related manifestations, NF1 and PN diagnostic tests and results, Morbidities related to PN
From baseline to 24 months after first dose of selumetinib
Selumetinib treatment profile
Dose and dose modification, start and stop dates, dose interruption and discontinuation, reasons for interruption, discontinuation and dose modification.
From baseline to 24 months after first dose of selumetinib
Physician's qualitative assessment of disease status for clinically significant PN and overall disease status
Improving with treatment, progression, stable
From baseline to 24 months after first dose of selumetinib
NF1 disease status
Presence, number, size (Diameter - centimeters or cubic centimeters of volume) and location (neck/trunk, trunk and limbs, limbs only, head only, head and neck, trunk only) of PN/ optic pathway gliomas/ café-au-lait macules/ cutaneous neurofibroma.
From baseline to 24 months after first dose of selumetinib
Secondary Outcomes (4)
To assess tumor activity of NF1-PN upon discontinuation of selumetinib
From baseline to 24 months after first dose of selumetinib
To describe the course of patients' disease and treatment following discontinuation of selumetinib
From baseline to 24 months after first dose of selumetinib
To describe caregiver- or patient-reported treatment adherence
From baseline to 24 months after first dose of selumetinib
To determine whether patients treated with selumetinib have a clinically meaningful decrease in pain intensity associated with pain scale
From baseline to 24 months after first dose of selumetinib
Eligibility Criteria
The target population of interest in this study is pediatric patients with neurofibromatosis type 1 (NF1) aged 3-16 years (3 and 16 years are inclusive) who have symptomatic, inoperable plexiform neurofibromas (PN) and intend to use selumetinib after enrollment. Each eligible patient should meet all of the inclusion criteria and none of the exclusion criteria. At least 80 and no more than 100 eligible patients will be enrolled in the study.
You may qualify if:
- (1) 3≤ age ≤16 years old at the time of study enrollment; (2) Diagnosed with NF1 as per the revised 2021 Guidelines; (3) Have symptomatic, inoperable PN: (4) Intend to use selumetinib after enrollment; (5) Able to give written informed consent. Data collection must only be done after obtaining written informed consent. Patient or patient's parent/legal guardian must be willing and able to give written informed consent. Parent or legal guardian consent is required in the assent process with appropriate documentation. Mandatory provision of signed and dated parent/legal guardian consent for the study along with the pediatric assent form, when applicable.
You may not qualify if:
- (1) Evidence of MPNST, prior malignancy or other cancer requiring treatment with chemotherapy or radiation therapy; (2) A life-threatening illness, medical condition, or organ system dysfunction; (3) Have had prior treatment with a MEKi, Ras or Raf inhibitor; (4) Patients currently participating in any clinical trials at the time of enrollment or initiation of selumetinib.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AstraZenecalead
Study Sites (25)
Research Site
Beijing, Beijing Municipality, China
Research Site
Chongqing, Chongqing Municipality, China
Research Site
Lanzhou, Gansu, China
Research Site
Guangzhou, Guangdong, China
Research Site
Shenzhen, Guangdong, China
Research Site
Nanning, Guangxi, China
Research Site
Guiyang, Guizhou, China
Research Site
Shijiazhuang, Hebei, China
Research Site
Zhengzhou, Henan, China
Research Site
Wuhan, Hubei, China
Research Site
Changsha, Hunan, China
Research Site
Nanjing, Jiangsu, China
Research Site
Suzhou, Jiangsu, China
Research Site
Nanchang, Jiangxi, China
Research Site
Shenyang, Liaoning, China
Research Site
Jinan, Shandong, China
Research Site
Shanghai, Shanghai Municipality, China
Research Site
Taiyuan, Shanxi, China
Research Site
Xi’an, Shanxi, China
Research Site
Chengdu, Sichuan, China
Research Site
Tianjin, Tianjin Municipality, China
Research Site
Kunming, Yunnan, China
Research Site
Hangzhou, Zhejiang, China
Research Site
Wenzhou, Zhejiang, China
Research Site
Nanjing, China
Biospecimen
No Biospecimen sample will be collected in this study，all participants will receive clinical examination base on routine clinical diagnosis and treatment procedures.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Xin Ni, PhD
Beijing Children hospital
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 8, 2023
First Posted
December 19, 2023
Study Start
December 20, 2023
Primary Completion (Estimated)
May 30, 2027
Study Completion (Estimated)
May 30, 2027
Last Updated
February 12, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA/PhRMA Data-Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
- Access Criteria
- When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. "Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be approved.