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
Eligibility
Sex
ALL
Min age
3 Years
Max age
16 Years
Healthy volunteers
No
Inclusion Criteria:
* (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.
Exclusion Criteria:
* (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.
Primary outcome measure(s)
NF1-PN patient demographics and disease characteristics at baseline and during selumetinib treatment — From baseline to 24 months after first dose of selumetinib Race,ethnicity,Gender,Date of birth,Relationship of caregiver to patient
NF1-PN disease characteristics — From baseline to 24 months after first dose of selumetinib 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
Selumetinib treatment profile — From baseline to 24 months after first dose of selumetinib Dose and dose modification, start and stop dates, dose interruption and discontinuation, reasons for interruption, discontinuation and dose modification.
Physician's qualitative assessment of disease status for clinically significant PN and overall disease status — From baseline to 24 months after first dose of selumetinib Improving with treatment, progression, stable
NF1 disease status — From baseline to 24 months after first dose of selumetinib 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.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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