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Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs

Active Not RecruitingN/A

Sponsor: AstraZeneca

Conditions: Neurofibroma, Plexiform

Countries: China

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 overview

Sex: ALL

Age: 3 Years to 16 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

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.

Locations (25)

Beijing, Beijing Municipality, China
Chongqing, Chongqing Municipality, China
Lanzhou, Gansu, China
Guangzhou, Guangdong, China
Shenzhen, Guangdong, China
Nanning, Guangxi, China
Guiyang, Guizhou, China
Shijiazhuang, Hebei, China
Zhengzhou, Henan, China
Wuhan, Hubei, China
Changsha, Hunan, China
Nanjing, Jiangsu, China
Suzhou, Jiangsu, China
Nanchang, Jiangxi, China
Shenyang, Liaoning, China
Jinan, Shandong, China
Shanghai, Shanghai Municipality, China
Taiyuan, Shanxi, China
Xi’an, Shanxi, China
Chengdu, Sichuan, China
Tianjin, Tianjin Municipality, China
Kunming, Yunnan, China
Hangzhou, Zhejiang, China
Wenzhou, Zhejiang, China
Nanjing, China