Status:
ACTIVE_NOT_RECRUITING
Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs
Lead Sponsor:
AstraZeneca
Conditions:
Neurofibroma, Plexiform
Eligibility:
All Genders
3-16 years
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...
Detailed Description
Neurofibromatosis type 1 (NF1) is a rare and incurable autosomal dominant genetic disorder that is characterized by manifestations in multiple organ systems including tumors of the nervous system. Alt...
Eligibility Criteria
Inclusion
- (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
- (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.
Key Trial Info
Start Date :
December 20 2023
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 30 2027
Estimated Enrollment :
409 Patients enrolled
Trial Details
Trial ID
NCT06175637
Start Date
December 20 2023
End Date
May 30 2027
Last Update
October 22 2025
Active Locations (25)
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1
Research Site
Beijing, Beijing Municipality, China
2
Research Site
Chongqing, Chongqing Municipality, China
3
Research Site
Lanzhou, Gansu, China
4
Research Site
Guangzhou, Guangdong, China