Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With N… (NCT06175637) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs
China409 participantsStarted 2023-12-20
Plain-language 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
Who can participate
Age range
3 Years – 16 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is an observational study that is no longer enrolling new patients, is there another active trial or approved treatment pathway involving selumetinib that might be worth exploring for my child's NF1-related plexiform neurofibromas?
2This study is tracking real-world safety and effectiveness of selumetinib in pediatric patients — based on what has been collected so far, what do doctors generally know about the risks and benefits of selumetinib for children with plexiform neurofibromas like my child's?
3Because this study collects information on disease characteristics at baseline and over time, could my child's current disease measurements — like tumor size or symptom burden — help us understand how their condition compares to what's being tracked in studies like this one?
4The study includes a physician's qualitative assessment of overall NF1 disease status, not just the plexiform neurofibroma itself — how do you evaluate my child's broader NF1 condition, and should that factor into any treatment decisions we're considering right now?
5Since selumetinib is already being used in pediatric NF1 patients outside of this observational study, is standard treatment with selumetinib something my child could access now, and how would that compare to waiting for findings from studies like this one?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
NF1-PN patient demographics and disease characteristics at baseline and during selumetinib treatment
Timeframe: From baseline to 24 months after first dose of selumetinib
2
NF1-PN disease characteristics
Timeframe: From baseline to 24 months after first dose of selumetinib
3
Selumetinib treatment profile
Timeframe: From baseline to 24 months after first dose of selumetinib
4
Physician's qualitative assessment of disease status for clinically significant PN and overall disease status
Timeframe: From baseline to 24 months after first dose of selumetinib
5
NF1 disease status
Timeframe: From baseline to 24 months after first dose of selumetinib