Selumetinib for NF2-Related Schwannomatosis (NCT07707947) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Selumetinib for NF2-Related Schwannomatosis
China20 participantsStarted 2026-07-15
Plain-language summary
The goal of this clinical trial is to evaluate the efficacy and safety of the MEK1/2 inhibitor selumetinib in treating patients with neurofibromatosis type 2-related schwannomatosis (NF2-SWN), including both adults and children with inoperable or progressive tumors.
Who can participate
Age range
3 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
. Patients must have a pathogenic variant in the NF2 gene (either in the germline or in two NF2-related tumors)OR a confirmed diagnosis of NF2 by fulfilling National Institute of Health (NIH)criteria or Manchester criteria:
. Subjects must have ≥1 measurable target vestibular schwannoma meeting all of the following conditions:
. The target tumor must be considered not amenable to surgery due to:
. Male or female subjects aged ≥3 years at the time of informed consent.
. Adequate functional status
. Laboratory values obtained within 28 days prior to enrollment must meet the following:
. Hematologic function
. Hepatic function
Exclusion criteria
. Concurrent involvement in study conduct:the subject is an employee of the Sponsor, Investigator, or study site who is directly involved in the planning, conduct, or management of this clinical study.
. Participation in another interventional clinical trial with an investigational medicinal product, or receipt of an investigational agent within 28 days (or 5 half-lives if known and longer)prior to first dose.
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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
Objective Response Rate (ORR)in NF2-related vestibular schwannoma assessed according to the REiNS criteria
Timeframe: 12 months
2
Hearing Response Rate Based on Word Recognition Score (WRS) in Target Vestibular Schwannoma
Timeframe: 12 months
3
Pure Tone Average (PTA) Response Rate in Target Vestibular Schwannoma
. Prior anti-cancer systemic therapies or prior radiotherapy that, in the investigator's judgment, would confound safety or efficacy assessment, unless completed ≥28 days prior to first dose (longer washout required for agents with prolonged biologic effect as specified in protocol appendix). Subjects who received prior local therapy (surgery or localized radiotherapy)are eligible if recovery is complete and target lesion remains measurable.
. Evidence or high suspicion of malignant peripheral nerve sheath tumor (MPNST)or other active malignancy requiring systemic therapy (past malignancy is allowed only if disease-free for ≥2 years, except for adequately treated basal cell carcinoma or in situ carcinoma).
. Significant cardiac disease or ECG abnormalities:
. Uncontrolled hypertension:systolic ≥140 mmHg or diastolic ≥90 mmHg despite optimal therapy (adult criteria);for pediatric subjects use age/height/gender percentiles per protocol appendix.
. Severe hepatic or renal impairment -e.g.,AST/ALT \>5 ×ULN (or per protocol specified threshold for severe impairment), total bilirubin \>3 ×ULN (unless Gilbert's syndrome).(See inclusion for minimum acceptable labs;exclude those with more severe dysfunction.)
. Ophthalmologic conditions:current or prior history of MEK-associated retinopathy / central serous retinopathy /retinal pigment epithelial detachment /retinal vein occlusion, or any active ocular condition judged by the investigator/ophthalmologist to increase the risk of serious ocular adverse events (e.g., uncontrolled glaucoma with elevated IOP and meaningful vision at risk). Subjects with stable, chronic ophthalmic findings that are not expected to worsen with MEK inhibition may be eligible after ophthalmology clearance.