A Phase 2 interventional study of Selumetinib 25mg/m2 and Luvometinib in Neurofibromatosis Type 2, Vestibular Schwannoma and Non-vestibular Schwannoma, sponsored by Beijing Tiantan Hospital. Not yet recruiting at 5 sites in China. Per ClinicalTrials.gov, last updated 2026-07-20.
Sponsored by Beijing Tiantan Hospital · Phase 2, Interventional, and Treatment
This is an adaptive platform-basket trial that aims to evaluate the safety and efficacy of multiple novel agents and combination therapies in patients with NF2-related schwannomatosis (NF2-SWN). The study employs a basket design to assess treatment responses across four tumor types commonly associated with NF2-SWN: vestibular schwannomas, non-vestibular schwannomas, meningiomas, and ependymomas.
A shared natural history observational cohort, receiving routine clinical follow-up without investigational treatment, serves as a common control for all substudies. The adaptive platform enables the dynamic addition or closure of substudies based on interim analyses, thereby optimizing trial efficiency.
Eligible patients who meet the master protocol criteria and satisfy substudy-specific safety requirements will be assigned to receive the corresponding intervention. Currently open substudies include:
This is an investigator-initiated, prospective, multicenter, adaptive platform-basket clinical trial designed to evaluate the safety and efficacy of multiple therapies in patients with NF2-related schwannomatosis (NF2-SWN). The study includes four tumor baskets: vestibular schwannoma, meningioma, non-vestibular schwannoma, and ependymoma.
MASTER STUDY All patients with a confirmed diagnosis of NF2-SWN who provide written informed consent will be enrolled in the master study and enter the natural history observational cohort. Patients who meet eligibility criteria for one or more active substudies may be assigned to a corresponding treatment arm. When multiple treatment arms are open, allocation will follow a predefined randomization scheme. When only one treatment arm is available, eligible patients may be enrolled directly into that substudy. Patients not eligible for any active intervention will remain in the master study cohort for standardized follow-up.
Patients who experience progression of the target tumor during substudy treatment may be considered for enrollment into another active treatment arm if eligibility criteria are met. Patients who are not eligible for any active substudy will return to the master study observational cohort. Data collected during follow-up may serve as shared control data across the platform.
Patients in the observational cohort will undergo standardized follow-up assessments every 12 months until study completion or voluntary withdrawal. Patients receiving treatment within a substudy will undergo efficacy and safety assessments approximately every 3 months according to the corresponding substudy protocol. The master study plans to enroll at least 200 patients with NF2-SWN, with enrollment continuing over time as eligible patients are identified across participating centers.
SUBSTUDY 1: Selumetinib
SUBSTUDY 2: Luvometinib + Serplulimab
Each substudy protocol will be incorporated into the master protocol as an appendix. Addition of new substudies requires review and approval by the Data and Safety Monitoring Board (DSMB).
Efficacy will be evaluated using tumor-specific endpoints. For vestibular schwannoma, the primary endpoint is Hearing Response Rate (HRR). For meningioma, non-vestibular schwannoma, and ependymoma, the primary endpoint is radiographic Objective Response Rate (ORR). All efficacy endpoints will undergo blinded central review by an Independent Review Committee (IRC).
The platform is expected to remain active for 5-10 years, or until all active substudies are completed and no additional treatment arms are planned.
47 studies on the registry are indexed under Neurofibromatosis 2; 10 are open to participants now.
This study's planned enrollment of 200 is above the median of 21 across 36 interventional studies indexed under Neurofibromatosis 2.
Browse Neurofibromatosis 2 studies →Beijing Tiantan Hospital is the lead sponsor of 489 studies on the registry; 293 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Eligibility Specific For MASTER STUDY
Inclusion Criteria:
Subjects must satisfy all of the following criteria to be enrolled into the main study natural history observation cohort:
(1) Must meet the 2022 International Consensus Criteria for NF2-SWN, defined by having at least one of the following:
Major criteria:
Minor criteria:
Can count >1 of a type (eg, 2 distinct schwannomas would count as 2 minor criteria)
Juvenile subcapsular or cortical cataract, retinal hamartoma, epiretinal membrane in a person aged \<40 years, meningioma
(2) Presence of at least one evaluable lesion: Vestibular schwannoma, meningioma, or non-vestibular schwannoma: clearly identifiable lesion on contrast-enhanced T1-weighted MRI; Ependymoma: clearly identifiable lesion on contrast-enhanced T1-weighted or T2/FLAIR sequences; (3) Expected ability to complete at least 12 months of follow-up assessments; (4) Ability to understand and voluntarily sign a written form, or a legally authorized guardian signs the informed consent form together ; (5) Sub-study-specific criteria (for intervention arms only): If the subject intends to enter an interventional sub-study, in addition to meeting the above main study criteria, the subject must also satisfy the specific inclusion criteria specified in that sub-study protocol (e.g., organ function, prior treatment restrictions, , etc.), as determined by the drug characteristics.
Exclusion Criteria:
Subjects meeting any of the following criteria will not be permitted to enter the main study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects will receive selumetinib 25 mg/m² by mouth twice daily (single dose not to exceed 50 mg) for up to 12 cycles (28 days per cycle).
Drug: Selumetinib 25mg/m2
Subjects will receive luvometinib 8 mg by mouth once daily in combination with serplulimab 4.5 mg/kg intravenously every 3 weeks for up to 12 cycles (28 days per cycle).
Drug: Luvometinib · Drug: Serplulimab
Participants who are not eligible for any active treatment substudy, or who choose not to receive investigational therapy, will remain in the Natural History Observation Arm. No study intervention will be administered. Participants will undergo standardized longitudinal clinical, imaging, and outcome assessments according to the master protocol. Data collected from this arm will be used to characterize the natural history of NF2-related schwannomatosis and will serve as a shared observational comparator for active treatment arms within the platform.
Drug page: AZD 6244
Oral twice daily per predetermined dosage per protocol.
Also known as: Koselugo, AZD6244
Oral once daily per predetermined dosage per protocol.
Also known as: FCN-159
Intravenous infusion per predetermined dosage per protocol.
Also known as: HLX10
Tumor-Type-Specific Response Rate in NF2-SWN Tumors
Vestibular schwannoma: HRR is defined as WRS improvement exceeding the 95% critical difference from baseline; if baseline WRS is \<20%, HRR is defined as a PTA decrease of at least 10 dB. Meningioma or non-vestibular schwannoma: ORR is defined as at least a 20% reduction in target tumor volume from baseline. Ependymoma: ORR is defined as at least a 30% reduction in maximum diameter from baseline according to RECIST v1.1.
Time frame: 12 months
Incidence of Adverse Events in Interventional Substudies
Percentage of participants receiving active treatment who experience at least one adverse event. Adverse events will be coded and graded according to NCI CTCAE v5.0.
Time frame: From first dose through 30 days after last dose (or as specified by individual substudy protocols)
Maximum Severity Grade of Adverse Events in Interventional Substudies
Maximum NCI CTCAE v5.0 grade of adverse events experienced by each participant during the reporting period.
Time frame: 12 months
Incidence of Serious Adverse Events in Interventional Substudies
Percentage of participants receiving active treatment who experience at least one serious adverse event.
Time frame: From first dose through 30 days after last dose.
Incidence of Dose Modifications Due to Adverse Events
Percentage of participants receiving active treatment who require at least one dose modification due to an adverse event.
Time frame: From first dose through 30 days after last dose.
Incidence of Treatment Interruptions Due to Adverse Events
Percentage of participants receiving active treatment who require at least one treatment interruption due to an adverse event.
Time frame: From first dose through 30 days after last dose.
Incidence of Treatment Discontinuations Due to Adverse Events
Percentage of participants receiving active treatment who discontinue treatment due to an adverse event.
Time frame: From first dose through 30 days after last dose.
Change From Baseline in MRI-Based Total Tumor Burden (TTB)
TTB will be assessed using serial MRI. TTB is calculated as the sum of the volumes of all prespecified measurable NF2-SWN-related tumors included in the tumor-burden assessment. The outcome is the relative percentage change in TTB from baseline to 12 months, based on blinded central radiology review.
Time frame: 12 months
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Jul 2026; primary completion for Jun 2036, 118 months later.
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Studies of the drugs tested here: AZD 6244 · Luvometinib