A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosi… (NCT03871257) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
United States, Canada, Puerto Rico165 participantsStarted 2020-01-15
Plain-language summary
This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Chemotherapy drugs, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether selumetinib works better in treating patients with NF1-associated low-grade glioma compared to standard therapy with carboplatin and vincristine.
Who can participate
Age range
2 Years – 21 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 be \>= 2 years and =\< 21 years at the time of enrollment
* Patients must have a body surface area (BSA) of \>= 0.5 m\^2 at enrollment
* Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing
* Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery
* For patients with optic pathway gliomas (OPGs):
* Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms (including visual dysfunction, as defined below) or other exam findings associated with the tumor
* Previously-diagnosed patients with OPG are eligible if they have new or worsening neurologic symptoms (including visual dysfunction, as defined below) or have tumor growth
* For both newly-diagnosed and previously-diagnosed OPG, the patient may be eligible, irrespective of whether there has been tumor growth or other neurological symptoms or worsening, if they meet at least one of the following visual criteria:
* Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR
* Significant visual dysfunction (defined as VA worse than normal for age by 0.6 logMAR \[20/80, 6/24, or 2.5/10\] or more in one or both eyes)
* For patients with LGG in other locations (i.e., not OPGs):
* Newly-diagnosed patients with LGG are eligible i…
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.
1This is a Phase 3 trial comparing selumetinib to carboplatin and vincristine — since it's already in Phase 3, does that mean there's more safety and effectiveness data available on selumetinib than there would be for an earlier-phase study, and how does what's known so far compare to the standard chemotherapy option?
2The trial is actively enrolling patients but no longer recruiting — does that mean my child or I could still potentially join, or is it now completely closed, and if it's closed, are there other ways to access selumetinib?
3One of the main things this trial is measuring is whether vision actually improves — given that this trial involves a visual pathway glioma, how would my doctor track and measure any changes in my vision over the course of treatment, and what counts as a meaningful improvement?
4Since this trial involves neurofibromatosis type 1 specifically, how important is it that I be evaluated at a center that specializes in NF1, and would participating in or following this trial's approach change where I'd need to go for care?
5How does selumetinib's mechanism — targeting a specific pathway involved in NF1-related tumors — compare to the way carboplatin and vincristine work, and based on what's known from this trial so far, is one approach considered better for preserving vision in NF1-related low-grade glioma?
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
Event-free survival (EFS)
Timeframe: From randomization to the first occurrence of any of the following events: clinical or radiographic disease progression, disease recurrence, second malignant neoplasm, or death from any cause, assessed up to 3 years after accrual completion
2
Number of participants with visual acuity (VA) improvement per arm
Timeframe: Baseline and end of about 12 months of treatment