Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas (NCT03231306) | Clinical Trial Compass
CompletedPhase 2
Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas
United States45 participantsStarted 2017-11-28
Plain-language summary
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.
Who can participate
Age range1 Year
SexALL
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Inclusion Criteria
* Clinical diagnosis of NF1 using the NIH Consensus Conference criteria OR a documented constitutional NF1 mutation
* Plexiform neurofibroma(s) that are progressive or causing significant morbidity
* Presence of new plexiform neurofibroma on MRI or CT (documented by comparison with prior MRI or CT)
* Measurable plexiform neurofibroma(s) amenable to volumetric MRI analysis. Tumors must be at least 3 mL in volume (most PNs 3 cm in longest diameter will meet this criteria)
* Patients must be ≥ 18 years of age at the time of enrollment.
* Performance Level: Karnofsky or Lansky ≥ 50%. If unable to walk due to paralysis, but in a wheelchair, patients will be considered ambulatory for the purpose of assessing the performance level
* Ability to swallow capsules/tablets
* Ability to comply with follow up procedures
* The effects of binimetinib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and 3 months after completion of binimetinib administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
* Negative urine or serum β-HCG test (females of childbearing potential only).
Prior Therapy:
* Patients are eligible if complete resectio…
What they're measuring
1
Change From Baseline Target Tumor Volume at 12 Months