Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas (NF108-BINI)
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|ClinicalTrials.gov Identifier: NCT03231306|
Recruitment Status : Recruiting
First Posted : July 27, 2017
Last Update Posted : December 23, 2019
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis Type 1 Plexiform Neurofibroma||Drug: Binimetinib||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study of Binimetinib in Children and Adults With NF1 Associated Plexiform Neurofibromas (PNOC010)|
|Actual Study Start Date :||November 28, 2017|
|Estimated Primary Completion Date :||December 31, 2022|
|Estimated Study Completion Date :||December 31, 2023|
Experimental: Open label study of Binimetinib (MEK162)
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
- Change from Baseline Target Tumor Volume at 12 months [ Time Frame: Approximately 12 months ]To determine the objective response defined as 20% or greater tumor volume reduction. Patients will undergo volumetric assays of their target PN using MRI.
- Incidence of Treatment-Emergent Adverse Events [ Time Frame: Up to 24 months ]To evaluate the toxicity of protracted binimetinib administration in this patient population. Subjects will be monitored continuously for adverse events and serious adverse events throughout the study.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03231306
|Contact: Sabine Mueller, MD, PhDfirstname.lastname@example.org|
|Contact: Karen C Plourde, BSemail@example.com|
|Study Chair:||Bruce Korf, MD, PhD||University of Alabama at Birmingham|