Neurofibromatosis 1, Plexiform Neurofibroma (PN)
Conditions
Interventions
Arm A: selumetinib 25 mg/m2 orally bid
Arm B: placebo orally bid
Sponsors
Kobayashi Hiroshi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Adults 18 years or more at enrollment with diagnosis of NF1 with symptomatic, inoperable PN - At least one inoperable target PN measurable by volumetric MRI analysis - Chronic target PN pain score documented for minimum period during screening period - Stable chronic PN pain medication use at enrollment - Adequate organ and marrow function
Exclusion criteria
Exclusion criteria: - Confirmed or suspected malignant glioma or MPNST (optic glioma not requiring chemotherapy or radiation therapy are exempt from this exclusion) - History of malignancy except for malignancy treated with curative intent with no known active disease 5 years or more before the first dose of study intervention and of low potential risk for recurrence - Clinically significant cardiovascular disease, including inherited coronary disease, acute coronary syndrome within 6 months prior to enrollment, uncontrolled angina, symptomatic heart failure, cardiomyopathy, severe valvular heart disease, abnormal LVEF and uncontrolled hypertension - Ophthalmological findings/conditions including intraocular pressure > 21 mmHg, RPED/CSR or RVO - Prior exposure to MEK inhibitors
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Confirmed Objective Response Rate (ORR) for Arm A [ Time Frame: Approximately 3 years ] ORR will be defined as the proportion of patients who have a confirmed complete response or confirmed partial response as determined by ICR per REiNS criteria | — |
Countries
Australia, Brasil, Canada, China, France, Germany, Italy, Japan, Poland, Russia, Spain, UK, USA
Contacts
Public ContactHiroshi Kobayashi
Alexion Pharma GK
Outcome results
None listed