Efficacy and Safety of Selumetinib in Adults with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas.
- Conditions
- eurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN).Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2023-507336-20-00
- Lead Sponsor
- Astrazeneca AB
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 145
'- Adults = 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN - At least one 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'
'- Confirmed or suspected malignant glioma or MPNST (low grade glioma, including optic glioma not requiring systemic therapy 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 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'
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method