Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

• Status: Recruiting
• Trial end date: 2024-11-22
• Target enrollment: 0
• Participant gender: All

Summary

A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AstraZeneca
• Collaborator: Merck Sharp & Dohme Corp.

Criteria

Key Inclusion Criteria:

• Adults ≥ 18 years 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

Key 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 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