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

AstraZeneca

Status and phase: Active, not recruiting; Phase 3

Conditions: Neurofibromatosis 1; Plexiform Neurofibroma (PN)

Treatments: Other: Placebo; Drug: Selumetinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT04924608

D134BC00001

2020-005607-39 (EudraCT Number)

Details and patient eligibility

About

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

Full description

This is a randomized, double-blind, placebo-controlled, 2 arm multicentre, global Phase III study to assess the efficacy and safety of selumetinib compared with placebo in adult participants with NF1 who have symptomatic, inoperable PN.

Enrollment: 146 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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 (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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

146 participants in 2 patient groups, including a placebo group

Arm A

Experimental group

Description:

Selumetinib

Treatment:

Drug: Selumetinib

Arm B

Placebo Comparator group

Description:

Placebo

Treatment:

Other: Placebo