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

Study Results

Results available

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

145 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-11-19

Study Completion Date

2029-02-15

Brief Summary

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

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Detailed Description

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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.

Conditions

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Neurofibromatosis 1 Plexiform Neurofibroma (PN)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Arm A

Selumetinib

Group Type EXPERIMENTAL

Selumetinib

Intervention Type DRUG

Selumetinib oral capsules (10 mg and 25 mg)

Arm B

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo oral capsules for Selumetinib masking (10 mg and 25 mg)

Interventions

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Selumetinib

Selumetinib oral capsules (10 mg and 25 mg)

Intervention Type DRUG

Placebo

Placebo oral capsules for Selumetinib masking (10 mg and 25 mg)

Intervention Type OTHER

Other Intervention Names

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AZD6244

Eligibility Criteria

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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

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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No