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A Phase III, Multicentre, International Study With a Parallel, Randomised, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

Phase 3
Conditions
eurofibromatosis 1, Plexiform Neurofibroma (PN)
Registration Number
JPRN-jRCT2041210036
Lead Sponsor
Kobayashi Hiroshi
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
15
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

- 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

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Confirmed Objective Response Rate (ORR) for Arm A [ Time Frame: Approximately 3 years ] <br>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
Secondary Outcome Measures
NameTimeMethod
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