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Clinical Trials in Spain / NCT04924608
Active, not recruiting Phase 3

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

NCT04924608 · tracked via the Priya Life Science Spain tracker
Sponsor
AstraZeneca
Phase
Phase 3
Started
2021-11-19
Last updated
2026-03-23

Condition(s) studied

Neurofibromatosis 1Plexiform Neurofibroma (PN)

Investigational drug(s) / intervention(s)

Selumetinib →Placebo

Selumetinib: Selumetinib oral capsules (10 mg and 25 mg)

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

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN. — From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days)
    Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of Cycle 16 as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD.

Trial sites (34)

FacilityCityRegionStatus
Research Site Gainesville Florida
Research Site Rockville Maryland
Research Site St Louis Missouri
Research Site Commack New York
Research Site Melbourne Australia
Research Site St Leonards Australia
Research Site Porto Alegre Brazil
Research Site Ribeirão Preto Brazil
Research Site São Paulo Brazil
Research Site Toronto Ontario
Research Site Montreal Quebec
Research Site Beijing China
Research Site Beijing China
Research Site Guangzhou China
Research Site Shenyang China
Research Site Créteil France
Research Site Lyon France
Research Site Toulouse France
Research Site Hamburg Germany
Research Site Tübingen Germany
Research Site Würzburg Germany
Research Site Milan Italy
Research Site Naples Italy
Research Site Roma Italy
Research Site Minatoku Japan
Research Site Nagoya Japan
Research Site Shinjuku-ku Japan
Research Site Bydgoszcz Poland
Research Site Moscow Russia
Research Site Moscow Russia
Research Site Badalona Spain
Research Site Madrid Spain
Research Site London United Kingdom
Research Site Manchester United Kingdom

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Other trials for the same condition

Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04924608 on ClinicalTrials.gov ↗ ← All trials in Spain