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

Part of paid clinical trials in Gainesville, Florida.

Sponsor
AstraZeneca
Study ID
NCT04924608
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Neurofibromatosis 1
  • Plexiform Neurofibroma (PN)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Selumetinib — DRUG
    Selumetinib oral capsules (10 mg and 25 mg)
  • Placebo — OTHER
    Placebo oral capsules for Selumetinib masking (10 mg and 25 mg)

Study Details

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

Key Dates

Start date
Nov 19, 2021
Status verified
Mar 2026
Primary completion
Aug 5, 2024
Completion
Feb 15, 2029

Study Design

Enrollment
145 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Arm A
    Selumetinib
  • Placebo Comparator: Arm B
    Placebo

Primary Outcome Measure

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. [ Time Frame: 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) ]

Locations (4)

FacilityCityStateZIPSite coordinators
Research SiteGainesvilleFlorida32610-
Research SiteRockvilleMaryland20852-
Research SiteSt LouisMissouri63156-
Research SiteCommackNew York11725-

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By research site
Research Site· Gainesville, FLResearch Site· Rockville, MDResearch Site· St Louis, MOResearch Site· Commack, NY

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