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MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

NCT02407405 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2026-03-05

No results posted yet for this study

Summary

Background:

Neurofibromatosis type 1 (NF1) is a disorder that can cause plexiform neurofibromas (PNs). These are tumors that grow along nerves. Some PNs cause serious health problems. PNs often can t be operated on because of their large size, location, or number. There are no effective treatments known for people with NF1 and PNs. Researchers want to test if the drug selumetinib (AZD6244 hydrogen sulfate) causes PNs to shrink or slows down their growth.

Objectives:

To test if selumetinib helps treat PNs. To test how the body handles selumetinib and how it affects peoples symptoms.

Eligibility:

People ages 18 and older with NF1, with an inoperable PN that causes morbidity or is growing

Design:

Participants will be screened with:

Medical history and physical exam

Blood, urine, and heart tests

Eye exam

MRI: They lie in a machine that takes pictures of the body.

PN biopsy: A small piece of the tumor is removed by a large needle.

Questionnaires

Participants will swallow selumetinib capsules every 12 hours for several 28-day cycles. The capsules are taken with a full glass of water on an empty stomach. Participants may have only water for 2 hours before and 1 hour after each dose.

Participants will keep a drug diary. They will continue taking the drug as long as they tolerate it and their disease doesn t progress.

Participants will have several visits throughout the study. These will include repeats of the screening tests.

Participants will have a final visit after they stop taking selumetinib.

Conditions

  • Neurofibromatosis 1 (NF1)
  • Plexiform Neurofibromas (PN)

Interventions

DRUG

Selumetinib

orally 50 mg/dose, every 12 hours every day continuously (1 cycle = 28 days)

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

  • Brigitte C Widemann, M.D. · National Cancer Institute (NCI)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-07
Primary Completion
2028-01-01
Completion
2030-01-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02407405 on ClinicalTrials.gov