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Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)

NCT03109301 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2019-04-05

No results posted yet for this study

Summary

Background:

Gastrointestinal stromal tumors (GIST) can cause serious medical problems. The only known treatment is surgery. But completely removing a GIST tumor with surgery is often not possible. Researchers want to see if a new drug, selumetinib, can help treat these tumors.

Objective:

To find out if selumetinib shrinks or slows the growth of GIST tumors and to see its side effects.

Eligibility:

People ages 3 and over who have one or more GIST tumors and may have neurofibromatosis type I (also called NF1). Their NF1 GIST has shown some growth or cannot be completely removed with surgery.

Design:

Participants will be screened with heart and eye tests and scans.

Participants will be told what foods and medicines they cannot take during the study. Participants will keep a diary of the medicine they take during the study.

Participants will take selumetinib capsules twice daily on an empty stomach for 28 days in a row. This is 1 cycle.

During the cycles, participants will have study visits. These may include:

Medical history

Physical exam

Blood and urine tests

Heart tests

Scans of their tumors

Eye exam

Positron emission tomography scan. They will be get radioactive glucose an IV line. They will lie quietly in a darkened room for 50-60 minutes then have the scan.

Participants will answer questions about how they are feeling.

Participants can stay in the study until they have bad side effects or their tumor grows.

After finishing treatment, participants will be watched for side effects for 30 days.

Conditions

  • Neoplasms, Nerve Tissue
  • Neurofibromatosis 1
  • Heredodegenerative Disorders, Nervous System
  • Peripheral Nervous System Diseases

Interventions

DRUG

Selumetinib (AZD6244 hyd sulfate) 50mg/dose

Selumetinib will be administered at a starting dose of 50 mg/dose orally in patients 18 years or older; drug will be given twice daily continuously in the absence of toxicity or disease progression, using 28-day cycles.

DRUG

Selumetinib (AZD6244 hyd sulfate) 25mg/m2

Selumetinib will be administered at a starting dose of 25 mg/m2/dose in children \< 18 years of age; drug will be given twice daily continuously in the absence of toxicity or disease progression, using 28-day cycles.

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

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

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-04-07
Primary Completion
2019-03-27
Completion
2019-03-27
FDA Drug
Yes

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 NCT03109301 on ClinicalTrials.gov