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

Sponsor
National Cancer Institute (NCI)
Study ID
NCT03109301
Phase
PHASE2
Status
Withdrawn

Conditions

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

Eligibility Criteria

Sex
ALL
Age
3 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Selumetinib (AZD6244 hyd sulfate) 50mg/dose — DRUG
    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.
  • Selumetinib (AZD6244 hyd sulfate) 25mg/m2 — DRUG
    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.

Study Details

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.

Key Dates

Start date
Apr 7, 2017
Status verified
Mar 2019
Primary completion
Mar 27, 2019
Completion
Mar 27, 2019

Study Design

Enrollment
0 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Arm 1
    Patients \> 18 years of age
  • Experimental: Arm 2
    Patients \< 18 years of age

Primary Outcome Measure

Efficacy [ Time Frame: End of treatment ]

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