Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)
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|ClinicalTrials.gov Identifier: NCT03109301|
Recruitment Status : Withdrawn (slow accrual)
First Posted : April 12, 2017
Last Update Posted : April 5, 2019
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.
To find out if selumetinib shrinks or slows the growth of GIST tumors and to see its side effects.
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.
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:
Blood and urine tests
Scans of their tumors
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.
|Condition or disease||Intervention/treatment||Phase|
|Neoplasms, Nerve Tissue Neurofibromatosis 1 Heredodegenerative Disorders, Nervous System Peripheral Nervous System Diseases||Drug: Selumetinib (AZD6244 hyd sulfate) 50mg/dose Drug: Selumetinib (AZD6244 hyd sulfate) 25mg/m2||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of the Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Patients With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)|
|Actual Study Start Date :||April 7, 2017|
|Actual Primary Completion Date :||March 27, 2019|
|Actual Study Completion Date :||March 27, 2019|
Experimental: Arm 1
Patients > 18 years of age
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.
Experimental: Arm 2
Patients < 18 years of age
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.
- Efficacy [ Time Frame: End of treatment ]Estimate the response rate (radiologic response RECIST v1.1) of selumetinib in children and adults with NF1- mutated measurable gastrointestinal stromal tumor (GIST).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03109301
|Principal Investigator:||Brigitte C Widemann, M.D.||National Cancer Institute (NCI)|