This study is currently recruiting participants.
National Cancer Institute (NCI)
Type: Participants currently recruited/enrolled Gender: Male & Female Min Age: 18 Years Max Age: 120 Years
Referral Letter Required
Mek1/2 Inhibitor; Neurofibromatosis Type 1; Neurofibromin; Autosomal Dominant Genetic Disorder; Raf-Mek-Erk Pathway
Drug: Placebo Drug: Selumetinib
National Cancer Institute
To test the effectiveness of a study drug (Selumetinib) against PNs and other symptoms.
People aged 18 years or older diagnosed with NF1 and inoperable PNs.
Participants will undergo screening tests. They will have a physical exam. They will have blood and urine tests and tests of their heart function. They will have imaging scans and an eye exam.
Participants will use a handheld device to complete an e-Diary every day. They will record: the level of pain they are feeling, any drugs they took for the pain, and whether they took the study drug on time. Use of the e-Diary will begin during screening and last to the end of the study.
Participants will take Selumetinib or a placebo (dummy tablet) twice per day in 28-day cycles. They will take the drugs by mouth, at home. After cycle 8, all participants will take Selumetinib.
Participants will come to the clinic on day 1 of cycles 1, 2, 4, and 6 and then once every 6 cycles. They will have additional screening tests during these visits. They will have at least 15 study visits.
Participants may give a blood sample for DNA testing.
Participant partners who become pregnant may be asked to answer questions. They will be asked about the pregnancy, delivery, and the baby s health.
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ELIGIBILITY: -Patients must be 18 years of age with symptomatic, inoperable PN.
-Patients must be 18 years of age with symptomatic, inoperable PN.
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