AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Status:
Active, not recruiting
Trial end date:
2030-01-01
Target enrollment:
Participant gender:
Summary
Background:
- Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat
them is with surgery. Some of these tumors cannot be completely removed. The tumors may be
too large, too numerous, or in a bad location for surgery. An experimental drug called
AZD6244 hydrogen sulfate may be able to prevent the tumors from growing, slow down their
growth, or shrink them. This drug has been tested in adults with cancer and in children with
some types of brain cancer. This study will test how well this drug works with these types of
tumors.
Objectives:
- To study the safety and effectiveness of AZD6244 hydrogen sulfate in children and young
adults with plexiform neurofibromas that cannot be completely removed by surgery.
Eligibility:
- Children and young adults between 12 and 18 years of age who have plexiform neurofibromas
that cannot be completely removed by surgery.
Design:
- Patients will be screened with a physical exam, medical history, blood tests, and
imaging studies.
- They will take the study drug twice a day with 8 ounces of water, every day for 28-day
cycles of treatment. During study visits, participants will have blood and urine tests
and physical exams. They will also have imaging studies to examine the tumor sizes and
locations. They will answer questions about their health. They may have other tests as
needed.
- Participants will continue to receive the study drug as long as they have no severe side
effects and the disease is not getting worse.