Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Status:
Recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
Background:
NF1 is a genetic disease that causes tumors called atypical neurofibromas. These tumors,
which arise from nerves, can cause serious medical problems. The only treatment is surgery.
Researchers want to see if a drug called abemaciclib can help.
Objective:
To find a safe, tolerable dose of abemaciclib for treating atypical neurofibromas.
Eligibility:
People ages 12 and older who have NF1 and have one or more atypical neurofibromas that cannot
or will not be removed with surgery
Design:
Participants will be screened with:
Medical history and physical exam
Blood, urine, and heart tests
MRI: Participants will lie in a machine that takes pictures of the body. A padding or coil
will be placed around their head. They may have a contrast agent injected into a vein.
Biopsy sample: A small piece of tumor will be removed using a large needle.
Participants will have frequent visits during the study. These will include repeats of the
screening tests as well as the following:
PET scan: Participants will lie in a machine that takes pictures of the body. They will have
a contrast agent injected into their arm.
Questionnaires about the effects of abemaciclib on pain and quality of life
Possible photographs of tumors
Participants will take abemaciclib capsules orally twice daily in 28-day cycles. They will
take the drug for up to 2 years. Some may be able to take it for longer.
Participants will have a follow-up visit about 30 days after their last dose of the study
drug. Then they will have visits every 3 months for 1 year.
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