Abemaciclib for Neurofibroma
What You Need to Know Before You Apply
What is the purpose of this trial?
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 surgeryDesign:Participants will be screened with:Medical history and physical examBlood, urine, and heart testsMRI: 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 lifePossible photographs of tumorsParticipants 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.
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop taking your current medications. However, you cannot participate if you require treatment with strong CYP3A inhibitors or inducers, as these can interact with the study drug, abemaciclib.
How is the drug Abemaciclib different from other treatments for neurofibroma?
Abemaciclib is unique because it is a CDK4/6 inhibitor, which means it targets specific proteins involved in cell division, potentially offering a novel approach for treating neurofibromas compared to other treatments like MEK inhibitors or tyrosine kinase inhibitors that have been used for similar conditions.12345
Who Is on the Research Team?
Andrea M Gross, M.D.
Principal Investigator
National Cancer Institute (NCI)
Are You a Good Fit for This Trial?
This trial is for individuals aged 12 or older with Neurofibromatosis Type I (NF1) and at least one inoperable tumor measuring over 3 cm. Participants must not be pregnant, agree to use effective contraception, have no severe allergies to abemaciclib, and should not require certain other medications that could interfere with the study drug.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Phase 0
Participants receive abemaciclib for 7-10 days prior to tumor resection to determine pharmacodynamic effects
Phase I/II Treatment
Participants receive abemaciclib orally twice daily in 28-day cycles to determine the recommended Phase II dose and objective response rate
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Abemaciclib
Abemaciclib is already approved in United States, European Union for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Cancer Institute (NCI)
Lead Sponsor