Actively Recruiting
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Led by National Cancer Institute (NCI) · Updated on 2026-04-24
55
Participants Needed
1
Research Sites
422 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must have a clinical diagnosis of NF1 confirmed by at least two diagnostic criteria or a confirmed NF1 mutation
- Participants must have at least one measurable atypical neurofibroma (ANF) of 3 cm or larger confirmed by biopsy
- Participants aged 12 years or older with body surface area (BSA) of at least 0.71 m2 and able to swallow whole tablets
- Willingness of participants aged 12 to under 18 to undergo pretreatment biopsy of ANF if feasible
- Willingness of participants aged 18 or older to undergo pre-treatment and on-treatment biopsies of ANF if feasible
- Participants who have recovered from prior chemotherapy, radiotherapy, or major surgery with required washout periods
- Adequate blood counts and organ function as defined by study criteria
- Willingness to avoid grapefruit or grapefruit juice during treatment
- Ability to understand and sign informed consent or have a legally authorized representative do so
- Women of childbearing potential and men must agree to use effective contraception during treatment and for 4 months after
You will not qualify if you...
- Pregnant or intending to become pregnant women and breastfeeding women
- Participants with NF1-related tumors requiring chemotherapy or surgery such as optic pathway glioma or malignant peripheral nerve sheath tumor
- Serious medical conditions that would prevent participation, including severe lung disease, gastrointestinal conditions affecting absorption, or chronic diarrhea
- Uncontrolled illnesses such as heart failure, unstable angina, arrhythmias, active bleeding, or psychiatric/social conditions limiting compliance
- History of certain heart conditions like syncope of cardiovascular cause or ventricular arrhythmia
- Active bacterial, fungal, or viral infections requiring intravenous antibiotics or known active hepatitis B or C
- Interstitial lung disease
- Need for treatment with strong CYP3A inhibitors or inducers
- Inability to swallow tablets or undergo MRI
- Severe nausea or vomiting limiting drug intake
- Known severe allergy to abemaciclib or related compounds
- Investigator judgment that participation is not in the participant's best interest
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
A
Amanda M Carbonell
CONTACT
B
Brigitte C Widemann, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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