Actively Recruiting
A Phase 0/I/II Study of the CDK4/6 Inhibitor Abemaciclib for Neurofibromatosis Type 1 (NF1) Related Atypical Neurofibromas
Led by National Cancer Institute (NCI) · Updated on 2026-06-05
55
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors called atypical neurofibromas, which can lead to serious health problems. These tumors often cannot be treated except with surgery. Researchers are studying the drug abemaciclib to find a safe and tolerable dose for treating these tumors. This study is a Phase 0/I/II trial focused on people aged 12 and older with NF1 and measurable atypical neurofibromas that cannot be safely removed by surgery or who have multiple tumors. Participants will take abemaciclib capsules orally twice daily in 28-day cycles for up to 2 years or longer if eligible. The study includes a Phase 0 group that receives the drug before tumor removal surgery, and Phase I/II groups where dosing is adjusted to find the best dose and evaluate tumor response. Dose escalation and monitoring for side effects will guide treatment, with MRI scans used to measure tumor size and response. Some participants will have biopsies before and during treatment to study drug effects on the tumor. Participants will undergo screening tests including medical exams, blood, urine, heart tests, MRI scans with or without contrast, biopsies, and PET scans with contrast. Researchers will assess safety, drug effects on tumor growth and pain, quality of life, and drug levels in the body. Follow-up visits occur about 30 days after the last dose, then every 3 months for a year. The total participation time varies but includes frequent visits for monitoring and imaging throughout treatment and after stopping the drug.
CONDITIONS
Brief 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 Neurofibromatosis type 1 (NF1) with at least two diagnostic criteria or confirmed NF1 mutation
- Participants must have at least one measurable atypical neurofibroma (ANF) of at least 3 cm suitable for volumetric MRI analysis
- Prior therapies allowed if acute toxic effects have resolved as specified
- Adequate organ function including hematologic, hepatic, renal, and cardiac criteria
- Participants aged 12 years or older, able to swallow whole tablets, with body surface area \u2265 0.71 m2
- Willingness to undergo tumor biopsy as appropriate by age group
- Ability to avoid grapefruit or grapefruit juice during treatment
- Ability to understand and sign informed consent
- Women and men of childbearing potential must agree to use effective contraception during treatment and for 4 months after
You will not qualify if you...
- Pregnant or breastfeeding women
- Participants with NF1-related tumors that require chemotherapy or surgery such as optic pathway glioma or malignant peripheral nerve sheath tumor
- Serious medical conditions that would preclude participation (e.g., severe lung disease, severe diarrhea, major surgery without healing)
- Uncontrolled illnesses including heart failure, unstable angina, arrhythmias, bleeding disorders, psychiatric conditions limiting compliance
- Personal history of serious cardiac events such as syncope, ventricular arrhythmia, or sudden cardiac arrest
- Active infections requiring IV antibiotics or significant viral infections (HIV with low CD4 counts excluded)
- Interstitial lung disease
- Need for strong CYP3A inhibitors or inducers
- Inability to swallow tablets or undergo MRI
- Severe hypersensitivity to abemaciclib or related compounds
- Investigator judgment that participation is not appropriate
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years or until disease progression or unacceptable treatment-related toxicity
Participants receive abemaciclib orally twice daily in 28-day cycles to treat atypical neurofibromas associated with NF1. Dose escalation occurs in Phase I to determine the recommended dose, followed by treatment at the recommended dose in Phase II.
Restaging MRI visits pre-cycles 3 and 5, then every 4 cycles during the first year; regular toxicity monitoring visits
Duration - 30 days after treatment
Participants are monitored for safety and resolution of any abemaciclib-related adverse events after treatment ends.
Approximately 1 follow-up visit
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
B
Brigitte C Widemann, M.D.
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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