Study of Abemaciclib, a CDK46 Inhibitor, for Treating Atypical Neurofibromas in People with Neurofibromatosis Type 1 NF1 Ages 12 and Older

What this Trial Is About

Neurofibromatosis type 1 NF1 is a genetic disorder that leads to the growth of tumors called atypical neurofibromas ANF, which arise from nerves and can cause serious health issues. These tumors may become aggressive and are difficult to treat with current therapies. Researchers are evaluating the drug abemaciclib, a cyclin-dependent kinase 46 CDK46 inhibitor, to find a safe and tolerable dose for treating people with NF1 and unresectable ANF. This trial includes phases 0, I, and II to assess drug effects, dose levels, and tumor response. Participants will take abemaciclib capsules orally twice daily in 28-day cycles for up to two years, with some possibly extending treatment longer. The study has a phase 0 portion where participants receive abemaciclib for 7 to 10 days before tumor removal surgery, followed by monitoring. The phase III portion enrolls participants with tumors that cannot be safely removed or who decline surgery, with dose escalation to find the recommended phase II dose and assessment of tumor shrinkage by MRI. Treatment tolerability and tumor response will be regularly evaluated during the study. During the study, participants will have frequent visits including medical history, physical exams, blood and urine tests, heart function tests, and MRI scans with contrast agents. Additional assessments involve PET scans, questionnaires on pain and quality of life, and possible tumor photographs. Participants will have a follow-up visit about 30 days after the last dose and then visits every three months for one year. The research team will monitor safety, drug effects on tumors, pharmacokinetics, and quality of life throughout the trial.

Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas