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

NCT04750928 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 55

Last updated 2026-04-24

No results posted yet for this study

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.

Conditions

  • Neurofibromatosis 1

Interventions

DRUG

Abemaciclib

Abemaciclib is to be administered orally on Days 1 through 28 of each 28-day cycle

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

  • Brigitte C Widemann, M.D. · National Cancer Institute (NCI)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-29
Primary Completion
2028-12-31
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04750928 on ClinicalTrials.gov