55 Participants Needed

Abemaciclib for Neurofibroma

AG
AM
AM
Overseen ByAmanda M Carbonell
Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: National Cancer Institute (NCI)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

AM

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

I can take care of myself but may not be able to do active work.
I have a neurofibroma or plexiform neurofibroma.
My blood, liver, kidney, and heart are functioning well.
See 12 more

Exclusion Criteria

I have interstitial lung disease.
I have a history of fainting due to heart issues, irregular heartbeats, or sudden cardiac arrest.
I need medication that strongly affects liver enzymes.
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Phase 0

Participants receive abemaciclib for 7-10 days prior to tumor resection to determine pharmacodynamic effects

1-2 weeks
1 visit (in-person)

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

Up to 2 years
Frequent visits including MRI and toxicity monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year
Visits every 3 months

What Are the Treatments Tested in This Trial?

Interventions

  • Abemaciclib
Trial Overview The trial is testing the safety and tolerability of a drug called abemaciclib for treating atypical neurofibromas associated with NF1. Patients will take this medication orally twice daily in cycles of 28 days for up to two years, possibly longer.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: 2/ Phase II Objective Response RateExperimental Treatment1 Intervention
Group II: 1/ Phase I Dose EscalationExperimental Treatment1 Intervention

Abemaciclib is already approved in United States, European Union for the following indications:

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Approved in United States as Verzenio for:
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Approved in European Union as Verzenio for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Published Research Related to This Trial

In a phase II trial involving 19 patients with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs), the MAPK/ERK kinase inhibitor mirdametinib showed a 42% partial response rate after 12 courses, indicating its efficacy in reducing tumor size.
Patients reported significant and durable decreases in pain, suggesting that mirdametinib not only affects tumor volume but also improves quality of life for those suffering from NF1-related PNs.
NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas.Weiss, BD., Wolters, PL., Plotkin, SR., et al.[2022]
A Phase I study involving 24 healthy male volunteers evaluated a new granule formulation of selumetinib, showing that its absorption is comparable to the existing capsule form, making it a potential alternative for younger patients or those who have difficulty swallowing capsules.
The study found that the granule formulation was well-tolerated with low incidence of mild adverse events, and participants rated its palatability positively, indicating they would be willing to take it again.
A Phase I, Open-label, Randomized, Crossover Study of the Relative Bioavailability of Capsule and Granule Formulations of Selumetinib.Cohen-Rabbie, S., Mattinson, A., So, K., et al.[2022]
Cantharidin, a protein phosphatase 2A (PP2A) inhibitor, was found to effectively slow down the growth of malignant peripheral nerve sheath tumor (MPNST) cells in patients with neurofibromatosis type 1 (NF1).
Nifedipine, an L-type calcium channel blocker, significantly reduced local tumor growth in an animal model of MPNST, suggesting that both Cantharidin and Nifedipine could be promising candidates for further preclinical studies in treating NF1-related tumors.
Medium throughput biochemical compound screening identifies novel agents for pharmacotherapy of neurofibromatosis type 1.Semenova, G., Stepanova, DS., Deyev, SM., et al.[2018]

Citations

NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. [2022]
A Phase I, Open-label, Randomized, Crossover Study of the Relative Bioavailability of Capsule and Granule Formulations of Selumetinib. [2022]
Medium throughput biochemical compound screening identifies novel agents for pharmacotherapy of neurofibromatosis type 1. [2018]
Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial. [2023]
Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review. [2021]
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