Selumetinib Pilot Study for Cutaneous Neurofibromas

Status Not yet recruiting

Clinical Trial Summary

This is a small study of the oral MEK1/2 inhibitor, selumetinib, to evaluate the potential utility of selumetinib in individuals ≥ 18 years old with Neurofibromatosis 1 (NF1) and cutaneous neurofibromas (cNFs). The study aims to determine whether selumetinib will result in shrinkage of existing cutaneous neurofibromas and if it prevents or delays the development of new cutaneous neurofibromas.

Clinical Trial Description

This is a limited institution open label pilot study of the oral MEK1/2 inhibitor, selumetinib, to evaluate the potential utility of selumetinib in up to 24 adults with NF1 and cutaneous neurofibromas. The study's primary objective is to determine whether selumetinib results in shrinkage of existing cutaneous neurofibromas; the secondary objective is to assess if selumetinib prevents or delays the development of new cutaneous neurofibromas.

All subjects will commence treatment with selumetinib orally at 50 mg /dose approximately every 12 hours (one cycle = 28 days). Patients will be able to escalate to 75mg every 12 hours [BID], if the medication is tolerated well for the first cycle, with no toxicities of grade 2 or greater. Patients will undergo regular evaluation for selumetinib related toxicities. In absence of treatment limiting toxicity, or progression of disease, patients may remain on treatment for a maximum of 24 cycles unless they experience a volume decrease in the target cutaneous neurofibromas, in which case treatment may continue for an additional 12 cycles off trial.

For response evaluation, target cutaneous neurofibromas in 3 different body regions will be measured using paper frames, calipers, and photography at baseline and then after every 4 cycles. Exploratory studies will also be performed during the trial to assess the effect of selumetinib on skin related morbidity as well as cutaneous neurofibroma target inhibition, tumor microenvironment, and pathology. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03105258
Study type	Interventional
Source	University of Alabama at Birmingham
Contact	Ashley Cannon, PhD, MS, CGC
Phone	205-996-2916
Email	ashleycannon@uabmc.edu
Status	Not yet recruiting
Phase	Phase 2
Start date	May 1, 2017
Completion date	December 2021

View Details

See also
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Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.