Study on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT02407405

Provider View

This clinical trial is testing if the drug selumetinib can help shrink or slow the growth of tumors in adults with Neurofibromatosis Type 1 (NF1) that cannot be removed by surgery.

Patient Parameters

Program Overview

Neurofibromatosis Type 1 (NF1) is a condition that can cause tumors called plexiform neurofibromas (PNs) to grow along nerves. These tumors can lead to serious health issues and are often too large or located in places that make surgery impossible. Currently, there are no effective treatments for NF1 with PNs. This study aims to find out if the drug selumetinib can help reduce the size of these tumors or slow their growth. Participants will take selumetinib capsules twice a day for several 28-day cycles, with regular check-ups and tests to monitor their health and the effect of the treatment.

Description

Neurofibromatosis Type 1 (NF1) is a genetic disorder that affects about 1 in 3,500 people in the U.S. It often leads to the development of benign tumors along nerves, known as plexiform neurofibromas (PNs), in about 25-40% of individuals. These tumors can cause pain, disfigurement, and other health issues. Selumetinib is a drug that may help shrink these tumors by blocking certain signals that make them grow. In previous studies, selumetinib has shown promise in reducing tumor size in children and young adults with NF1.

This study will test selumetinib in adults with NF1 who have PNs that cannot be surgically removed. Participants will take selumetinib capsules every 12 hours, with regular visits to monitor their health and tumor response. The study will continue as long as the drug is tolerated and the disease does not progress. Participants will also undergo various tests, including MRIs and biopsies, to assess the treatment's effectiveness.

The study aims to enroll up to 60 participants over two years. Participants must be at least 18 years old, have a confirmed diagnosis of NF1, and have a PN that is inoperable and causing health issues or growing. They must also be willing to undergo biopsies and regular MRIs. The study will evaluate the safety and effectiveness of selumetinib in reducing tumor size and improving symptoms.

Eligibility Criteria

Inclusion Criteria

  • Must be 18 years or older with a confirmed diagnosis of Neurofibromatosis Type 1 (NF1).
  • Have an inoperable plexiform neurofibroma (PN) that is causing health issues or is growing.
  • Willing to undergo tumor biopsies and regular MRI scans.
  • Have normal organ and bone marrow function as determined by specific blood tests.
  • Able to swallow capsules and follow the study's medication schedule.
  • Agree to avoid excessive sun exposure and certain foods like grapefruit during the study.
  • Have not received selumetinib or similar treatments before.
  • Women of childbearing potential must agree to use effective contraception during the study.

Exclusion Criteria

  • Currently taking other investigational drugs or have taken them in the past 30 days.
  • Have other NF1-related tumors that require treatment with chemotherapy or surgery.
  • Have uncontrolled health issues, such as infections or heart problems, that could interfere with the study.
  • Pregnant or breastfeeding women, or those not willing to use contraception.
  • Have a history of severe allergic reactions to selumetinib or similar drugs.
  • Have had recent major surgery, except for procedures to place a vascular access device.
  • Have unresolved side effects from previous treatments for NF1.

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