Access to Selumetinib for Treating Neurofibromatosis Type 1

Sponsor: AstraZeneca

Expanded Access
Approved for Marketing
Sponsor score: 40
Inactive

ClinicalTrials ID:NCT03259633

Provider View

This study provides access to the drug selumetinib for patients with neurofibromatosis type 1 (NF1) who have tumors that cannot be removed by surgery and have no other treatment options.

Patient Parameters

Program Overview

This study allows patients with neurofibromatosis type 1 (NF1) to receive the drug selumetinib if they have tumors called plexiform neurofibromas (PN) that cannot be surgically removed and are causing symptoms or growing. The study is open to about 100 patients in the US who meet the criteria. Patients can continue taking selumetinib as long as it helps them and does not cause unacceptable side effects.

Description

Patients eligible for this study must have been diagnosed with NF1 and have tumors that cannot be removed by surgery without significant risk. These tumors must be causing symptoms or growing. The study is open to patients aged 2 years and older who developed the disease before age 18 and can swallow capsules. There is no set time limit for how long patients can take selumetinib; they can continue as long as it is beneficial and safe. If patients stop taking selumetinib, their doctor will decide on other treatment options. Patients must sign a consent form to participate.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with neurofibromatosis type 1 (NF1) and have tumors that cannot be surgically removed and are causing symptoms or growing.
  • Tumors must be inoperable, meaning they cannot be completely removed without significant risk.
  • Patients must be 2 years or older, have developed the disease before age 18, and be able to swallow capsules.
  • Normal heart function and blood pressure.
  • Have tried all other approved treatments for NF1 with inoperable tumors.
  • Must sign a consent form to participate.
  • Female patients of childbearing age must have a negative pregnancy test or be post-menopausal.

Exclusion Criteria

  • Ongoing severe side effects from previous treatments.
  • Eligible for other clinical trials with selumetinib.
  • Eye conditions like retinal issues or high eye pressure.
  • Not using effective birth control if of reproductive age.
  • Breastfeeding women.
  • Any other significant health issues that make participation unsafe.
  • Severe or uncontrolled diseases like heart, liver, or kidney disease, active infections, or bleeding disorders.
  • Severe nausea, vomiting, or bowel issues that affect medication absorption.

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