Study Comparing Selumetinib to Carboplatin/Vincristine in Patients with Neurofibromatosis and Low-Grade Glioma

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT03871257

Provider View

This clinical trial is testing whether the drug selumetinib is as effective as the standard chemotherapy treatment with carboplatin and vincristine for patients with low-grade glioma associated with neurofibromatosis type 1 (NF1). The study also aims to see if selumetinib can improve vision in patients with tumors affecting the optic nerves.

Patient Parameters

Program Overview

This study is exploring whether selumetinib, a drug that blocks certain enzymes needed for tumor growth, works as well as the standard chemotherapy drugs carboplatin and vincristine in treating low-grade glioma in patients with neurofibromatosis type 1 (NF1). The trial also investigates if selumetinib can improve vision in patients with tumors affecting the optic nerves. Researchers want to find out if selumetinib is better than the standard treatment in these aspects.

Description

The main goals of this study are to see if selumetinib can keep the cancer from getting worse for as long as the standard treatment with carboplatin and vincristine, and to check if it improves vision in patients with optic nerve tumors.

Secondary goals include:

  • Estimating how well each treatment shrinks tumors and how long patients live.
  • Evaluating vision using different tests in patients old enough to perform them.
  • Assessing improvements in motor skills for patients with motor issues.
  • Comparing quality of life and cognitive, social, emotional, and behavioral functioning between the two treatments.

Exploratory goals include:

  • Using advanced imaging techniques to measure changes in the eye and brain.
  • Collecting blood and tumor samples for future research.

Participants are randomly assigned to one of two groups:

Group 1: Receives carboplatin and vincristine through an IV, with regular MRI scans to monitor progress. Treatment is given in cycles, with breaks in between, for up to 8 cycles.

Group 2: Takes selumetinib pills twice a day continuously, with regular MRI scans to monitor progress. Treatment continues for up to 27 cycles.

After treatment, patients will have follow-up visits with MRIs and physical exams every few months for several years to monitor their health.

Eligibility Criteria

Inclusion Criteria

  • Aged between 2 and 21 years.
  • Body surface area of at least 0.5 m².
  • Diagnosed with neurofibromatosis type 1 (NF1) and low-grade glioma (LGG).
  • Newly diagnosed or previously diagnosed NF1-associated LGG that hasn't been treated with anything other than surgery.
  • For optic pathway gliomas (OPGs):
    • New or worsening neurological symptoms or tumor growth.
    • Visual issues documented within the past year.
  • For LGG in other locations:
    • New or worsening neurological symptoms or tumor growth.
  • Tumor size of at least 1 cm².
  • Metastatic disease or multiple primary LGGs allowed.
  • Adequate kidney, liver, and heart function.
  • Ability to swallow capsules.
  • English or Spanish language skills for assessments.
  • Signed informed consent from patient or guardian.

Exclusion Criteria

  • Previous treatment with chemotherapy, radiation, or immunotherapy.
  • Concurrent or recent history of another cancer.
  • Serious medical or psychiatric conditions that could interfere with the study.
  • Pregnant or breastfeeding women.
  • Not using effective contraception if of reproductive potential.
  • Certain heart conditions or eye conditions.
  • Taking medications or supplements that interfere with the study.
  • Recent surgery, unless healed.
  • Uncontrolled infections.

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