Phase II Study of Poly-ICLC for Treating Low-Grade Brain Tumors in Children with NF1

Sponsor: University of Alabama at Birmingham

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04544007

Provider View

This clinical trial is testing the effectiveness of a treatment called poly-ICLC for children with low-grade brain tumors associated with NF1. The study aims to see if this treatment can help shrink tumors and improve patient outcomes over a 48-week period.

Patient Parameters

Program Overview

This study is exploring the use of poly-ICLC (Hiltonol®) to treat children with low-grade brain tumors linked to a condition called NF1. The main goal is to see if the treatment can reduce tumor size within the first 48 weeks. Researchers will also look at how long patients remain free from tumor progression over 12, 24, and 60 months, and assess any side effects. Additionally, the study will explore how the treatment affects vision in children with tumors affecting the optic pathway, as well as overall quality of life.

Description

This trial is focused on children with low-grade brain tumors related to NF1, a genetic condition. The treatment being tested is poly-ICLC, which will be given over 12 cycles, each lasting four weeks. The main aim is to see if the treatment can shrink tumors within the first 48 weeks. Secondary goals include evaluating how long patients stay free from tumor progression and assessing any side effects. Researchers will also explore how the treatment affects vision in children with optic pathway tumors and overall quality of life.

Participants must be under 22 years old and have a confirmed diagnosis of NF1. They should have a low-grade brain or spinal cord tumor that has progressed or returned after previous treatment. Patients must have measurable tumors and meet specific health criteria, including adequate organ function and recovery from previous treatments.

The study excludes those who have had prior radiation for their tumor, previous exposure to poly-ICLC, or other ongoing cancer treatments. Patients with certain medical conditions or who are pregnant or breastfeeding are also not eligible. Participants must be able to start treatment within seven days of enrollment and have stable neurological symptoms for at least one week before joining the study.

Eligibility Criteria

Inclusion Criteria

  • Must be under 22 years old.
  • Have a confirmed diagnosis of NF1, either through genetic testing or clinical criteria.
  • Have a low-grade brain or spinal cord tumor (WHO Grade 1 or 2).
  • Tumor must have progressed or returned after previous treatment.
  • Must have measurable tumor size greater than 1 cm².
  • Have had at least one prior treatment for the tumor.
  • Be in good general health with a performance status of 50 or higher.
  • Must have recovered from any side effects of previous treatments.
  • Must meet specific organ function requirements, including adequate blood counts, kidney, liver, and lung function.
  • Female patients of childbearing potential must have a negative pregnancy test and agree to use birth control.
  • Must be able to start treatment within seven days of enrollment.
  • Must have stable neurological symptoms for at least one week before joining the study.
  • Surgery must not be a feasible option for complete tumor removal, or the patient must refuse surgery.
  • Parents or guardians must provide informed consent and agree to comply with study requirements.

Exclusion Criteria

  • Have had prior radiation treatment for the tumor.
  • Have been previously treated with poly-ICLC.
  • Are currently receiving other cancer treatments.
  • Have a diagnosis of a more aggressive brain tumor (WHO Grade III or IV).
  • Have had another cancer requiring treatment in the last 48 months.
  • Have a fever of 38.5°C or higher within three days of enrollment.
  • Have an active autoimmune disease.
  • Are pregnant or breastfeeding.
  • Are not willing to use effective birth control during the study and for 90 days after.
  • Have a severe infection requiring IV antibiotics.
  • Have significant uncontrolled medical conditions.
  • Require high doses of steroids or other immunosuppressive therapy.

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