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AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

NCT01362803 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 99

Last updated 2026-05-22

No results posted yet for this study

Summary

Background:

\- Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat them is with surgery. Some of these tumors cannot be completely removed. The tumors may be too large, too numerous, or in a bad location for surgery. An experimental drug called AZD6244 hydrogen sulfate may be able to prevent the tumors from growing, slow down their growth, or shrink them. This drug has been tested in adults with cancer and in children with some types of brain cancer. This study will test how well this drug works with these types of tumors.

Objectives:

\- To study the safety and effectiveness of AZD6244 hydrogen sulfate in children and young adults with plexiform neurofibromas that cannot be completely removed by surgery.

Eligibility:

\- Children and young adults between 12 and 18 years of age who have plexiform neurofibromas that cannot be completely removed by surgery.

Design:

* Patients will be screened with a physical exam, medical history, blood tests, and imaging studies.
* They will take the study drug twice a day with 8 ounces of water, every day for 28-day cycles of treatment. During study visits, participants will have blood and urine tests and physical exams. They will also have imaging studies to examine the tumor sizes and locations. They will answer questions about their health. They may have other tests as needed.
* Participants will continue to receive the study drug as long as they have no severe side effects and the disease is not getting worse.

Conditions

  • Neurofibromatosis 1
  • Neurofibromatosis Type 1
  • NF1
  • Neurofibroma, Plexiform

Interventions

DRUG

AZD6244

AZD6244 orally (at recommended Ph2 dose) every 12 hours on continuous daily schedule for cycles of 28 days until unacceptable toxicity, patient withdrawal or PD

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

  • Brigitte C Widemann, M.D. · National Cancer Institute (NCI)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-21
Primary Completion
2027-01-01
Completion
2032-01-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01362803 on ClinicalTrials.gov