RECRUITING

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in children with NF1 and results in clinically meaningful benefit such as improvement in pain or range of motion. However, many morbidities, such as blindness or nerve damage, cannot be fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient population to determine if young participants with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.

Official Title

Phase 2 Trial of Selumetinib for the Prevention of Plexiform Neurofibroma Growth and Morbidity in Neurofibromatosis Type 1

Quick Facts

Study Start:2025-08-27
Study Completion:2032-09-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06188741

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Year to 8 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. 1. Age: \> 1 (\>12 months) and ≤8 years of age at the time of study enrollment.
  2. 2. Diagnosis: Participants with a diagnosis of NF1 based on the 2021 revised consensus criteria \[52\] and
  3. 3. No known PN (prior to enrollment on Part 1). Participants for whom there is clinical suspicion for a PN (e.g., subtle facial asymmetry or large overlying hyperpigmented area) may be included in the study after discussion with the Study Chair so long as they have not previously had an MRI of the region of concern and are otherwise asymptomatic.
  4. 4. Physical exam at your institution within 1 year prior to consent.
  5. 5. Written informed consent must be obtained from the legal guardians of all participants \<18 years of age.
  1. 1. Presence of a known, symptomatic PN with or without previous MRI imaging.
  2. 2. Patients who have had previous whole-body MRI (WBMRI) are excluded from the study. However, patients who have had regional MRI(s) for an indication other than a PN and did not have a PN identified on previous MRI may still be eligible for the study.
  3. 3. Inability to undergo MRI and/or contraindication for MRI examinations following the MRI protocol.
  4. 4. Prior treatment with selumetinib or another specific MEK1/2 inhibitor.
  5. 5. Evidence of an optic pathway or other low-grade glioma, high grade glioma, malignant peripheral nerve sheath tumor, or other cancer/tumor requiring treatment with chemotherapy, biologic therapy or radiation therapy.
  6. 6. Ongoing radiation therapy, chemotherapy, hormonal therapy directed at a tumor, immunotherapy, or biologic therapy.
  7. 7. Clinical judgement by the investigator that the patient should not participate in the study.

Contacts and Locations

Study Contact

Karen Cole-Plourde, BA
CONTACT
2055141317
kplourde@uab.edu
Juliette Southworth, BS
CONTACT
2055298967
jsouthworth@uab.edu

Study Locations (Sites)

Childrens of Alabama
Birmingham, Alabama, 35233
United States
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229-
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: University of Alabama at Birmingham

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-08-27
Study Completion Date2032-09-01

Study Record Updates

Study Start Date2025-08-27
Study Completion Date2032-09-01

Terms related to this study

Additional Relevant MeSH Terms

  • Neurofibromatosis 1
  • Plexiform Neurofibroma