Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 (NCT06188741)
Phase 2 Trial of Selumetinib for the Prevention of Plexiform Neurofibroma Growth and Morbidity in Neurofibromatosis Type 1
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Childrens of Alabama — Birmingham, Alabama
Age range: 1 Year to 8 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (15 Sites)
- Childrens of Alabama — Birmingham, Alabama
- Children's Hospital of Los Angeles — Los Angeles, California
- Stanford University — Palo Alto, California
- Children's National Hospital — Washington D.C., District of Columbia
- Lurie Children's Hospital of Chicago — Chicago, Illinois
- University of Chicago — Chicago, Illinois
- Riley Hospital for Children/Indiana University — Indianapolis, Indiana
- Johns Hopkins University — Baltimore, Maryland
- National Cancer Institute/ National Institutes of Health — Bethesda, Maryland
- Boston Children's Hospital — Boston, Massachusetts
- Mayo Clinic — Rochester, Minnesota
- Washington University - St. Louis — St Louis, Missouri
- Cincinnati Childrens Hospital Medical Center — Cincinnati, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- University of Texas, Southwestern — Dallas, Texas
📞 Contact the Study Coordinator
Phone: (205) 514-1317
Email: kplourde@uab.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Neurofibromatosis 1
- Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1 — Baltimore, Maryland
- Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1 — Washington D.C., District of Columbia
- NF-1, Nutraceutical Intervention — Minneapolis, Minnesota
- AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors — Washington D.C., District of Columbia
- Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas — Gainesville, Florida
- Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 — Los Angeles, California
Explore by condition: All Neurofibromatosis 1 clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.