Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 (NCT04954001)
A Multi-center, Open-label, Single-arm Phase I Dose-escalation and Phase II Dose-expansion Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
🩺 Plain-English Summary (8th-Grade Level)
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and neurofibromatosis type 1.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital Los Angeles — Los Angeles, California
Age range: 2 Years to 70 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 (14 Sites)
- Children's Hospital Los Angeles — Los Angeles, California
- Principal Investigator Hans — Gainesville, Florida
- John Hopkins All Children Hospital — St. Petersburg, Florida
- Henry Ford Health System — Detroit, Michigan
- Medical University of South Carolina - Hollings Cancer Center - PPDS — Charleston, South Carolina
- Research Site — Beijing,
- Research Site — Guangzhou,
- Research Site — Hangzhou,
- Research Site — Shanghai,
- Research Site — Shijiazhuang,
- Research Site — Wuhan,
- Hospital Universitario Vall d'Hebron — Barcelona,
- Hospital Universitario 12 de Octubre — Madrid,
- Hospital Universitario La Paz — Madrid,
💰 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
Explore by condition: All Neurofibromatosis 1 clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.