Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas (NCT06502171)
A Phase 1/1b/2 Study of Cabozantinib in Combination With Selumetinib for Plexiform Neurofibroma in Adults and Adolescents With Neurofibromatosis Type 1
🩺 Plain-English Summary (8th-Grade Level)
Based on the clinical activity of both selumetinib and cabozantinib as monotherapies in clinical trials, the demonstrated activity of these agents in reduced doses in preclinical studies, and the non-overlapping toxicity profiles, the study will assess the tolerability and efficacy of selumetinib and cabozantinib in combination in participants with NF1 ≥16 years old with progressive and/or symptomatic PN in a phase 1/1b/2 clinical trial. Trial Design Phase 1 This will be an open label, dose escalation phase. Dose level escalation will be determined by a rolling six design. In this design, up to 6 participants can be enrolled at a given dose level and then evaluated for dose limiting toxicity (DLT) within the DLT window. The DLT window is defined as 16 weeks in this study based on the long half-life of cabozantinib and the desire to have maximum confidence about long-term tolerability of the combination prior to proceeding to the next dose level. Phase 1b Once the recommended phase 2 dose has been determined in phase 1, an expanded cohort of 12 participants will be enrolled in phase 1b portion of the study. Phase 2 This will be an open label, single-arm phase using the recommended phase 2 dose.
Who can join: Review the eligibility criteria below.
Location: Childrens of Alabama — Birmingham, Alabama
Age range: 16 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 (6 Sites)
- Childrens of Alabama — Birmingham, Alabama
- Children's National Hospital — Washington D.C., District of Columbia
- Boston Children's Hospital — Boston, Massachusetts
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- University of Texas, Southwestern — Dallas, Texas
📞 Contact the Study Coordinator
Phone: (205) 529-8967
Email: jsouthworth@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.