Selumetinib for Plexiform Neurofibromas
(KOMET Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if selumetinib is effective for individuals with neurofibromatosis type 1 (NF1) who have painful and non-removable plexiform neurofibromas (PNs), which are nerve tumors. Participants will receive either the experimental treatment, selumetinib, or a placebo, a harmless pill resembling the treatment. The trial seeks adults diagnosed with NF1 who experience chronic pain from these tumors and are already on stable pain medication. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.
Do I have to stop taking my current medications for the trial?
The trial does not specify if you need to stop taking your current medications. However, it mentions that participants should have stable chronic pain medication use at enrollment, which suggests you may continue your current pain medications.
Is there any evidence suggesting that selumetinib is likely to be safe for humans?
Previous studies have shown that selumetinib is generally safe for patients. Common side effects include vomiting, diarrhea, and skin rashes, which might require dosage adjustments for some individuals. However, long-term use in children with neurofibromatosis type 1 has helped shrink tumors and reduce pain. The FDA has approved selumetinib for children with this condition, suggesting it is reasonably safe based on existing research.12345
Why do researchers think this study treatment might be promising for plexiform neurofibromas?
Selumetinib is unique because it targets a specific pathway in the cells called the MEK pathway, which plays a crucial role in the growth of plexiform neurofibromas. Unlike standard treatments like surgery or chemotherapy, which can have significant side effects and aren't always effective, selumetinib offers a targeted approach that could potentially shrink these tumors. Researchers are excited because this drug could improve quality of life for patients by reducing tumor size and associated symptoms, offering a non-invasive alternative with promising results.
What evidence suggests that selumetinib might be an effective treatment for plexiform neurofibromas?
Research has shown that selumetinib yields promising results in treating plexiform neurofibromas, tumors associated with neurofibromatosis type 1 (NF1). One study found that selumetinib significantly reduced tumor size in about 20% of participants, meaning some patients experienced smaller tumors, which can alleviate symptoms. In the current trial, participants will receive either selumetinib or a placebo. Previous studies have found that selumetinib led to noticeable improvements compared to a placebo, especially in adults with tumors that cannot be surgically removed. These findings suggest selumetinib could be an effective option for managing this condition.56789
Who Is on the Research Team?
Alice P. Chen, MD
Principal Investigator
National Cancer Institute (NCI)
Are You a Good Fit for This Trial?
Adults over 18 with Neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas can join. They must have stable pain medication use, measurable tumors by MRI, and proper organ/marrow function. Excluded are those with certain malignancies or severe heart disease.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive selumetinib or placebo in a double-blind, placebo-controlled setting
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Selumetinib
Selumetinib is already approved in United States, European Union for the following indications:
- Neurofibromatosis type 1 (NF1) in pediatric patients 2 years of age and older with symptomatic, inoperable plexiform neurofibromas (PN)
- Neurofibromatosis type 1 (NF1) in pediatric patients 2 years of age and older with symptomatic, inoperable plexiform neurofibromas (PN)
Find a Clinic Near You
Who Is Running the Clinical Trial?
AstraZeneca
Lead Sponsor
Sir Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Dr. Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Merck Sharp & Dohme LLC
Industry Sponsor
Chirfi Guindo
Merck Sharp & Dohme LLC
Chief Marketing Officer since 2022
Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business
Robert M. Davis
Merck Sharp & Dohme LLC
Chief Executive Officer since 2021
JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University