Selumetinib for Plexiform Neurofibromas

(KOMET Trial)

Recruiting at 36 trial locations

Overseen By AstraZeneca Clinical Study Information Center

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: AstraZeneca

Must not be taking: MEK inhibitors

Disqualifiers: Malignant glioma, Cardiovascular disease, Ophthalmological conditions, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately 3 years

146 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called selumetinib to see if it can help adults with a condition called NF1 who have tumors that cause symptoms and cannot be removed by surgery. The medication works by blocking signals that make the tumors grow.

Research Team

Alice P. Chen, MD

Principal Investigator

National Cancer Institute (NCI)

Eligibility Criteria

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

I am 18 or older with NF1 and have a tumor that can't be surgically removed.

Chronic target PN pain score documented for minimum period during screening period

My organs and bone marrow are working well.

Exclusion Criteria

I had cancer treated over 5 years ago with no signs of return.

I have a confirmed or suspected brain tumor, but not a low-grade glioma needing no treatment.

I do not have serious heart problems.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive selumetinib or placebo in a double-blind, placebo-controlled setting

Approximately 3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Treatment Details

Interventions

• Selumetinib

Trial Overview The trial is testing the effectiveness of Selumetinib versus a placebo in adults with NF1 who have painful and inoperable plexiform neurofibromas to see if it helps reduce symptoms.

Participant Groups

Group I: Arm A Experimental Treatment 1 Intervention

Selumetinib

Group II: Arm B Placebo Group 1 Intervention

Placebo

Selumetinib is already approved in United States, European Union for the following indications:

Approved in United States as Koselugo for:

• Neurofibromatosis type 1 (NF1) in pediatric patients 2 years of age and older with symptomatic, inoperable plexiform neurofibromas (PN)

Approved in European Union as Koselugo for:

• Neurofibromatosis type 1 (NF1) in pediatric patients 2 years of age and older with symptomatic, inoperable plexiform neurofibromas (PN)

Who Is Running the Clinical Trial?

AstraZeneca

Lead Sponsor

Trials

4,491

Recruited

290,540,000+

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

Trials

4,096

Recruited

5,232,000+

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