Protocol Details

A Phase III, Multicenter, International Study with a Parallel, Randomized, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET)

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number	000696-C
Sponsoring Institute	National Cancer Institute (NCI)
Recruitment Detail	Type: No longer recruiting/follow-up only Gender: Male & Female Min Age: 18 Years Max Age: 120 Years
Referral Letter Required	No
Population Exclusion(s)	None
Keywords	Mek1/2 Inhibitor; Neurofibromatosis Type 1; Neurofibromin; Autosomal Dominant Genetic Disorder; Raf-Mek-Erk Pathway
Recruitment Keyword(s)	None
Condition(s)	Plexiform Neurofibromas
Investigational Drug(s)	Selumetinib
Investigational Device(s)	None
Intervention(s)	Drug: Placebo Drug: Selumetinib
Supporting Site	National Cancer Institute

Background: Neurofibromatosis type 1 (NF1) is a genetic disease that affects the nerves. Many people with NF1 develop tumors called plexiform neurofibromas (PNs) that grow along nerve cells. These tumors can cause pain and affect eyesight and the ability to move freely.

Objective:

To test the effectiveness of a study drug (Selumetinib) against PNs and other symptoms.

Eligibility:

People aged 18 years or older diagnosed with NF1 and inoperable PNs.

Design:

Participants will undergo screening tests. They will have a physical exam. They will have blood and urine tests and tests of their heart function. They will have imaging scans and an eye exam.

Participants will use a handheld device to complete an e-Diary every day. They will record: the level of pain they are feeling, any drugs they took for the pain, and whether they took the study drug on time. Use of the e-Diary will begin during screening and last to the end of the study.

Participants will take Selumetinib or a placebo (dummy tablet) twice per day in 28-day cycles. They will take the drugs by mouth, at home. After cycle 8, all participants will take Selumetinib.

Participants will come to the clinic on day 1 of cycles 1, 2, 4, and 6 and then once every 6 cycles. They will have additional screening tests during these visits. They will have at least 15 study visits.

Participants may give a blood sample for DNA testing.

Participant partners who become pregnant may be asked to answer questions. They will be asked about the pregnancy, delivery, and the baby s health.

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Eligibility

ELIGIBILITY:

-Patients must be 18 years of age with symptomatic, inoperable PN.

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Citations:

Not Provided

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Contacts:

Principal Investigator	Referral Contact	For more information:
Alice P. Chen, M.D. National Cancer Institute (NCI) NIHBC 10 - CLINICAL CENTER BG RM 8D53 10 CENTER DR BETHESDA MD 20892 (240) 781-3320 chenali@mail.nih.gov	Nancy Moore, R.N. National Cancer Institute (NCI) National Institutes of Health Building 10 Room 8D53 10 Center Drive Bethesda, Maryland 20892 (240) 760-6045 nancy.moore@nih.gov	NCI Referral Office National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office 1-888-NCI-1937 ncimo_referrals@mail.nih.gov

Clinical Trials Number:

NCT04924608

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