Study on the Effect of Food on Selumetinib in Adolescents with Neurofibromatosis Type 1 Plexiform Neurofibromas

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What is this study about?

This clinical trial is focused on studying a condition called Neurofibromatosis Type 1 (NF1), which is a genetic disorder that often leads to the development of tumors along nerves in the skin, brain, and other parts of the body. Specifically, the study is looking at a type of tumor known as plexiform neurofibromas (PN), which are complex nerve tumors that can cause various health issues. The treatment being tested in this study is a medication called Selumetinib, also known by its code name AZD6244. This medication is taken in the form of hard capsules and is being evaluated for its effects when taken with food.

The purpose of the study is to understand how a low-fat meal affects the way Selumetinib is processed in the body and its impact on the stomach and intestines. The study involves adolescent children who have NF1-related plexiform neurofibromas. Participants will take part in different treatment periods where they will receive multiple doses of Selumetinib. During these periods, the study will assess how the medication is absorbed and tolerated by the body, especially when taken with or without food.

Throughout the study, participants will be monitored for any side effects, and their overall health will be assessed through various tests, including blood tests and heart function tests. The study aims to gather information on the safety and tolerability of Selumetinib in young patients with NF1-related plexiform neurofibromas, providing valuable insights into how the medication can be used effectively in managing this condition.

1 joining the study

Upon joining the study, participants are required to provide signed consent from a parent or legal guardian, along with their own assent if applicable. This consent includes agreement to comply with study requirements and restrictions.

Participants must be between 12 and 18 years old and diagnosed with Neurofibromatosis Type 1 (NF1) with inoperable plexiform neurofibromas (PN).

2 treatment period 1 (T1)

Participants begin taking selumetinib capsules, which are available in 10 mg and 25 mg doses. The medication is taken orally.

The effect of a low-fat meal on the medication’s absorption and gastrointestinal tolerability is assessed during this period.

3 treatment period 2 (T2)

Participants continue taking selumetinib capsules, but this time in a fasted state to compare the effects with those observed in T1.

The focus is on understanding how fasting affects the medication’s absorption and any gastrointestinal side effects.

4 treatment period 3 (T3)

The decision to proceed to T3 depends on the results from T1 and T2.

Participants may take selumetinib capsules again with a low-fat meal to further evaluate the medication’s effects.

5 monitoring and assessments

Throughout the study, participants undergo regular assessments to monitor safety and tolerability. This includes checking for any side effects, conducting laboratory tests, and performing physical examinations.

Participants are required to maintain a diary to record any gastrointestinal symptoms, using a specific rating scale.

6 completion of the study

The study is estimated to conclude by June 2028. Participants will have a final assessment to evaluate the overall effects of the treatment.

The study aims to provide insights into how food affects the absorption and side effects of selumetinib in adolescents with NF1-related PN.

Who Can Join the Study?

Participants must be boys or girls aged 12 to less than 18 years old.
A signed consent form from a parent or legal guardian is required, along with the child’s agreement to participate, if applicable. If the participant reaches the age of legal consent during the study, they will need to sign a new consent form.
Participants must have a diagnosis of Neurofibromatosis Type 1 (NF1) and have inoperable Plexiform Neurofibromas (PN). NF1 is a genetic condition that can cause tumors to grow on nerves. PN are a type of tumor that cannot be completely removed by surgery without causing significant harm.
Participants must need treatment for NF1 and inoperable PN due to symptoms or the risk of serious health problems, as determined by the study doctor. This includes issues like tumors that could affect breathing, cause nerve damage, or lead to significant disfigurement or pain.
Participants who have previously been treated with any MEK inhibitor (a type of medication), including selumetinib, may join the study.
Participants older than 16 must have a Karnofsky performance level of 70 or higher, and those 16 or younger must have a Lansky performance level of 70 or higher. These are scales used to measure a person’s ability to perform daily activities. Participants who use a wheelchair due to paralysis from a PN are considered able to move around if they can do so in their wheelchair.
Participants must have a Body Surface Area (BSA) between 1.3 and 2.5 square meters. BSA is a measurement used to calculate medication doses based on a person’s size.
Participants must be able to swallow whole capsules.
Participants must agree to participate in all three treatment periods of the study.
Female participants who can become pregnant must use a highly effective method of birth control if they are sexually active. They must have a negative pregnancy test before starting the study and before each treatment period. Male participants must use a condom with spermicide if they have a female partner who can become pregnant, and they must not donate sperm during the study.

Who Cannot Join the Study?

Patients who are not diagnosed with Neurofibromatosis Type 1 (NF1), a genetic disorder that causes tumors to grow on nerves.
Patients who do not have Plexiform Neurofibromas (PN), which are a type of tumor associated with NF1.
Patients who are outside the age range specified for the study.
Patients who are not able to take the study medication as required.
Patients who have other medical conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who are participating in another clinical trial.
Patients who have allergies to the study medication or its ingredients.
Patients who are unable to follow the study procedures.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Szpital Uniwersytecki Nr 1 Im. Dr. A. Jurasza W Bydgoszczy	Bydgoszcz	Poland
Uvrvdbqprmjazj Cetjouw Kreqpzptr	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Poland	Not recruiting	21.07.2021

Trial locations

Investigated drugs:

Selumetinib

Selumetinib is a medication being studied for its effects on children with a condition called Neurofibromatosis Type 1 (NF1), which can cause tumors known as plexiform neurofibromas. This trial is looking at how the body absorbs and tolerates the medication when taken with food. The goal is to understand if eating a low-fat meal changes how the medication works in the body after taking it multiple times.

Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) – This is a genetic disorder characterized by the development of multiple benign tumors called plexiform neurofibromas. These tumors grow along nerves and can affect various parts of the body, including the skin, eyes, and bones. The growth of these tumors is typically slow, but they can become large and cause physical disfigurement or functional impairment. Plexiform neurofibromas are often present at birth or develop in early childhood. They can vary greatly in size and number among individuals with NF1. The condition is caused by mutations in the NF1 gene, which is responsible for producing a protein that helps regulate cell growth.

Trial ID:

2024-517216-29-00

Protocol code:

D1346C00015

NCT ID:

NCT05101148

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Effect of Food on Selumetinib in Adolescents with Neurofibromatosis Type 1 Plexiform Neurofibromas

What is this study about?

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