Study on Selumetinib for Children Aged 1 to 6 with Neurofibromatosis Type 1 and Inoperable 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 can cause tumors to grow on nerves. Specifically, the study is looking at a type of tumor known as plexiform neurofibromas (PN), which are often difficult to remove with surgery. The trial is testing a medication called Selumetinib, also known by its code name AZD6244. This medication is being studied in a granule form that is suitable for children. The purpose of the study is to understand how the body processes Selumetinib, as well as to evaluate its safety and how well it is tolerated by young children.

The study involves children aged 1 to 7 years who have symptomatic, inoperable plexiform neurofibromas. Participants will receive the Selumetinib granule formulation orally, and researchers will monitor how the medication is absorbed and processed in the body. The study will also assess the safety of the medication by observing any side effects and conducting various health checks, including physical exams and imaging tests like MRI scans. Parents will also provide feedback on how easy it is for their children to take the medication.

This trial is designed to gather important information over several years, with an estimated completion date in 2028. The findings from this study could help improve treatment options for children with NF1-related plexiform neurofibromas, providing insights into the effectiveness and safety of Selumetinib in this young age group.

1 joining the study

Upon joining the study, the legally authorized representative (parent or guardian) of the participant signs the informed consent. The participant must be between 1 and 7 years old and diagnosed with symptomatic, inoperable plexiform neurofibromas (PN) related to neurofibromatosis type 1 (NF1).

2 initial assessment

An initial assessment is conducted to ensure the participant meets all eligibility criteria. This includes having a measurable PN of at least 3 cm, a Lansky performance score of at least 70 (or 40 for those with limited mobility), and a body surface area (BSA) between 0.4 and 1.09 m².

3 medication administration

The participant receives the medication selumetinib in a granule formulation. The medication is administered orally. The specific dosage and frequency are determined based on the participant’s condition and study protocol.

4 monitoring and assessments

Throughout the study, the participant undergoes regular monitoring and assessments. These include checking for any side effects, conducting laboratory tests, physical examinations, and imaging studies such as MRI or X-ray. Vital signs, weight, and performance status are also evaluated.

5 safety and efficacy evaluation

The primary goal is to evaluate the safety and tolerability of selumetinib. This involves monitoring adverse events, their frequency, and their relationship to the medication. The study also assesses the medication’s effectiveness in reducing the size or symptoms of the PN.

6 completion of the study

The study is estimated to conclude by April 28, 2028. Upon completion, the data collected will be analyzed to determine the overall safety and efficacy of the treatment.

Who Can Join the Study?

Participants must be boys or girls aged between 1 and 6 years old when their parent or guardian signs the consent form.
All participants need to have a condition called Neurofibromatosis Type 1 (NF1) with Plexiform Neurofibromas (PN), which are tumors that cannot be removed by surgery and cause symptoms.
Participants must have at least one measurable PN, which means the tumor is at least 3 cm in size and can be clearly seen on medical images. If a participant had surgery to remove a PN but it wasn’t completely removed, they can still join if the remaining tumor can be measured.
Participants need to have a Lansky performance score of 70 or higher. This score measures how well a child can perform daily activities. If a child uses a wheelchair or needs help with breathing, they need a score of 40 or higher.
Participants must have a Body Surface Area (BSA) between 0.4 and 1.09 square meters at the start of the study. BSA is a way to measure the size of a person’s body.
The parent or guardian of the participant must sign a consent form for the study, and if suitable, the child should also agree to participate.

Who Cannot Join the Study?

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Ludwig Maximilian University Of Munich	Munich	Germany
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Uwbhderigc Mmfntps Czrrkl Hwfhhelkrqhltaixt	Hamburg	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	18.04.2024
Italy	Not recruiting	18.04.2024
Spain	Not recruiting	18.04.2024

Trial locations

Investigated drugs:

Selumetinib

Selumetinib is a medication being studied in this trial. It is used in a granule form and is being tested for its effects on children aged 1 to 7 years who have a condition called Neurofibromatosis Type 1 (NF1). This condition can cause tumors called plexiform neurofibromas, which are often difficult to remove with surgery. The trial aims to understand how the body processes selumetinib, as well as to evaluate its safety and how well it is tolerated by the children.

Investigated diseases:

Neurofibromatosis

Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN) – This is a genetic disorder characterized by the development of multiple non-cancerous tumors called plexiform neurofibromas. These tumors grow along nerves and can cause a variety of symptoms depending on their size and location. They often appear in childhood and can lead to physical disfigurement, pain, and functional impairment. The growth of these tumors is typically slow, but they can become quite large over time. Plexiform neurofibromas are associated with the NF1 gene mutation, which affects cell growth and division. The condition is considered rare and can vary significantly in its presentation and severity among individuals.

Trial ID:

2023-506357-38-00

Protocol code:

SPRINKLE D1346C00004

NCT ID:

NCT05309668

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Selumetinib for Children Aged 1 to 6 with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

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