Study on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

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What is this study about?

This study focuses on a condition known as Neurofibromatosis Type 1 (NF1), which can cause non-cancerous tumors called plexiform neurofibromas. These tumors are symptomatic and cannot be operated on, making them a challenge to treat. The investigation will explore the effectiveness and safety of a drug named Selumetinib, taken in capsule form, compared to a placebo. The goal of the research is to evaluate how well Selumetinib works in treating these tumors.

Participants in the study will receive either Selumetinib or a placebo without knowing which one they are taking to ensure impartiality in the results. The focus will be on monitoring changes in the size and symptoms of the tumors and other related health aspects. Researchers will use a type of imaging called volumetric MRI to assess the effectiveness of the treatment by measuring any changes in tumor size.

Through this approach, the study aims to determine whether Selumetinib can help manage NF1-related plexiform neurofibromas by reducing tumor size and alleviating symptoms. The results will help understand if Selumetinib is a viable treatment for this condition.

1 enrollment

Upon joining the study, eligibility is confirmed. This includes being an adult aged 18 or older with a diagnosis of Neurofibromatosis Type 1 (NF1) and having symptomatic, inoperable plexiform neurofibromas (PN).

A target PN must be measurable by MRI, and chronic PN pain must be documented. Stable use of pain medication is required, along with adequate organ and marrow function.

2 randomization

Participants are randomly assigned to one of two groups: one receiving selumetinib and the other receiving a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.

3 treatment administration

Participants in the selumetinib group take the medication orally. The dosage is either 10 mg or 25 mg, depending on the specific requirements of the study.

The placebo group receives capsules that match the selumetinib in appearance but contain no active medication.

4 monitoring and assessments

Regular monitoring occurs throughout the study. This includes MRI scans to assess changes in the size of the PN and evaluations of pain intensity and quality of life.

Participants’ responses to the treatment are measured using specific criteria, including the Objective Response Rate (ORR) and other health-related quality of life assessments.

5 completion

The study is estimated to conclude by May 25, 2025. At the end of the trial, data from all participants are analyzed to determine the efficacy and safety of selumetinib compared to the placebo.

Who Can Join the Study?

Must be an adult aged 18 years or older.
Must have a diagnosis of Neurofibromatosis Type 1 (NF1), which is a genetic disorder that causes tumors to grow on nerves.
Must have symptomatic, inoperable Plexiform Neurofibromas (PN), meaning the tumors cause symptoms and cannot be removed by surgery.
Must have at least one target PN that can be measured using a special type of scan called volumetric MRI.
Must have a record of chronic pain from the target PN for a certain period during the screening phase.
Must be using stable medication for chronic PN pain at the time of joining the study.
Must have adequate function of organs and bone marrow, which are important for overall health and blood cell production.

Who Cannot Join the Study?

Patients who do not have Neurofibromatosis Type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas (PN) cannot participate. NF1 is a genetic disorder that causes tumors to grow on nerves, and PN are a type of tumor that cannot be removed by surgery.
Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
Patients who are not willing or able to follow the study procedures and requirements cannot participate.
Patients who have other medical conditions that might interfere with the study or its results cannot participate.
Patients who are taking medications that might interfere with the study treatment cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have participated in another clinical trial recently may not be eligible to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Universita’ Degli Studi Della Campania Luigi Vanvitelli	Naples	Italy
Oncopole Claudius Regaud	Toulouse	France

Other Sites

Site Name	City	Country
Hospital Universitario 12 De Octubre	Madrid	Spain
Szpital Uniwersytecki Nr 1 Im. Dr. A. Jurasza W Bydgoszczy	Bydgoszcz	Poland
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Uvygdpyfhs Mmvxomj Cexvqv Hcgjizyqnfkuxtmvl	Hamburg	Germany
Uypzzovvkxycllnetpigc Wdhersaxp Amw	Wuerzburg	Germany
Hbqgwynf Ulhhyhyrpfpfm Heavpopr Tusxd y Pdijiz Iljpzfgb Cqfrjh daghejzztrlbfewhs (ptio	Badalona	Spain
Hfehcus Hapjc Malxhb &ecdfbb 1 rye Gwjjbto Eglpht	Creteil	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	11.04.2024
Germany	Not recruiting	11.04.2024
Italy	Not recruiting	11.04.2024
Poland	Not recruiting	11.04.2024
Spain	Not recruiting	11.04.2024

Trial locations

Investigated drugs:

Selumetinib

Selumetinib is a medication being studied for its potential to help people with a condition called NF1, which can cause tumors to grow on nerves. These tumors, known as plexiform neurofibromas, can be painful and difficult to remove with surgery. The trial is investigating whether selumetinib can reduce the size of these tumors and improve symptoms by using MRI scans to measure changes in tumor size.

Investigated diseases:

Neurofibromatosis

Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN) – This is a genetic disorder characterized by the development of multiple non-cancerous tumors along nerves in the skin, brain, and other parts of the body. These tumors, known as neurofibromas, can cause a variety of symptoms depending on their size and location. Plexiform neurofibromas are a type of tumor associated with NF1 that can grow along nerve pathways and may become large and problematic. When these tumors are symptomatic and cannot be surgically removed, they can lead to pain, disfigurement, and functional impairment. The progression of the disease can vary widely among individuals, with some experiencing mild symptoms and others facing significant challenges. The condition is considered a rare disease and is often diagnosed in childhood.

Trial ID:

2023-507336-20-00

Protocol code:

KOMET D134BC00001

NCT ID:

NCT04924608

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

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