Not recruitingRare disease

A study testing the safety and how the body processes TAK-861 in children and teenagers aged 6 to 17 years with narcolepsy type 1

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study is looking at a condition called narcolepsy type 1, which is a sleep disorder where people experience excessive daytime sleepiness and sudden muscle weakness triggered by emotions, a symptom known as cataplexy. The study will use a medication called TAK-861, also known as oveporexton, which comes in tablet form and is taken by mouth. This medication has been designated as an orphan drug, meaning it is being developed to treat a rare condition.

The purpose of this study is to understand how the body of children and teenagers processes this medication by measuring drug levels in the blood over time, and to check if the medication is safe and acceptable for young people to take. The study will include children from 6 years old up to 18 years old who have been diagnosed with narcolepsy type 1. Participants will need to have either a specific genetic marker or very low levels of a brain substance called hypocretin in their spinal fluid, which are both signs of this type of narcolepsy. They will also need to have a body mass index within a certain range and be in generally good health as determined by various medical tests including blood work, physical examination, heart rhythm recording, and vital signs measurements.

During the study, participants will receive a single dose of the medication, and doctors will collect blood samples at different times to see how the drug moves through the body. The study will measure things like the highest amount of drug in the blood, how long it takes to reach that level, and how long the drug stays in the body. Researchers will also watch for any unwanted effects and ask participants and their parents or guardians about how acceptable the medication is to take and what it tastes like. This is an open-label study, which means both the doctors and participants will know that everyone is receiving the active medication rather than a placebo.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment and confirmation of eligibility
Your eligibility for this trial will be confirmed based on several factors. You must have a diagnosis of narcolepsy type 1, which is a sleep disorder characterized by excessive daytime sleepiness and sudden muscle weakness triggered by emotions.
Your age must be between 6 and 17 years at the time of joining the trial.
Your body mass index must be within the range of 18 to 30 kg/m². Body mass index is a measure that uses height and weight to estimate body fat.
You must have either a positive result for a specific genetic marker called HLA-DQB1*06:02, or your cerebrospinal fluid must show a concentration of a substance called orexin/hypocretin-1 below 110 pg/mL. These tests help confirm the diagnosis of narcolepsy type 1.
A physician will evaluate your overall health through laboratory tests, review of your medical history, physical examination, a 12-lead electrocardiogram (a test that records the electrical activity of your heart), and vital sign measurements (such as blood pressure, heart rate, and temperature).
Your parent or legal guardian, and you if old enough to understand, will need to sign an informed consent form after reviewing all information about the trial.
Step 2
Single dose administration of the trial medication
You will receive a single dose of the trial medication called oveporexton (also known as TAK-861).
The medication is provided in tablet form and is taken by mouth.
The specific dosage will be determined by the trial physician based on your individual characteristics.
Step 3
Blood sample collection for medication level monitoring
Blood samples will be collected at specific times after you take the medication to measure the levels of oveporexton in your bloodstream.
These measurements will determine how quickly the medication reaches its highest level in your blood (maximum concentration and time to reach maximum concentration), and how long the medication stays in your body (area under the concentration-time curve).
Step 4
Safety monitoring
Throughout the trial, you will be monitored for any treatment-emergent adverse events, which are any unwanted or unexpected medical occurrences that happen after taking the medication.
The trial physician will assess whether any health changes are related to the trial medication.
Step 5
Evaluation of medication acceptability
You will be asked to provide your opinion on the acceptability and palatability of the medication.
Acceptability refers to how easy or difficult it is for you to take the medication, while palatability refers to how the medication tastes.

Who can join the trial?

7 criteria

The participant must have a confirmed diagnosis of Narcolepsy Type 1, which is a sleep disorder that causes sudden muscle weakness and extreme daytime sleepiness, according to the ICSD-3 or ICSD-3-TR classification systems (these are international guidelines doctors use to identify sleep disorders)
The participant must be between 6 and 17 years old at the time of signing the consent form
The participant must be either male or female
The participant must have a body mass index (a measure that uses height and weight to estimate body fat) between 18 and 30 kg/m2
The participant must test positive for a specific genetic marker called HLA-DQB1*06:02 (a gene linked to Narcolepsy Type 1), or test results must show that the level of a brain chemical called hypocretin-1 in the cerebrospinal fluid (the liquid surrounding the brain and spinal cord) is less than 110 pg/mL
The participant must be considered healthy enough to take part in the study based on the doctor's evaluation, which includes blood tests, medical history review, physical examination, heart monitoring with an ECG (a test that records the electrical activity of the heart), and measurements of blood pressure, heart rate, and temperature
The parent or legal guardian, and the participant if old enough to understand, must agree to participate and complete the study by signing a consent form after receiving and reviewing all study information

Who cannot join the trial?

5 criteria

The study information does not list specific reasons why patients cannot participate in this clinical trial
If you are considering joining this study, the research team will review your individual medical situation to determine if you are eligible
General factors that often prevent participation in clinical trials may include having other serious medical conditions, taking certain medications that could interfere with the study drug, or having allergies to the study medication
Being pregnant or breastfeeding (feeding a baby with breast milk) may also be a reason for exclusion in many studies
Having problems with your liver (the organ that helps clean your blood and process medications) or kidneys (the organs that filter waste from your blood) might prevent participation

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Verified sites

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Verified
Azienda Unita Sanitaria Locale Di Bologna
Bologna, Italy

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Investigated drugs

Oveporexton is a medication being tested in this trial for children and teenagers with narcolepsy type 1. Narcolepsy type 1 is a sleep disorder that causes excessive daytime sleepiness and sudden loss of muscle control. This medication is given as a single dose to study how the body processes it, how safe it is, and whether children find it acceptable to take.

N-{(2S,3R)-4,4-Difluoro-1-(2-Hydroxy-2-Methylpropanoyl)-2-[(2,3',5'-Trifluoro[1,1'-Biphenyl]-3-Yl)Methyl]Pyrrolidin-3-Yl}Ethanesulfonamide

What is already known about the treatment

Oveporexton – Oveporexton is an investigational medication currently being studied in clinical trials for the treatment of narcolepsy type 1, a sleep disorder characterized by excessive daytime sleepiness and sudden loss of muscle control called cataplexy. This drug is being tested in children and adolescents aged 6 to 17 years to understand how the body processes it and whether it is safe and acceptable for young patients. Oveporexton works by targeting specific receptors in the brain that help regulate the sleep-wake cycle, potentially helping patients stay awake during the day and reducing episodes of cataplexy. The medication is classified as a central nervous system agent that affects brain chemicals involved in controlling alertness and sleep patterns.

Investigated diseases

Narcolepsy Type 1 – Narcolepsy Type 1 is a chronic neurological disorder that affects the brain's ability to regulate sleep-wake cycles properly. People with this condition experience sudden and uncontrollable episodes of falling asleep during the day, regardless of the circumstances or how much sleep they had the night before. A key feature of this type is cataplexy, which involves a sudden loss of muscle tone triggered by strong emotions such as laughter, surprise, or anger. During a cataplexy episode, a person may experience anything from slight weakness, like drooping eyelids or slurred speech, to a complete collapse, though they remain conscious throughout. The disorder typically begins in childhood or adolescence and continues throughout life. These symptoms significantly interfere with daily activities, work, and social interactions.