Double-blind, randomized, placebo-controlled study of Leriglitazone in pediatric Rett Syndrome
Rett syndrome is a rare genetic condition that mainly affects girls, causing severe physical, movement, and communication difficulties. This clinical trial is testing an experimental medicine called leriglitazone (also known as min-102) to see if it is safe and can help improve day-to-day symptoms. During the trial, participants take either leriglitazone or a dummy liquid (placebo) by mouth. Neither families nor the study doctors will know who receives the active medicine until the study ends. Doctors will carefully track any side effects and check whether the medicine helps with movement, communication, seizures, and breathing issues such as breath-holding. Carers will also complete questionnaires about daily behaviour and family life. The main goal is to find out if leriglitazone is gentle on the body and safe to explore further as a possible future treatment.
Överblick
Vad handlar denna studie om?
Rett syndrome is a rare neurological condition that almost always affects girls. As children grow, it often leads to challenges with speech, hand movements, walking, breathing, and seizures. Right now, there is no cure for Rett syndrome, and existing treatments can only help manage individual symptoms. This study is testing an experimental medicine called leriglitazone to see if it could offer meaningful support for children living with the condition.
Leriglitazone is provided as a liquid taken by mouth. In this study, some participants will receive the active medicine, while others will receive a placebo. A placebo looks and tastes just like the study drug but contains no active medicine. Comparing the two groups allows researchers to see whether any changes or improvements are genuinely caused by the medicine rather than by chance.
The most important goal of this trial is to test safety. Doctors want to ensure that children can take the medicine comfortably without unwanted or harmful side effects. Alongside safety, the research team will measure whether the treatment helps improve everyday skills, walking, communication, and alertness. They will also look at whether it reduces the number of seizures or irregular breathing episodes, such as hyperventilation and breath-holding.
Living with Rett syndrome can be demanding for both children and their families. By looking at overall well-being and caregiver stress alongside clinical symptoms, researchers hope to find out whether leriglitazone can make daily life easier, safer, and more comfortable for patients and carers in the future.
Viktiga punkter
- Tests an experimental liquid medicine (leriglitazone) for Rett syndrome.
- The primary goal is to ensure the treatment is safe and well tolerated.
- Also monitors changes in movement, communication, breathing issues, and seizures.
- Half of the participants will take the study drug and half will take a dummy medicine (placebo).
- Participation is entirely voluntary and includes close, specialised medical monitoring.
Vem kan vara berättigad?
This study is designed for females with a confirmed diagnosis of Rett syndrome. While the trial focuses on paediatric care, doctors assess each child individually to determine whether taking part is suitable and safe based on their age and overall health.
To be considered, participants generally need to be in stable medical health. Children who currently experience seizures or breathing difficulties may still be able to take part, provided their symptoms and any regular medications have been steady for a set period of time before joining.
Children may not be able to take part if they have severe liver, kidney, or heart problems, or if they are taking other medicines that could interfere with the study drug. A doctor from the study team will review your child's complete medical history to ensure that taking part is as safe as possible.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Confirmed medical diagnosis of Rett syndrome
- Female
- Able to swallow liquid medicine by mouth or via a feeding tube
- Parent or carer available to attend hospital appointments and complete daily diaries
- General health is currently stable without severe, uncontrolled organ disease
Vad innebär deltagande?
Taking part involves receiving a liquid medicine that comes in an amber glass bottle to take by mouth at home. Half of the participants will receive leriglitazone, and half will receive a matching placebo. Families will need to attend regular clinic visits at a study hospital. During these visits, doctors will perform routine health checks, including physical examinations, weight checks, and blood tests to measure how the body processes the medicine. Parents or carers will be asked to fill in questionnaires about their child's motor skills, communication, and daily behaviour. Families will also be asked to keep a simple diary at home to track any seizures or breathing changes. The study team will provide close guidance and follow-up care throughout the entire study period.
Potentiella risker och fördelar
Platser (1)
- —ObekräftadSpanien
Vanliga frågor
What is a placebo and why is it used?
A placebo is a dummy medicine that looks and tastes identical to the real drug but contains no active ingredient. It helps researchers prove whether any health changes are truly caused by the medicine itself.
Will my child definitely receive the active medicine?
No. A computer assigns participants to either leriglitazone or the placebo by chance, like flipping a coin. Neither you nor the study team will know which one your child is taking during the study.
Can my child keep taking their regular medicines?
In most cases, children can continue their usual stable medications. The study doctor will check all current treatments beforehand to make sure they are safe to use alongside the study medicine.
Does taking part cost anything?
No. The study medicine, medical appointments, and trial tests are provided free of charge. Most clinical trials also offer help with travel and parking expenses.
Can we leave the study if we change our minds?
Yes. You can withdraw your child from the trial at any point. Deciding to stop will not affect your child's relationship with their usual healthcare team or their normal NHS treatment.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
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