Not recruitingRare disease

Study on the Safety and Tolerability of Leriglitazone for Children with Rett Syndrome

Verified siteInvestigational

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What is this trial about?

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

This clinical trial is focused on studying Rett Syndrome, a rare genetic disorder that affects brain development, leading to severe physical and cognitive impairments. The study will evaluate a treatment called leriglitazone, which is provided as an oral suspension. The purpose of the study is to assess the safety and tolerability of leriglitazone in children with Rett Syndrome.

Participants in the study will be randomly assigned to receive either leriglitazone or a placebo, which looks like the treatment but does not contain the active substance. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This approach helps ensure that the results are not biased. The study will last for a period of up to 36 weeks, during which participants will take the oral suspension daily.

Throughout the study, various assessments will be conducted to monitor the participants' health and any changes in their condition. These assessments will include questionnaires and scales that measure behavior, motor skills, communication, and overall clinical impressions. The study will also track the number of seizures, apnea episodes, and hyperventilation episodes experienced by participants each week. The goal is to gather comprehensive data on how leriglitazone affects individuals with Rett Syndrome and to determine its potential benefits and risks.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
The study involves a double-blind, randomized, placebo-controlled trial for pediatric Rett Syndrome.
Participants are female subjects aged between 5 and 12 years with a diagnosis of classical Rett Syndrome and a documented mutation of the MECP2 gene.
Participants must be able to swallow the study treatment provided as an oral liquid suspension.
Step 2
Treatment administration
The treatment involves the administration of leriglitazone or a placebo.
The medication is provided as an oral suspension and can be taken orally, through a nasogastric tube, or a percutaneous endoscopic gastrostomy tube.
The appearance of the placebo is similar to the leriglitazone drug product.
Step 3
Monitoring and assessments
The primary goal is to evaluate the safety and tolerability of leriglitazone.
Safety and tolerability are assessed based on summary statistics for adverse events and other safety considerations.
Secondary assessments include changes in behavior, motor skills, communication, and caregiver burden, as well as the number of seizures, apnea, and hyperventilation episodes recorded weekly.
Step 4
Study duration
The estimated end date for the trial is November 30, 2025.
The recruitment for the study is expected to start on October 1, 2024.

Who can join the trial?

6 criteria

The patient's parent(s) or legal guardian(s) must provide a signed written agreement, called an Informed Consent Form, before any study procedures begin.
The patient must be a female between the ages of 5 and 12 years old at the time of signing the consent form.
The patient must have a diagnosis of classical or typical Rett Syndrome, confirmed by a specific change in the MECP2 gene.
The patient must have a severity score between 10 and 36 on a specific scale used to measure the condition.
The patient must be able to swallow the study medication, which is given as a liquid.
If the patient is of childbearing age, she must either not be able to have children or agree to use a reliable method to prevent pregnancy from at least 4 weeks before starting the study until 4 weeks after the study ends or if she leaves the study early.

Who cannot join the trial?

7 criteria

Participants cannot be male. Only females are allowed to join the study.
Participants must not have any other serious health conditions that could interfere with the study.
Participants should not be taking any medications that might affect the study results.
Participants must not have a history of allergic reactions to similar medications.
Participants should not be pregnant or planning to become pregnant during the study.
Participants must not have participated in another clinical trial recently.
Participants should not have any mental health conditions that could affect their ability to follow study instructions.

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Verified sites

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Hospital Sant Joan De Deu Barcelona
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Esplugues De Llobregat, Spain

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Investigated drugs

Leriglitazone is a medication being studied for its potential to help children with Rett Syndrome. Rett Syndrome is a rare genetic disorder that affects brain development, leading to severe physical and mental disabilities. The trial aims to assess how safe and well-tolerated leriglitazone is for these patients.

Leriglitazone

What is already known about the treatment

Leriglitazone – Leriglitazone is administered orally and is currently being studied in clinical trials for its potential use in treating Rett Syndrome, a rare neurological disorder. The medication is in the investigational stage, with ongoing research to assess its safety and tolerability in pediatric patients. Leriglitazone is believed to work by modulating specific pathways in the brain, potentially improving neurological function. It is classified as a neuroprotective agent, aiming to support brain health and function.

Investigated diseases

Rett Syndrome – Rett Syndrome is a rare genetic neurological disorder that primarily affects girls. It typically becomes apparent after 6 to 18 months of age, following a period of normal development. The condition is characterized by a loss of purposeful hand skills, distinctive hand movements, slowed growth, and problems with walking. Individuals with Rett Syndrome may also experience difficulties with communication and social engagement. As the syndrome progresses, motor skills and cognitive abilities can decline, leading to increased dependency on caregivers. Seizures, breathing irregularities, and other health issues may also occur as part of the condition's progression.