Not recruitingRare disease

Study on Cerebrolysin and Sodium Chloride for Patients with CADASIL

Verified siteRegistered drug

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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 a rare genetic condition called , which stands for Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy. This condition affects the small blood vessels in the brain, leading to strokes and other brain-related issues. The trial will test the effects of a treatment called , which is a solution for injection, to see if it can help patients with CADASIL. Cerebrolysin is compared to a placebo to evaluate its safety and effectiveness.

The purpose of the study is to assess the overall benefits and risks of using Cerebrolysin in patients who have been genetically confirmed to have CADASIL. Participants in the study will receive either Cerebrolysin or a placebo through intravenous infusion, which means the solution is given directly into a vein. The study will take place over two periods, each lasting several months, and will involve regular assessments of the participants' cognitive abilities, mood, and brain imaging results.

Throughout the study, participants will be monitored to see how they respond to the treatment. The trial aims to gather important information about how Cerebrolysin affects the brain and overall health of people with CADASIL. This research could provide valuable insights into potential new treatments for this challenging condition.

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
Participation begins after providing written informed consent.
Eligibility is confirmed based on age, diagnosis of CADASIL, cognitive assessment, and other criteria.
Step 2
Treatment phase I
Receive Cerebrolysin or placebo through an intravenous infusion.
Dosage: Cerebrolysin 215.2 mg/ml solution for injection.
Duration: Treatment continues for 12 months.
Assessments occur at months 6 and 12, focusing on cognition, mood, and imaging.
Step 3
Crossover and treatment phase II
Switch to the alternate treatment (placebo or Cerebrolysin).
Continue receiving treatment through intravenous infusion.
Duration: Treatment continues for another 15 months.
Assessments occur at months 21 and 27, focusing on cognition, mood, and imaging.
Step 4
End of study
Final assessments are conducted at the end of month 27.
The study concludes with a comprehensive evaluation of the treatment's safety and effectiveness.

Who can join the trial?

6 criteria

Patients must be **18 years or older**, and all genders are welcome.
Must have a diagnosis of **CADASIL**, which is confirmed through symptoms, MRI scans, and genetic tests.
Must have a **MoCA score greater than 11**. MoCA is a test that checks memory and thinking skills.
Must have good enough **vision, hearing, and language skills** to understand and follow the study procedures without needing a translator.
Women must not be able to have children. This means they are either post-menopausal for two years, have had surgery to prevent pregnancy, or are using effective birth control methods like hormonal contraception with a barrier method, an intrauterine device, or have a partner who has had a vasectomy. Sexual abstinence is also acceptable.
Must agree to participate **voluntarily** and provide **written informed consent**.

Who cannot join the trial?

2 criteria

Patients who do not have a genetic confirmation of **CADASIL**. This means that if you haven't been tested and confirmed to have this specific genetic condition, you cannot participate.
Patients who are part of a vulnerable population. This refers to groups of people who might need special protection or care, such as children, pregnant women, or those with certain disabilities.

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Verified sites

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Fakultni nemocnice Motol a Homolka
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Prague, Czechia

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Investigated drugs

Cerebrolysin is a medication being studied for its potential benefits in patients with CADASIL, a genetic condition that affects blood flow in the brain. The trial aims to assess the safety and effectiveness of Cerebrolysin in improving symptoms or slowing the progression of this condition.

What is already known about the treatment

Cerebrolysin – Cerebrolysin is administered through intravenous infusion, typically in a clinical setting. It is currently being studied in clinical trials for its safety and effectiveness in treating Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL). The medication is believed to support brain health by promoting nerve cell repair and growth, potentially improving cognitive function. It is classified as a neuroprotective agent, aiming to protect and enhance brain function in patients with neurological conditions.

Investigated diseases

Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) – This is a genetic disorder that affects the small blood vessels in the brain. It is characterized by the thickening of the walls of these vessels, which can lead to reduced blood flow and damage to the brain tissue. Over time, individuals with CADASIL may experience a range of symptoms, including migraines, strokes, and cognitive decline. The disease often progresses with recurrent strokes and can lead to difficulties with thinking and memory. Mood disturbances, such as depression, may also occur as the disease advances. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder.