Ongoing Clinical Trials for CADASIL
Currently, there is 1 ongoing clinical trial investigating potential treatments for CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy). This trial is evaluating the medication Cerebrolysin to determine whether it may help improve symptoms or slow disease progression in patients with this genetic condition affecting the brain’s small blood vessels.
Clinical trial locations
Study on Cerebrolysin and Sodium Chloride for Patients with CADASIL
This clinical trial is examining whether Cerebrolysin, a medication given through intravenous infusion, can benefit patients with CADASIL. The condition affects the small blood vessels in the brain and can lead to strokes, cognitive difficulties, and mood changes over time. This study compares Cerebrolysin to a placebo to evaluate both its safety and effectiveness.
Main inclusion criteria:
- Patients must be at least 18 years old, of any gender
- Must have a confirmed diagnosis through symptoms, MRI scans, and genetic testing
- Must have a MoCA score greater than 11 (MoCA is a test that checks memory and thinking skills)
- Must have adequate vision, hearing, and language skills to understand and follow study procedures without needing a translator
- Women must either be post-menopausal for two years, have had surgery to prevent pregnancy, or be using effective birth control methods
- Must voluntarily agree to participate and provide written informed consent
Main exclusion criteria:
- Patients without genetic confirmation of the condition
- Individuals who are part of vulnerable populations, such as children, pregnant women, or those with certain disabilities requiring special protection
Focus and goal of the trial:
The study is structured in multiple phases over 27 months. Participants will receive either Cerebrolysin or placebo through intravenous infusion during the first 12-month treatment period. Regular assessments of cognitive abilities, mood, and brain imaging will occur at months 6 and 12. After the first phase, participants will switch to the alternate treatment (a crossover design) and continue for another 15 months, with assessments at months 21 and 27. The trial aims to gather comprehensive information about how Cerebrolysin affects brain health and overall well-being in people living with this challenging genetic condition.
Investigational drug:
Cerebrolysin is a neuroprotective medication believed to support brain health by promoting nerve cell repair and growth. In this trial, it is administered as a 215.2 mg/ml solution for injection through intravenous infusion. The medication is being studied for its potential to improve cognitive function and protect brain tissue in patients with conditions affecting the brain’s blood vessels.
Summary
Currently, only one clinical trial is actively recruiting patients with CADASIL. This trial is taking place in Czechia and focuses exclusively on evaluating Cerebrolysin as a potential treatment option. The study uses a crossover design, allowing all participants to receive both the investigational medication and placebo at different stages, which helps researchers better understand the treatment’s effects. The trial spans more than two years and includes comprehensive monitoring of cognitive function, mood, and brain imaging results. This research represents an important step in exploring new treatment possibilities for this rare genetic condition that currently has limited therapeutic options.
