Clinical Trials for Corticobasal Degeneration
There are currently 2 ongoing clinical trials investigating new treatments for corticobasal degeneration. These studies are testing different approaches, including psilocybin therapy for psychological distress and glycerol phenylbutyrate for reducing nerve damage markers. Trials are being conducted in Germany, Czechia, Netherlands, Denmark, and Portugal. (Also known as: Corticobasal Syndrome, CBS)
Clinical trial locations
- Czechia
- Denmark
- Germany
- Netherlands
- Portugal
Study of psilocybin therapy for psychological distress in patients with COPD, ALS, multiple sclerosis, or atypical Parkinson’s disorders
This trial is investigating whether psilocybin therapy can help reduce symptoms of depression in patients with serious progressive conditions, including atypical Parkinson’s disorders such as corticobasal syndrome. The study will test different doses of psilocybin combined with psychological support to help patients cope with the emotional challenges of their illness.
Main inclusion criteria: To join this study, you must be at least 18 years old with a confirmed diagnosis of an atypical Parkinson disorder, which includes corticobasal syndrome. For patients with atypical Parkinson disorders specifically, you must be in the advanced to late stage of the condition and show evidence of both motor and non-motor symptoms that change over time. You must also have been diagnosed with major depressive disorder and score higher than 19 on a depression rating scale called MADRS. Additionally, you need to have a life expectancy of at least 6 months, be able to attend all study visits, have an identified caregiver or support person, and be able to swallow capsules.
Main exclusion criteria: You cannot participate if you have a personal or family history of certain severe mental health conditions like schizophrenia, psychosis, or bipolar disorder. Other reasons for exclusion include having treatment-resistant depression with psychotic features, suicide attempts in the past year or current suicidal thoughts with specific plans, active substance abuse problems in the past 6 months, severe heart conditions, uncontrolled high blood pressure, severe liver or kidney problems, pregnancy or breastfeeding, or if you are currently taking medications that could interact with psilocybin, including certain antidepressants.
Focus and goals: The main goal of this research is to evaluate whether medium to high doses of psilocybin therapy are safe and effective in reducing depression symptoms compared to low doses in patients with serious medical conditions. You will receive two doses of psilocybin capsules during the study, with your first dose being either 1 mg (low dose) or 15-25 mg (medium or high dose). Six weeks after your second dose, researchers will evaluate your depression symptoms again using the same scale as the initial assessment. Throughout the study, researchers will monitor changes in your psychological well-being, your ability to cope with end-of-life concerns, your overall quality of life, and your general mental health. The treatment period will last approximately two months, with follow-up evaluations to track your progress.
Investigational drug: Psilocybin is a naturally occurring psychedelic compound that is being studied as a potential therapy for psychological distress and depression. In this trial, it is being investigated as a treatment for patients with atypical Parkinson disorders who are experiencing depression. The therapy involves administering psilocybin in different doses along with psychological support to help patients process their experiences and potentially reduce their depressive symptoms. The substance works by binding to serotonin receptors in the brain, which can lead to altered mood and perception in ways that may help reduce psychological distress.
Study on the Effects of Glycerol Phenylbutyrate in Patients with Corticobasal Syndrome
This clinical trial is studying a treatment called glycerol phenylbutyrate in patients with corticobasal syndrome. The study aims to see if this medication can help reduce levels of a protein called neurofilament light chain, which is a marker that can indicate nerve cell damage.
Main inclusion criteria: You must be 18 years or older with a confirmed diagnosis of corticobasal syndrome or progressive supranuclear palsy-CBS. You should not have regularly used glycerol phenylbutyrate in the last 6 months. You need to be able to understand the study information and provide informed consent, and be willing to follow all trial procedures. Women who can have children must use highly effective birth control methods and have a negative pregnancy test. You must have been taking stable doses of your current medications for at least 1 month before starting the trial, including any medications for Parkinson-like symptoms such as Levodopa and Dopamine-Agonists, as well as other medications like antidepressants or antidementia drugs. These medications should not need to be changed during the 26-week treatment period.
Main exclusion criteria: You cannot participate if you do not have a diagnosis of corticobasal syndrome, are not within the specified age range, or are part of a vulnerable population such as children or pregnant women. There may be other specific health criteria that could exclude you from the study.
Focus and goals: The primary goal of this study is to assess whether glycerol phenylbutyrate can help reduce levels of neurofilament light chain in your blood. This protein is a marker of nerve cell damage, so lowering its levels might indicate that the treatment is helping protect nerve cells. The study will last 26 weeks, during which you will be randomly assigned to receive either the active medication or a placebo. Neither you nor the researchers will know which treatment you are receiving to ensure unbiased results. Throughout the study, researchers will regularly monitor your safety through physical and neurological examinations and vital sign checks. They will also observe any changes in clinical scales that measure movement and cognitive abilities, as well as your quality of life.
Investigational drug: Glycerol phenylbutyrate is an oral liquid medication that is being studied for its potential to lower neurofilament light chain levels in patients with corticobasal syndrome. It is classified as a nitrogen-binding agent and is traditionally used in the management of urea cycle disorders. At the molecular level, it works by modulating metabolic pathways that may influence the production or clearance of neurofilament light chain. This phase II study will help determine if the medication is effective, safe, and well-tolerated over a 26-week period.
Summary
Currently, there are 2 ongoing clinical trials exploring different treatment approaches for corticobasal degeneration. One trial is testing psilocybin therapy to help manage depression and psychological distress in patients with atypical Parkinson’s disorders, including corticobasal syndrome. This multi-country study is being conducted in Czechia, Netherlands, Denmark, and Portugal. The second trial, taking place in Germany, is investigating glycerol phenylbutyrate as a potential treatment to reduce markers of nerve cell damage.
These trials represent two distinct therapeutic strategies: one focusing on improving quality of life by addressing the psychological burden of the disease, and the other attempting to slow disease progression by protecting nerve cells. The variety of approaches reflects ongoing efforts to find effective treatments for this rare and challenging condition. Patients interested in participating should discuss eligibility criteria and potential benefits and risks with their healthcare providers.
