In short

Currently, there is one ongoing clinical trial investigating a novel stem cell-based treatment for children and adolescents with Rasmussen encephalitis, a rare inflammatory brain disease. This trial is being conducted in Spain and focuses on evaluating the safety of repeated intra-arterial infusion of mesenchymal stem cells in patients whose epilepsy does not respond to standard treatments.

Clinical trial locations

Spain
- Study on the Safety of Repeated Intra-Arterial Infusion of Mesenchymal Stem Cells for Children and Adolescents with Refractory Epilepsy

Study on the Safety of Repeated Intra-Arterial Infusion of Mesenchymal Stem Cells for Children and Adolescents with Refractory Epilepsy

This clinical trial is investigating a new treatment approach for children and adolescents with refractory epilepsy, including those with Rasmussen encephalitis. Refractory epilepsy means the condition does not respond well to standard treatments, making it particularly challenging to manage.

Main inclusion criteria:

Children under 16 years of age
Epilepsy caused by immune system problems that does not respond to usual anti-seizure medications and standard immune therapies
Patients who have had epilepsy surgery due to Rasmussen encephalitis are specifically included
Ability to undergo necessary medical tests including heart scans, electrical activity tests, imaging tests, and brain function assessments
Written agreement (informed consent) from the patient or legal guardian to participate in the study

Main exclusion criteria:

Patients with epilepsy that is not related to the immune system
Those with other serious health conditions that might interfere with the study
Patients currently participating in another clinical trial
Recent infection or illness that could affect study results
Pregnant or breastfeeding patients
History of allergic reactions to similar treatments
Inability to follow study procedures or attend required visits

Trial focus and goal: The primary purpose of this study is to evaluate the safety and tolerability of a treatment called AloCelyvir. The trial monitors patients closely for any side effects, particularly within the first 28 days after treatment. The study also observes any changes in epilepsy symptoms and overall condition. The trial involves several stages, starting with a comprehensive initial assessment, followed by obtaining informed consent, then the actual treatment administration, ongoing monitoring, and finally a complete evaluation at the end of the trial, which is expected to conclude by December 31, 2027.

Investigational treatment: The treatment being tested uses mesenchymal stem cells, which are special cells taken from the patient's own body (autologous cells). These cells are modified in a laboratory setting and then infused directly into the arteries through a procedure called intra-arterial infusion. The treatment is given in repeated doses according to the trial protocol. These stem cells are believed to work by modulating the immune system and promoting repair of damaged brain tissue, potentially reducing inflammation and seizure activity. This represents a novel approach to treating refractory epilepsy and Rasmussen encephalitis, offering hope where conventional treatments have not been successful.

Summary

There is currently one active clinical trial available for patients with Rasmussen encephalitis, located in Spain. This Phase I trial represents an innovative approach to treating this rare and challenging condition by using stem cell therapy. The trial is specifically designed for children and adolescents whose epilepsy has not responded to conventional treatments, including those who have already undergone epilepsy surgery.

The focus on stem cell-based therapy reflects growing interest in cell-based treatments for neurological conditions. The trial's emphasis on safety evaluation is appropriate for this early-phase study, as this type of treatment is still experimental. Patients interested in participating should be aware that the trial requires the ability to undergo various medical tests and attend regular follow-up visits through the end of 2027.

Rasmussen encephalitis Trials in Disease

Clinical trials for Rasmussen encephalitis