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

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What is this study about?

This clinical trial is focused on studying a condition known as refractory epilepsy, which is a type of epilepsy that does not respond well to standard treatments. The study also includes a specific form of epilepsy called Rasmussen Encephalitis, which is a rare, chronic inflammatory neurological disease. The treatment being tested in this trial is called AloCelyvir, which involves the use of special cells known as allogenic bone marrow-derived mesenchymal stem cells. These cells are modified in a laboratory setting to potentially help manage the condition.

The purpose of this study is to evaluate the safety and how well patients respond to this treatment. Participants in the trial will receive repeated doses of these stem cells through a method called intra-arterial infusion, which means the cells are delivered directly into the arteries. The study is designed for children and adolescents who have not had success with other epilepsy treatments. The trial will monitor participants over a period to observe any changes in their condition and to ensure the treatment is safe.

Throughout the study, researchers will be looking for any side effects that might occur from the treatment, as well as any improvements in the participants’ epilepsy symptoms. The trial aims to provide new insights into managing refractory epilepsy and Rasmussen Encephalitis, potentially offering new hope for those affected by these challenging conditions.

1 initial assessment

The initial assessment involves a comprehensive evaluation to confirm eligibility for the trial. This includes reviewing medical history and conducting necessary tests such as CMR (cardiac magnetic resonance), electrophysiological tests, PET (positron emission tomography), and neuropsychological evaluations.

2 informed consent

Informed consent is obtained from the patient or their legal guardian. This step ensures understanding of the trial’s purpose, procedures, potential risks, and benefits.

3 treatment administration

The treatment involves the administration of AloCelyvir, a cell suspension for injection. This product contains allogenic bone marrow-derived mesenchymal stem cells that have been transduced with icovir-5 and expanded outside the body.

The administration route is intra-arterial, meaning the cells are injected directly into an artery. The treatment is given in repeated doses, although the specific frequency and duration are determined by the trial protocol.

4 monitoring and follow-up

After each administration, monitoring is conducted to assess safety and tolerability. This includes observing for any dose-limiting toxicities, which are defined as grade 3 toxicities related to the stem cell infusion occurring within the first 28 days of treatment.

Regular follow-up visits are scheduled to evaluate the clinical response and any changes in the patient’s condition.

5 end of trial

The trial is estimated to conclude by December 31, 2027. At the end of the trial, a final assessment is conducted to evaluate the overall safety and effectiveness of the treatment.

Who Can Join the Study?

  • Acceptance of informed consent, which means agreeing to participate in the study after understanding all the details.
  • Children under 16 years of age can participate.
  • Having epilepsy that is caused by the immune system and does not respond to the usual treatments, such as anti-seizure medications and standard immune therapies. This includes cases where there are problems with movement or thinking. A special group includes those who have had epilepsy surgery due to Rasmussen’s Encephalitis.
  • Ability to undergo certain medical tests, such as CMR (a type of heart scan), electrophysiological tests (tests that measure electrical activity in the body), PET (a type of imaging test), and neuropsychological evaluations (tests that assess brain function).

Who Cannot Join the Study?

  • Patients who have a different type of epilepsy that is not related to the immune system.
  • Patients who have any other serious health condition that might interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent infection or illness that could affect the study results.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of allergic reactions to similar treatments.
  • Patients who are unable to follow the study procedures or attend the required visits.

Where you can join this trial?

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Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Spain Spain
Recruiting
24.01.2025

Trial locations

Autologous Mesenchymal Stem Cells are cells taken from the patient’s own body. In this trial, these cells are used to help treat children and adolescents with epilepsy that does not respond to other treatments. The cells are infused directly into the arteries to see if they can help reduce seizures and improve the patient’s condition. The main goal is to check if this method is safe and how well it works for these patients.

Investigated diseases:

Autoimmune Refractory Epilepsy – This is a type of epilepsy that does not respond to standard treatments and is believed to be caused by the immune system attacking the brain. It is characterized by frequent and severe seizures that are difficult to control. Over time, the condition can lead to persistent neurological symptoms due to ongoing inflammation and damage in the brain. The seizures can vary in type and severity, and they may occur without warning. The condition is chronic and requires ongoing management to address the symptoms and prevent further complications.

Rasmussen Encephalitis – This is a rare, chronic inflammatory neurological disease that typically affects one hemisphere of the brain. It is characterized by frequent and severe seizures, loss of motor skills and speech, paralysis on one side of the body, and cognitive decline. The disease usually progresses over time, leading to significant neurological impairment. The inflammation in the brain is thought to be caused by an autoimmune response, where the body’s immune system mistakenly attacks healthy brain tissue. The condition primarily affects children but can also occur in adults.

Trial ID:
2024-520427-86-00
Protocol code:
Rasmusscell
Trial Phase:
Human Pharmacology (Phase I) – First administration to humans

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