Nasal drops using stem cell exosomes for treating refractory focal epilepsy

Exploratory Clinical Study on Induced Pluripotent Stem Cell Derived Exosomes (GD-iEXo-002) Nasal Drops for the Treatment of Refractory Focal Epilepsy

This study is testing nasal drops made from stem cell exosomes to see if they can help people with hard-to-treat focal epilepsy have fewer seizures.

Quick facts

• Phase: EARLY_PHASE1
• Study type: Interventional
• Enrollment: 34 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Peking Union Medical College Hospital (other)
• Locations: 1 site (Beijing, Beijing)
• Trial ID: NCT05886205 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety, tolerability, and preliminary efficacy of nasal drops containing induced pluripotent stem cell-derived exosomes (iPSC-Exos) for patients suffering from refractory focal epilepsy. The study aims to address the urgent need for effective treatments for the approximately 30% of epilepsy patients who do not respond to conventional medications. Participants will be monitored for their seizure frequency and overall response to the treatment over the course of the trial. The trial is conducted at a single center, ensuring a controlled environment for data collection and analysis.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 to 70 with focal epilepsy that is resistant to at least one anti-epileptic drug.

Not a fit: Patients who are not experiencing drug-resistant epilepsy or those who are pregnant or lactating may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with refractory focal epilepsy, potentially improving their quality of life.

How similar studies have performed: While the use of exosomes in treatment is a novel approach, similar studies have shown promise in other conditions, indicating potential for success in this area.

Eligibility criteria

Inclusion Criteria:

1. Before conducting this study, understand and sign the informed consent form and comply with the requirements of this study;
2. The subjects are patients with focal epilepsy aged 18 to 70 years (inclusive), regardless of gender;
3. The subjects must comply with the definition of drug resistant epilepsy (ILAE);
4. Subjects must take 1-6 types of anti-epileptic drugs (AEDs), and before entering the screening period, they must take unadjusted types and stable doses of AEDs for at least 4 weeks;
5. Throughout the entire research process, the subject/legal guardian must be able to accurately record the log of epileptic seizures;
6. Subjects must experience at least 4 countable seizures within 28 days prior to the screening period;
7. The subjects are willing and able to comply with the research requirements.

Exclusion Criteria:

1. Unwilling or unable to follow the procedures stipulated in the agreement;
2. Pregnant or lactating women, or women with reproductive potential who are unable or unwilling to take appropriate contraceptive measures;
3. Other uncontrollable diseases that may interfere with the research results, including but not limited to infection, hypertension, diabetes, cardiovascular and cerebrovascular diseases, etc;
4. There are situations that may increase the risk of participating in experiments or research product use, such as liver enzyme elevation exceeding twice the normal upper limit and/or GFR\<60 mL/min/1.73 m2;
5. Have a history of drug abuse, alcohol dependence, or smoking within one month;
6. Patients with status epilepticus within one month;
7. Have potential progressive nervous system disease, such as encephalitis, brain tumor, multiple sclerosis or dementia;
8. Patients who plan to undergo epilepsy surgery within six months;
9. Patients with abnormal or diseased nasal structures;
10. Patients with cerebrospinal fluid rhinorrhea;
11. Patients whose family members have not been able to proficiently and correctly master the nasal drip method through standardized training

Where this trial is running

Beijing, Beijing

• Peking Union Medical College Hospital — Beijing, Beijing, China (RECRUITING)

Study contacts

• Principal investigator: Xiaohong Han — Peking Union Medical College Hospital
• Study coordinator: Xue Zhao

Conditions: Refractory Focal Epilepsy