Induced Pluripotent Stem Cell Derived Exosomes Nasal Drops for the Treatment of Refractory Focal Epilepsy

NCT05886205 | Unknown Early Phase 1

• 1 other identifier: K2153-K22C1488
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

Evaluate the safety, tolerability, and preliminary efficacy of GD-iEXo-002 nasal drops in the treatment of focal refractory epilepsy

Conditions

Refractory Focal Epilepsy

Outcome Measures

Primary Outcomes (1)

• adverse events as assessed by CTCAE

  all potentially treated subjects to assess the safety

  24 weeks from post-administration

Secondary Outcomes (7)

• Number of participants with abnormal vital signs and abnormal Physical examination findings

  Screening, after the first administration 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16 weeks, 24 weeks

• Number of participants with abnormal Neurological examination

  Screening, after the first administration 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16 weeks, 24 weeks

• Number of participants with abnormal laboratory tests results

  Screening, after the first administration 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16 weeks, 24 weeks

• Number of participants with abnormal Urine analysis

  Screening, after the first administration 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16 weeks, 24 weeks

• Seizures frequency

  before administration; administration; after the first administration 1 week,2 weeks,4 weeks,8 weeks,12 weeks,16 weeks,24 weeks

Other Outcomes (2)

• Exploratory research-1

  Screening, after the first administration 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16 weeks, 24 weeks

• Exploratory research-2

  Screening, after the first administration 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16 weeks, 24 weeks

Study Arms (1)

iPSC-exosome treatment

EXPERIMENTAL

group1-low-dose group, 8 papatients are treated with 2 μg iPSC-Exos in 200 μL. group2-mid-dose group, 8 papatients are treated with 6 μg iPSC-Exos in 200 μL. group3-mid-dose group, 8 papatients are treated with 18 μg iPSC-Exos in 200 μL. group4-Dose expansion, 10 papatients are treated with iPSC-Exos in 200 μL. iPSC-Exos were administrated for nasal drip, bid for 12 weeks.

Drug: iPSC-Exos

Interventions

iPSC-Exos DRUG

iPSC-Exos were administrated for nasal drip, bid for 12 weeks.

iPSC-exosome treatment

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Before conducting this study, understand and sign the informed consent form and comply with the requirements of this study;
• The subjects are patients with focal epilepsy aged 18 to 70 years (inclusive), regardless of gender;
• The subjects must comply with the definition of drug resistant epilepsy (ILAE);
• 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;
• Throughout the entire research process, the subject/legal guardian must be able to accurately record the log of epileptic seizures;
• Subjects must experience at least 4 countable seizures within 28 days prior to the screening period;
• The subjects are willing and able to comply with the research requirements.

You may not qualify if:

• Unwilling or unable to follow the procedures stipulated in the agreement;
• Pregnant or lactating women, or women with reproductive potential who are unable or unwilling to take appropriate contraceptive measures;
• Other uncontrollable diseases that may interfere with the research results, including but not limited to infection, hypertension, diabetes, cardiovascular and cerebrovascular diseases, etc;
• 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;
• Have a history of drug abuse, alcohol dependence, or smoking within one month;
• Patients with status epilepticus within one month;
• Have potential progressive nervous system disease, such as encephalitis, brain tumor, multiple sclerosis or dementia;
• Patients who plan to undergo epilepsy surgery within six months;
• Patients with abnormal or diseased nasal structures;
• Patients with cerebrospinal fluid rhinorrhea;
• Patients whose family members have not been able to proficiently and correctly master the nasal drip method through standardized training

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead
• Guidon Pharmaceutics Ltd.: collaborator

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Related Publications (1)

• Li A, Zhao Z, Mi R, Xue G. The Evolving Role of Mesenchymal Stem Cells and Their Exosomes in Epilepsy Management: From Bench to Bedside. Stem Cells Int. 2026 Jan 28;2026:4989846. doi: 10.1155/sci/4989846. eCollection 2026.

  PMID: 41613983 DERIVED

MeSH Terms

Conditions

Epilepsies, Partial

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Xiaohong Han

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xue Zhao

CONTACT

+8601069154786; zhaoxue_pumch@sina.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2023
• First Posted: June 2, 2023
• Study Start: June 5, 2023
• Primary Completion: June 13, 2025
• Study Completion: November 13, 2025
• Last Updated: June 7, 2023

Record last verified: 2023-06

Locations

CN (1)