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Refractory Focal EpilepsyEarly Phase 1NCT05886205

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

A Early Phase 1 study testing iPSC-Exos in people with Refractory Focal Epilepsy, run by Peking Union Medical College Hospital at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

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

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Refractory Focal Epilepsy
What is being tested?
iPSC-Exos
Could I receive a placebo?
No, everyone receives an active treatment
How many people
34 participants
Where
1 siteChina
Study dates
5 Jun 2023to 13 Nov 2025
Phase
Early Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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;
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  • 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

Trial locations and contacts

1 site in 1 country.

Xue Zhao contact

+8601069154786

zhaoxue_pumch@sina.com

China1 site, 1 recruiting

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100730

    Yang Zhang, +8601069154126

    Recruiting

Other trials in China

About this study

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

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

Read the detailed description

Epilepsy patients can achieve good control after treatment, but still 30% of patients are medically refractory epilepsy, with the vast majority being focal epilepsy. Recurrent seizures seriously affect the normal development, learning, and life of patients. There is an urgent need for effective drugs to treat refractory focal epilepsy in clinical practice.

Exosomes are a kind of vesicle structures secreted by cells, with a diameter of 30-150 nm, carrying proteins, nucleic acids and other substances. Exosomes have many advantages. As naturally occurring nanoscale secretory membrane vesicles, they have extremely low immunogenicity and good safety, and can cross biological barriers such as the blood-brain barrier and the blood-tumor barrier. Exosomes have specific bioactive substances related to source cells, while stem cell exosomes contain TGF- β、 Functional factors such as BDNF can inhibit cell apoptosis, inhibit inflammatory response, promote angiogenesis, inhibit fibrosis, and enhance tissue repair potential, with a wide range of potential applications.

Induced pluripotent stem cell (iPSC) originates from single cell amplification, with infinite proliferation ability, good consistency and stability; MSCs exhibit significant heterogeneity.

The purpose of this single center, open label clinical trial is to evaluate the safety, tolerability, and preliminary efficacy of induced pluripotent stem cell derived exosomes (iPSC-Exos) nasal drops in the treatment of focal refractory epilepsy.

Being tested
iPSC-Exos
Medical subject headings
Epilepsies, Partial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • iPSC-ExosDrug

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

Study groups in NCT05886205
Study groupRoleWhat they receive
iPSC-exosome treatmentExperimentalgroup1-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.
Receives: Drug: iPSC-Exos

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. adverse events as assessed by CTCAE

    all potentially treated subjects to assess the safety

    Measured: 24 weeks from post-administration

Secondary outcomes (7)

  1. Number of participants with abnormal vital signs and abnormal Physical examination findings

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

  2. Number of participants with abnormal Neurological examination

    Evaluate general neurological status, muscle strength and tension, sensory ataxia, and pathological signs

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

  3. Number of participants with abnormal laboratory tests results

    Blood routine test, blood biochemistry test ,and electrolytes test

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

  4. Number of participants with abnormal Urine analysis

    Urine routine examination

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

  5. Seizures frequency

    Seizure frequency:

    1. no seizures: any type of seizure disappeared after 28 days of observation; ii. significantly effective: 75%-99% reduction in seizure frequency compared with baseline; iii. Effective: 50%-75% reduction in seizure frequency compared with baseline; iv. Improvement: 25%-50% reduction in the number of seizures compared with baseline, or prolongation, reduction in degree, and shortening of duration between episodes; v. Ineffectiveness and exacerbation: Ineffectiveness refers to a decrease or increase of <25% in the number of seizures compared with baseline, and exacerbation refers to an increase in the frequency of seizures from baseline ≥25%.

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

  6. Scalp electroencephalogram monitoring

    Measured: Screening, after the first administration 12 weeks

  7. Head magnetic resonance imaging (MRI) examination

    Measured: Screening, after the first administration 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jun 2023
  2. Study start5 Jun 2023 (estimated)
  3. Primary completion13 Jun 2025 (estimated)
  4. Study completion13 Nov 2025 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
34 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05886205
Sponsor's protocol ID
K2153-K22C1488
Status last verified
Jun 2023
First submitted
8 May 2023
Last updated
7 Jun 2023
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • 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.

    PubMed 41613983

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing iPSC-Exos in people with Refractory Focal Epilepsy, run by Peking Union Medical College Hospital at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 7 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Xue Zhao (+8601069154786, zhaoxue_pumch@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

adverse events as assessed by CTCAE (time frame: 24 weeks from post-administration). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05886205, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jun 2023.

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