Atopic DermatitisEarly Phase 1NCT05969717
Induced Pluripotent Stem Cell Derived Exosomes for the Treatment of Atopic Dermatitis
A Early Phase 1 study testing GD-iExo-001 and normal saline in people with Atopic Dermatitis, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
Evaluate the safety, tolerability, and preliminary efficacy of GD-iExo-001 in the treatment of atopic dermatitis Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Atopic Dermatitis
- What is being tested?
- GD-iExo-001 and normal saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 12 Apr 2023to 30 Jun 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
- Age 18 to 70 years, body mass index (BMI) between 18 and 35 kg/m2 (including boundary values);
- Overall good health except AD;
- Participants diagnosed with atopic dermatitis (AD) as defined by the Hanifin and Rajka criteria;
- Investigator overall assessment (IGA) score of 2 to 3;
- Participants with a history of subacute or chronic AD symptoms for at least 6 months;
- Participants with body surface area (BSA) of AD involvement of ≤5% at screening and baseline;
- Participants and their partners agreed to use effective contraception throughout the study period (from screening to 3 months after completion of treatment);
- Participants understood and voluntarily signed the informed consent form.
Who cannot join
- There are obvious active systemic or local infections, including but not limited to the infection of AD secondary infection, local bacterial infection in target lesion, local viral infection in target lesion, and local fungal infection in target lesion. Note: After the infection resolves, the patients can be re-screened;
- Presence of any of the following conditions: HB surface antigen (HBsAg) positive and / or HB e antigen (HBeAg) positive, HB e antibody (HBeAb) and / or hepatitis B core antibody (HBcAb) positive and hepatitis B virus deoxyribonucleic acid (HBV-DNA) copy number> 2000 IU / mL; limited hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA positive; positive human immunodeficiency virus (HIV) antibody; positive for syphilis (TPA) test;
- Inoculate live or attenuated vaccine within 4 weeks before screening or during the study period;
- Received allergen specific immunotherapy within 6 months before screening;
- Use of topical drugs known or may affect AD within 2 weeks before screening (including but not limited to topical glucocorticoids; calcineurin inhibitors: such as tacrolimus, pirolimus, etc.);
- Use of immunosuppressants and Janus kinase inhibitors within 4 weeks before screening;
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- Use of any biological agent (such as IL-4 receptor inhibitors, IL-13 inhibitors) for 12 weeks before screening or 5 half-lives (whichever is longer);
- Received systemic or local Chinese medicine treatment (including Chinese medicine immersion treatment) within 2 weeks before screening;
- Treated with UV and photochemistry within 4 weeks prior to screening;
- Required systemic treatment of antiviral, antiparasitic, antigenic, or antifungals within 4 weeks prior to screening;
- With significant abnormal findings or laboratory values and clinical significance, such as white blood cell count <3.0e9 / L; neutrophils <1 LLN; hemoglobin <90g / L; platelet <100e9 / L; serum creatinine> 1.5 ULN, ALT or AST 2 ULN; QTcF> 450 msec (male) or QTcF> 470 msec (female);
- Other combined (or co-occurrence) skin diseases that may affect the study evaluation, such as acne, psoriasis, lupus erythematosus, etc.; or large tattoos, birthmarks, skin scars, skin ulcers and other conditions that may affect the judgment of the investigator;
- Except for AD, any history of clinical major disease or clinically significant circulatory system abnormality, endocrine system abnormality, neurological or hematological disorders, immune system disease, psychiatric illness and metabolic instability; clinical significance is defined as the risk of the safety of the subject or aggravating the disease / disease during the study;
- Patients with a history of severe skin allergy and / or allergy to any ingredients of the product;
- History of cancer in the past 5 years (except for surgically removed squamous cell carcinoma, basal cell carcinoma, or skin carcinoma in situ);
- Any major surgery within 8 weeks before screening; or subjects expected to undergo surgery during the trial or within 4 weeks after the end of the trial; or 400 mL nonphysiological blood loss (including trauma, blood collection, blood donation within 12 weeks before screening);
- Pregnant women, lactating women, or women who plan to become pregnant during the study (fertile women must have a urine pregnancy test with negative results);
- Subjects who are currently participating in other clinical trials or who have participated in other clinical trials within 90 days;
- The investigator judged any other condition not suitable to participate in this study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Chinese Academy of Medical Science & Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Ju Qiao, PhD, +8615201125788, qiaoqiaoju@aliyun.com
About this study
Official title: Exploratory Clinical Study on Induced Pluripotent Stem Cell Derived Exosomes (GD-iExo-001) for the Treatment of Atopic Dermatitis
Evaluate the safety, tolerability, and preliminary efficacy of GD-iExo-001 in the treatment of atopic dermatitis
Read the detailed description
Atopic dermatitis (AD) is a chronic inflammatory skin disease that is characterised by intense itching and recurrent eczematous lesions. Although it most often starts in infancy and affects two of ten children, it is also highly prevalent in adults. It is the leading non-fatal health burden attributable to skin diseases, inflicts a substantial psychosocial burden on patients and their relatives, and increases the risk of food allergy, asthma, allergic rhinitis, other immune-mediated inflammatory diseases, and mental health disorders. Originally regarded as a childhood disorder mediated by an imbalance towards a T-helper-2 response and exaggerated IgE responses to allergens, it is now recognised as a lifelong disposition with variable clinical manifestations and expressivity, in which defects of the epidermal barrier are central. Present prevention and treatment focus on restoration of epidermal barrier function, which is best achieved through the use of emollients. Topical corticosteroids are still the first-line therapy for acute flares, but they are also used proactively along with topical calcineurin inhibitors to maintain remission.
Exosomes are nano-sized biovesicles released into surrounding body fluids upon fusion of multivesicular bodies and the plasma membrane. They were shown to carry cell-specific cargos of proteins, lipids, and genetic materials, and can be selectively taken up by neighboring or distant cells far from their release, reprogramming the recipient cells upon their bioactive compounds. Therefore, the regulated formation of exosomes, specific makeup of their cargo, cell-targeting specificity are of immense biological interest considering extremely high potential of exosomes as non-invasive diagnostic biomarkers, as well as therapeutic nanocarriers.
Induced pluripotent stem cells (iPSCs) are pluripotent stem cells generated from adult cells by reprogramming. iPSCs have the same properties as embryonic stem cells, and therefore self-renew and can differentiate into all cell types of the body except for cells in extra-embryonic tissues such as the placenta. iPSC-derived exosomes contain TGF- β、 BDNF etc., which can inhibit cell apoptosis, inhibit inflammatory response, promote angiogenesis, inhibit fibrosis, and enhance tissue repair potential, with a wide range of potential applications. iPSC-derived exosomes have emerged as an important paracrine factor for iPSCs to repair injured cells through the delivery of bioactive components.
The purpose of this trial is to evaluate the safety, tolerability, and preliminary efficacy of iPSC-derived exosomes in the treatment of atopic dermatitis.
- Conditions
- Atopic Dermatitis
- Being tested
- GD-iExo-001, normal saline
- Medical subject headings
- Dermatitis, Atopic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GD-iExo-001Drug
One drop (about 50 μL) of GD-iExo-001 was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day.
normal salineOther
One drop (about 50 μL) of normal saline was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day.
| Study group | Role | What they receive |
|---|---|---|
| GD-iExo-001 treatment | Experimental | Group1 (low-dose group), 8 papatients are treated with 10 μg/mL GD-iExo-001. Group2 (high-dose group), 8 papatients are treated with 50 μg/mL GD-iExo-001. One drop (about 50 μL) of GD-iExo-001 was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day. Receives: Drug: GD-iExo-001 |
| Normal saline control | Placebo comparator | Group1 (low-dose group), 2 papatients are treated with normal saline. Group2 (high-dose group), 2 papatients are treated with normal saline. One drop (about 50 μL) of normal saline was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day. Receives: Other: normal saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
adverse events as assessed by CTCAE
all potentially treated subjects to assess the safety
Measured: 42 days from post-administration
Secondary outcomes (7)
The proportion of subjects whose IGA score improved by 2 points or more compared with the baseline score.
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects with an IGA score of 0-1 and an improvement of 2 or more over the baseline score
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects whose EASI score improved by more than 50% compared with the baseline period
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects whose EASI score improved by more than 75% compared with the baseline period
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects whose EASI score improved by more than 90% compared with the baseline period
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
Change in the score of the NRS
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
Change in the score of the DLQI
Measured: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2023
- Study start12 Apr 2023
- Primary completion30 Apr 2025 (estimated)
- Study completion30 Jun 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Atopic DermatitisStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Guidon Pharmaceutics Ltd.
- Responsible party
- Sponsor
- Investigators
- Hongzhong Jin, MD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05969717
- Sponsor's protocol ID
- K3016
- Status last verified
- Jul 2023
- First submitted
- 23 Jul 2023
- Last updated
- 1 Aug 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploratory Clinical Study on Induced Pluripotent Stem Cell Derived Exosomes (GD-iExo-001) for the Treatment of Atopic Dermatitis
- ClinicalTrials.gov ID
- NCT05969717
- Acronym / sponsor ID
- K3016
- Conditions
- Atopic Dermatitis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Collaborators
- Guidon Pharmaceutics Ltd.
- Study start
- 12 Apr 2023 (actual)
- Primary completion
- 30 Apr 2025 (estimated)
- Study completion
- 30 Jun 2025 (estimated)
- First posted
- 1 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluate the safety, tolerability, and preliminary efficacy of GD-iExo-001 in the treatment of atopic dermatitis
Detailed description
Atopic dermatitis (AD) is a chronic inflammatory skin disease that is characterised by intense itching and recurrent eczematous lesions. Although it most often starts in infancy and affects two of ten children, it is also highly prevalent in adults. It is the leading non-fatal health burden attributable to skin diseases, inflicts a substantial psychosocial burden on patients and their relatives, and increases the risk of food allergy, asthma, allergic rhinitis, other immune-mediated inflammatory diseases, and mental health disorders. Originally regarded as a childhood disorder mediated by an imbalance towards a T-helper-2 response and exaggerated IgE responses to allergens, it is now recognised as a lifelong disposition with variable clinical manifestations and expressivity, in which defects of the epidermal barrier are central. Present prevention and treatment focus on restoration of epidermal barrier function, which is best achieved through the use of emollients. Topical corticosteroids are still the first-line therapy for acute flares, but they are also used proactively along with topical calcineurin inhibitors to maintain remission.
Exosomes are nano-sized biovesicles released into surrounding body fluids upon fusion of multivesicular bodies and the plasma membrane. They were shown to carry cell-specific cargos of proteins, lipids, and genetic materials, and can be selectively taken up by neighboring or distant cells far from their release, reprogramming the recipient cells upon their bioactive compounds. Therefore, the regulated formation of exosomes, specific makeup of their cargo, cell-targeting specificity are of immense biological interest considering extremely high potential of exosomes as non-invasive diagnostic biomarkers, as well as therapeutic nanocarriers.
Induced pluripotent stem cells (iPSCs) are pluripotent stem cells generated from adult cells by reprogramming. iPSCs have the same properties as embryonic stem cells, and therefore self-renew and can differentiate into all cell types of the body except for cells in extra-embryonic tissues such as the placenta. iPSC-derived exosomes contain TGF- β、 BDNF etc., which can inhibit cell apoptosis, inhibit inflammatory response, promote angiogenesis, inhibit fibrosis, and enhance tissue repair potential, with a wide range of potential applications. iPSC-derived exosomes have emerged as an important paracrine factor for iPSCs to repair injured cells through the delivery of bioactive components.
The purpose of this trial is to evaluate the safety, tolerability, and preliminary efficacy of iPSC-derived exosomes in the treatment of atopic dermatitis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GD-iExo-001 treatment | Experimental | Drug: GD-iExo-001Group1 (low-dose group), 8 papatients are treated with 10 μg/mL GD-iExo-001. Group2 (high-dose group), 8 papatients are treated with 50 μg/mL GD-iExo-001. One drop (about 50 μL) of GD-iExo-001 was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day. |
| Normal saline control | Placebo comparator | Other: normal salineGroup1 (low-dose group), 2 papatients are treated with normal saline. Group2 (high-dose group), 2 papatients are treated with normal saline. One drop (about 50 μL) of normal saline was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age 18 to 70 years, body mass index (BMI) between 18 and 35 kg/m2 (including boundary values);
- Overall good health except AD;
- Participants diagnosed with atopic dermatitis (AD) as defined by the Hanifin and Rajka criteria;
- Investigator overall assessment (IGA) score of 2 to 3;
- Participants with a history of subacute or chronic AD symptoms for at least 6 months;
Key exclusion criteria (19 listed)
- There are obvious active systemic or local infections, including but not limited to the infection of AD secondary infection, local bacterial infection in target lesion, local viral infection in target lesion, and local fungal infection in target lesion. Note: After the infection resolves, the patients can be re-screened;
- Presence of any of the following conditions: HB surface antigen (HBsAg) positive and / or HB e antigen (HBeAg) positive, HB e antibody (HBeAb) and / or hepatitis B core antibody (HBcAb) positive and hepatitis B virus deoxyribonucleic acid (HBV-DNA) copy number> 2000 IU / mL; limited hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA positive; positive human immunodeficiency virus (HIV) antibody; positive for syphilis (TPA) test;
- Inoculate live or attenuated vaccine within 4 weeks before screening or during the study period;
- Received allergen specific immunotherapy within 6 months before screening;
- Use of topical drugs known or may affect AD within 2 weeks before screening (including but not limited to topical glucocorticoids; calcineurin inhibitors: such as tacrolimus, pirolimus, etc.);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GD-iExo-001One drop (about 50 μL) of GD-iExo-001 was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day. | Drug | GD-iExo-001 treatment |
| normal salineOne drop (about 50 μL) of normal saline was given to the affected skin area of 2-4 cm2 for 14 consecutive days, twice per day. | Other | Normal saline control |
Primary outcome
adverse events as assessed by CTCAE
Time frame: 42 days from post-administration
all potentially treated subjects to assess the safety
7 secondary outcomes
The proportion of subjects whose IGA score improved by 2 points or more compared with the baseline score.
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects with an IGA score of 0-1 and an improvement of 2 or more over the baseline score
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects whose EASI score improved by more than 50% compared with the baseline period
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects whose EASI score improved by more than 75% compared with the baseline period
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
The proportion of subjects whose EASI score improved by more than 90% compared with the baseline period
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
Change in the score of the NRS
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
Change in the score of the DLQI
Time frame: Screening, after the first administration 8 day, 15 days, 21 days, 42 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hongzhong Jin, MD principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing GD-iExo-001 and normal saline in people with Atopic Dermatitis, 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 8 inclusion and 19 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 1 Aug 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. Some participants in this study may receive a placebo. 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 Ju Qiao, PhD (+8615201125788, qiaoqiaoju@aliyun.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: 42 days from post-administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Aug 2023.