Stable VitiligoEarly Phase 1NCT06810869
Induced Pluripotent Stem Cell Derived Exosomes Nasal Drops for the Treatment of Stable Vitiligo
A Early Phase 1 study testing human induced pluripotent stem cell derived exosomes （GD-iExo-003） in people with Stable Vitiligo, run by Second Affiliated Hospital of Wannan Medical College at 1 site in China.
Trial summary
Evaluate the efficacy of induced pluripotent stem cell-derived exosomes (iPSC-Exos) for the treatment of stable vitiligo Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Stable Vitiligo
- What is being tested?
- human induced pluripotent stem cell derived exosomes （GD-iExo-003）
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 29 participants
- Where
- 1 siteChina
- Study dates
- 5 Feb 2025to 17 Nov 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Agree to follow the trial treatment protocol and visit schedule, voluntarily enroll in the study, and provide written informed consent;
- Aged 18-60 years old (inclusive of 18 and 60 years);
- Clinically diagnosed with stable localized vitiligo (in accordance with the 2021 Consensus on the Diagnosis and Treatment of Vitiligo); Stability criteria: ① A score of 0 on the Vitiligo Disease Activity (VIDA) scale; ② Clinical characteristics: white patches appear porcelain white, with clear or pigmented edges; ③ No Koebner phenomenon (for ≥1 year); ④ Wood's lamp examination: lesions appear white with clear boundaries, and the area under Wood's light is ≤ visual estimation area, indicating stability. Skin CT and dermatoscopy images may also be referenced for diagnostic assistance. Localization criteria: Refers to single lesions, with an area classified as grade 1 (lesion area <1% of total body surface area), where it is not yet possible to determine whether the presentation is segmental or non-segmental at the time of consultation.
- Lesions have been stable for more than 6 months, with a number of foci ≥3, ensuring that at least 2 lesions have an area >2cm²;
- Have no plans for conception during the study period and for 3 months after the last administration, and can adopt effective contraceptive measures;
- Agree to suspend any other anti-vitiligo treatments outside of the study protocol during participation in this clinical research (excluding cosmetic cover-ups).
Who cannot join
- Individuals with clinically significant multiple or severe drug allergies, or those who have had serious allergic reactions to treatments, or where the investigator anticipates that the participant may be allergic to the study medication or any of its components;
- Participants diagnosed with progressive vitiligo or moderate-to-severe stable vitiligo;
- Total depigmented area ≥1% of body surface area (BSA);
- Those who have received other treatments for vitiligo within the recent past (within 3 months), such as corticosteroids, JAK inhibitors, topical herbal medicines, depigmentation therapy, melanocyte-keratinocyte transplant surgery, phototherapy, etc.;
- Participants with other active pigmented skin diseases during the screening period, such as pityriasis alba, senile leukoderma, chemical/drug-induced vitiligo, hyperpigmentation due to malignancy, post-inflammatory hyperpigmentation, ataxia telangiectasia, tuberous sclerosis, melasma, congenital hypopigmentation disorders, etc.;
- Evidence of active inflammatory skin disease or skin condition during the screening period, such as atopic dermatitis, psoriasis, discoid lupus, leprosy, syphilis, seborrheic dermatitis, etc., which the investigator assesses might interfere with the evaluation of response and safety regarding vitiligo treatment;
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- Pregnant or breastfeeding women;
- Participants with a history or current diagnosis of other autoimmune diseases and major illnesses: tuberculosis, AIDS, malignancies, cardiovascular and cerebrovascular diseases, liver and kidney diseases, etc.;
- Individuals with a history of substance or alcohol abuse or those suffering from mental health conditions;
- Abnormal blood test results that the investigator judges could affect the assessment of efficacy and safety of the participant or the trial outcomes;
- Any other circumstances where the investigator believes that the participant's compliance may be affected or they are unsuitable to participate in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The Second Affiliated Hospital of Wannan Medical College
Wuhu, Anhui, 241000
Li Lin, 008618755377606, 18755377606@163.com
Ruzhi Zhang
About this study
Official title: Exploratory Clinical Study on Induced Pluripotent Stem Cell Derived Exosomes (iPSC-Exos) for the Treatment of Stable Vitiligo
Evaluate the efficacy of induced pluripotent stem cell-derived exosomes (iPSC-Exos) for the treatment of stable vitiligo
Read the detailed description
This study aims to evaluate the efficacy and safety of iPSC-Exos in the treatment of localized stable vitiligo. iPSC-Exos have been found to possess the ability to promote cell survival, proliferation, and repair, potentially improving skin pigmentation in vitiligo patients by modulating the survival and function of melanocytes.
This study will consist of 2 parts, with part 1 being a dose-escalation study and part 2 being an expanded safety study based on part 1 findings.
A traditional 3+3 dose escalation design will be implemented in part 1. Cohort 1: receive 1×10^8 particles per time; cohort 2: 3×10^8 particles per time and cohort 3: 9×10^8 particles per time.
In part 2, 20 subjects will be randomized in a 1:1 ratio [exosome (n=10) or exosome placebo (n=10)]. The dose level will be determined by Data Safety Monitoring Board based on part 1.
- Conditions
- Stable Vitiligo
- Being tested
- human induced pluripotent stem cell derived exosomes （GD-iExo-003）, a placebo of exosomes derived from human induced pluripotent stem cell for injection
- Keywords
- iPSC, exosome, Stable Vitiligo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human induced pluripotent stem cell derived exosomes （GD-iExo-003）Drug
human induced pluripotent stem cell derived exosomes （GD-iExo-003）
a placebo of exosomes derived from human induced pluripotent stem cell for injectionDrug
a placebo of exosomes derived from human induced pluripotent stem cell for injection
| Study group | Role | What they receive |
|---|---|---|
| Exosomes group | Experimental | group1-low-dose group, 3 patients will receive doses of GD-iExo-003 at 1×10^8 particles per 4 square centimeters of lesion area in 100 μL. group2-mid-dose group, 3 patients will receive doses of GD-iExo-003 at 3×10^8 particles per 4 square centimeters of lesion area in 100 μL. group3-high-dose group, 3 patients will receive doses of GD-iExo-003 at 9×10^8 particles per 4 square centimeters of lesion area in 100 μL. The administration method is microneedle injection, with each injection being 100 ul. Once every 2 weeks, for a total treatment duration of 3 months, with an additional follow-up period of 6 months Receives: Drug: human induced pluripotent stem cell derived exosomes （GD-iExo-003） |
| placebo group | Placebo comparator | Patients in this arm will be given a placebo. The administration method is microneedle injection, with each injection being 100 ul. Once every 2 weeks, for a total treatment duration of 3 months, with an additional follow-up period of 6 months. Receives: Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injection |
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: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Secondary outcomes (6)
Local Adverse Reactions
Changes in the lesions at the site of the white patches after treatment, such as erythema, blisters, erosion, scaling, pigmentation, Koebner phenomenon, etc
Measured: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Systemic Adverse Reactions
blood tests to check liver function indicators-Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)-and kidney function indicators-Creatinine (Cr), Blood Urea Nitrogen (BUN)
Measured: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Changes in the area of white patches
Using the grid counting method to calculate the area of white patches
Measured: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Vitiligo Area Scoring Index (VASI) change
VASI = Number of Hand Palm Units × Percentage of Depigmentation, with VASI values ranging from 0 to 100. One palm area is divided into 32 finger-joint units, where the palm center area accounts for 18 finger-joint units representing 0.54%, and one finger-joint unit represents 0.03%.
VASI Improvement Rate = (VASI score before treatment - VASI score after treatment) / VASI score before treatment × 100%. VASI50 is defined as an improvement in the VASI score of ≥50% from baseline; VASI75 is defined as an improvement in the VASI score of ≥75% from baseline.
Measured: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Vitiligo Noticeability Scale (VNS) change
This questionnaire includes assessments of appearance noticeability and psychosocial impact, with higher scores indicating a greater level of impact.
Measured: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Dermatology Life Quality Index (DLQI) change
The questionnaire contains 10 questions, with each option scored from 0 to 3. If a question is not relevant, it is also scored as 0. The score range is from 0 to 30, with higher scores indicating a greater impact on the quality of life.
Measured: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2025
- Study start5 Feb 2025 (estimated)
- Primary completion17 Nov 2026 (estimated)
- Study completion17 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stable VitiligoStudies of the same condition, open and under way.
- 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
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Second Affiliated Hospital of Wannan Medical College
- Sponsor type
- University, hospital or other
- Collaborators
- Guidon Pharmaceutics Ltd.
- Responsible party
- Principal investigator, Ruzhi Zhang, Second Affiliated Hospital of Wannan Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06810869
- Sponsor's protocol ID
- WYEFYLS2024121
- Status last verified
- Feb 2025
- First submitted
- 22 Jan 2025
- Last updated
- 6 Feb 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The study will not share individual participant data to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2025. 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 (iPSC-Exos) for the Treatment of Stable Vitiligo
- ClinicalTrials.gov ID
- NCT06810869
- Acronym / sponsor ID
- WYEFYLS2024121
- Conditions
- Stable Vitiligo
- Keywords
- iPSC, exosome, Stable Vitiligo
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- Second Affiliated Hospital of Wannan Medical CollegeUniversity, hospital or other
- Collaborators
- Guidon Pharmaceutics Ltd.
- Study start
- 5 Feb 2025 (estimated)
- Primary completion
- 17 Nov 2026 (estimated)
- Study completion
- 17 Nov 2026 (estimated)
- First posted
- 6 Feb 2025 (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 efficacy of induced pluripotent stem cell-derived exosomes (iPSC-Exos) for the treatment of stable vitiligo
Detailed description
This study aims to evaluate the efficacy and safety of iPSC-Exos in the treatment of localized stable vitiligo. iPSC-Exos have been found to possess the ability to promote cell survival, proliferation, and repair, potentially improving skin pigmentation in vitiligo patients by modulating the survival and function of melanocytes.
This study will consist of 2 parts, with part 1 being a dose-escalation study and part 2 being an expanded safety study based on part 1 findings.
A traditional 3+3 dose escalation design will be implemented in part 1. Cohort 1: receive 1×10^8 particles per time; cohort 2: 3×10^8 particles per time and cohort 3: 9×10^8 particles per time.
In part 2, 20 subjects will be randomized in a 1:1 ratio [exosome (n=10) or exosome placebo (n=10)]. The dose level will be determined by Data Safety Monitoring Board based on part 1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exosomes group | Experimental | Drug: human induced pluripotent stem cell derived exosomes （GD-iExo-003）group1-low-dose group, 3 patients will receive doses of GD-iExo-003 at 1×10^8 particles per 4 square centimeters of lesion area in 100 μL. group2-mid-dose group, 3 patients will receive doses of GD-iExo-003 at 3×10^8 particles per 4 square centimeters of lesion area in 100 μL. group3-high-dose group, 3 patients will receive doses of GD-iExo-003 at 9×10^8 particles per 4 square centimeters of lesion area in 100 μL. The administration method is microneedle injection, with each injection being 100 ul. Once every 2 weeks, for a total treatment duration of 3 months, with an additional follow-up period of 6 months |
| placebo group | Placebo comparator | Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injectionPatients in this arm will be given a placebo. The administration method is microneedle injection, with each injection being 100 ul. Once every 2 weeks, for a total treatment duration of 3 months, with an additional follow-up period of 6 months. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Agree to follow the trial treatment protocol and visit schedule, voluntarily enroll in the study, and provide written informed consent;
- Aged 18-60 years old (inclusive of 18 and 60 years);
- Clinically diagnosed with stable localized vitiligo (in accordance with the 2021 Consensus on the Diagnosis and Treatment of Vitiligo); Stability criteria: ① A score of 0 on the Vitiligo Disease Activity (VIDA) scale; ② Clinical characteristics: white patches appear porcelain white, with clear or pigmented edges; ③ No Koebner phenomenon (for ≥1 year); ④ Wood's lamp examination: lesions appear white with clear boundaries, and the area under Wood's light is ≤ visual estimation area, indicating stability. Skin CT and dermatoscopy images may also be referenced for diagnostic assistance. Localization criteria: Refers to single lesions, with an area classified as grade 1 (lesion area <1% of total body surface area), where it is not yet possible to determine whether the presentation is segmental or non-segmental at the time of consultation.
- Lesions have been stable for more than 6 months, with a number of foci ≥3, ensuring that at least 2 lesions have an area >2cm²;
- Have no plans for conception during the study period and for 3 months after the last administration, and can adopt effective contraceptive measures;
Key exclusion criteria (11 listed)
- Individuals with clinically significant multiple or severe drug allergies, or those who have had serious allergic reactions to treatments, or where the investigator anticipates that the participant may be allergic to the study medication or any of its components;
- Participants diagnosed with progressive vitiligo or moderate-to-severe stable vitiligo;
- Total depigmented area ≥1% of body surface area (BSA);
- Those who have received other treatments for vitiligo within the recent past (within 3 months), such as corticosteroids, JAK inhibitors, topical herbal medicines, depigmentation therapy, melanocyte-keratinocyte transplant surgery, phototherapy, etc.;
- Participants with other active pigmented skin diseases during the screening period, such as pityriasis alba, senile leukoderma, chemical/drug-induced vitiligo, hyperpigmentation due to malignancy, post-inflammatory hyperpigmentation, ataxia telangiectasia, tuberous sclerosis, melasma, congenital hypopigmentation disorders, 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 |
|---|---|---|
| human induced pluripotent stem cell derived exosomes （GD-iExo-003）human induced pluripotent stem cell derived exosomes （GD-iExo-003） | Drug | Exosomes group |
| a placebo of exosomes derived from human induced pluripotent stem cell for injectiona placebo of exosomes derived from human induced pluripotent stem cell for injection | Drug | placebo group |
Primary outcome
adverse events as assessed by CTCAE
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
all potentially treated subjects to assess the safety
6 secondary outcomes
Local Adverse Reactions
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Systemic Adverse Reactions
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Changes in the area of white patches
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Vitiligo Area Scoring Index (VASI) change
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Vitiligo Noticeability Scale (VNS) change
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Dermatology Life Quality Index (DLQI) change
Time frame: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2025.
- Individual participant dataSharing planned: No. The study will not share individual participant data to other researchers.
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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.
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 human induced pluripotent stem cell derived exosomes （GD-iExo-003） in people with Stable Vitiligo, run by Second Affiliated Hospital of Wannan Medical College 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 6 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: opening soon, as of the sponsor's last update on 6 Feb 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ruzhi Zhang, MD; PhD (05532863172, 18755377606@163.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: Screening and 1 day, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks, 14 weeks，18 weeks，22 weeks，24 weeks after the first injection). 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 6 Feb 2025.