Induced Pluripotent Stem Cell Derived Exosomes Nasal Drops for the Treatment of Stable Vitiligo

February 1, 2025 updated by: Ruzhi Zhang, Second Affiliated Hospital of Wannan Medical College

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

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

29

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Anhui
      • Wuhu, Anhui, China, 241000
        • The Second Affiliated Hospital of Wannan Medical College
        • Contact:
        • Principal Investigator:
          • Ruzhi Zhang

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Agree to follow the trial treatment protocol and visit schedule, voluntarily enroll in the study, and provide written informed consent;
  2. Aged 18-60 years old (inclusive of 18 and 60 years);
  3. 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.
  4. 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;
  5. Have no plans for conception during the study period and for 3 months after the last administration, and can adopt effective contraceptive measures;
  6. Agree to suspend any other anti-vitiligo treatments outside of the study protocol during participation in this clinical research (excluding cosmetic cover-ups).

Exclusion Criteria:

  1. 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;
  2. Participants diagnosed with progressive vitiligo or moderate-to-severe stable vitiligo;
  3. Total depigmented area ≥1% of body surface area (BSA);
  4. 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.;
  5. 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.;
  6. 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;
  7. Pregnant or breastfeeding women;
  8. 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.;
  9. Individuals with a history of substance or alcohol abuse or those suffering from mental health conditions;
  10. Abnormal blood test results that the investigator judges could affect the assessment of efficacy and safety of the participant or the trial outcomes;
  11. Any other circumstances where the investigator believes that the participant's compliance may be affected or they are unsuitable to participate in this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Exosomes group
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
Drug: human induced pluripotent stem cell derived exosomes （GD-iExo-003）
human induced pluripotent stem cell derived exosomes （GD-iExo-003）
Placebo Comparator: placebo group
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.
Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injection
a placebo of exosomes derived from human induced pluripotent stem cell for injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Changes in the lesions at the site of the white patches after treatment, such as erythema, blisters, erosion, scaling, pigmentation, Koebner phenomenon, etc
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
blood tests to check liver function indicators-Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)-and kidney function indicators-Creatinine (Cr), Blood Urea Nitrogen (BUN)
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
Using the grid counting method to calculate the area of white patches
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

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.
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
This questionnaire includes assessments of appearance noticeability and psychosocial impact, with higher scores indicating a greater level of impact.
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
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.
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Second Affiliated Hospital of Wannan Medical College

Collaborators

Guidon Pharmaceutics Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 5, 2025

Primary Completion (Estimated)

November 17, 2026

Study Completion (Estimated)

November 17, 2026

Study Registration Dates

First Submitted

January 22, 2025

First Submitted That Met QC Criteria

February 1, 2025

First Posted (Actual)

March 25, 2025

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 1, 2025

Last Verified

February 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • WYEFYLS2024121

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

The study will not share individual participant data to other researchers.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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