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

NCT06810869 | Not Yet Recruiting Early Phase 1

• 1 other identifier: WYEFYLS2024121
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

Evaluate the efficacy of induced pluripotent stem cell-derived exosomes (iPSC-Exos) for the treatment of stable vitiligo

Conditions

Stable Vitiligo

Keywords

iPSC; exosome; Stable Vitiligo

Outcome Measures

Primary Outcomes (1)

• adverse events as assessed by CTCAE

  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 Outcomes (6)

• Local Adverse Reactions

  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

  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

  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

  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

  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

Study Arms (2)

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

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.

Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injection

Interventions

human induced pluripotent stem cell derived exosomes （GD-iExo-003） DRUG

human induced pluripotent stem cell derived exosomes （GD-iExo-003）

Exosomes group

a placebo of exosomes derived from human induced pluripotent stem cell for injection DRUG

a placebo of exosomes derived from human induced pluripotent stem cell for injection

placebo group

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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;
• 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.

Sponsors & Collaborators

• Second Affiliated Hospital of Wannan Medical College: lead
• Guidon Pharmaceutics Ltd.: collaborator

Study Sites (1)

The Second Affiliated Hospital of Wannan Medical College

Wuhu, Anhui, 241000, China

Location

MeSH Terms

Interventions

Injections

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Central Study Contacts

Ruzhi Zhang, MD; PhD

CONTACT

05532863172; 18755377606@163.com

Li Lin

CONTACT

008618755377606; 18755377606@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD; PhD

Study Record Dates

• First Submitted: January 22, 2025
• First Posted: February 6, 2025
• Study Start: February 5, 2025
• Primary Completion (Estimated): November 17, 2026
• Study Completion (Estimated): November 17, 2026
• Last Updated: February 6, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)