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

Официальное название

Exploratory Clinical Study on Induced Pluripotent Stem Cell Derived Exosomes (iPSC-Exos) for the Treatment of Stable Vitiligo

Обзор

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

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• adverse events as assessed by CTCAE [Срок оценки: 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]

Вторичные конечные точки (6)

• Local Adverse Reactions [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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]

Критерии участия

Критерии включения

• 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).

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• The Second Affiliated Hospital of Wannan Medical College — Wuhu

Идентификаторы

NCT: NCT06810869 · WYEFYLS2024121

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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