Evaluate the efficacy of induced pluripotent stem cell-derived exosomes (iPSC-Exos) for the treatment of stable vitiligo
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
29
human induced pluripotent stem cell derived exosomes (GD-iExo-003)
a placebo of exosomes derived from human induced pluripotent stem cell for injection
The Second Affiliated Hospital of Wannan Medical College
Wuhu, Anhui, China
adverse events as assessed by CTCAE
all potentially treated subjects to assess the safety
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
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
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
blood tests to check liver function indicators-Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)-and kidney function indicators-Creatinine (Cr), Blood Urea Nitrogen (BUN)
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
Using the grid counting method to calculate 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
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.
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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
This questionnaire includes assessments of appearance noticeability and psychosocial impact, with higher scores indicating a greater level of impact.
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
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.
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