hUC-MSC-Exosomes for T2DM - Safety & Efficacy

Официальное название

An Exploratory Clinical Study Assessing the Safety and Preliminary Efficacy of Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes in the Treatment of Adult Type 2 Diabetes Mellitus

Обзор

The goal of this clinical trial is to Evaluate the Safety and Preliminary Efficacy of Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes in the Treatment of Adult Type 2 Diabetes Mellitus.

Подробное описание

A two-stage, progressive mixed design will be adopted to balance ethical considerations and scientific rigor.

Stage 1 (Open-Label, Before-and-After Study) will employ a sequential, open-label dose-escalation design:

A total of 18 patients will be enrolled, all receiving a 2-month course (once weekly, 8 doses total) of intravenous exosome infusion therapy. This study uses a sequential dose-escalation design with three dose cohorts: low-dose (1×10¹¹ particles), medium-dose (3×10¹¹ particles), and high-dose (9×10¹¹ particles). Dose escalation will strictly adhere to the following procedure:

Sequential Progression: The study for the next dose cohort can only commence after all subjects in the previous dose cohort have completed the required safety observation period (i.e., the 5-week follow-up post-last dose) and the safety data has been reviewed.

Stage 2 (Randomized, Double-Blind, Active-Comparator Parallel Study):

Provided no major safety issues are identified in the first stage, the second stage will commence. This stage plans to enroll 48 patients, randomized 2:1 to either the exosome treatment group (n=32) or the active-comparator control group (n=16).

Follow-up Plan: Multiple visits are scheduled, including a baseline period, treatment period (after each dose), end of treatment (Week 8/Month 2), and post-treatment follow-up periods (Week 12, Month 3, Month 6, Month 12), to systematically evaluate efficacy and safety.

Вмешательства

• Биопрепарат exosomes treatment group
  GoldenExo（NATX） treatment group (n=32) The specific therapeutic dose for the second stage will be evaluated and determined jointly by the investigators and the Safety Monitoring Committee (SMC) after all three dose escalation trials (1×10¹¹, 3×10¹¹, 9×10¹¹ particles) in the first stage.Subjects in both groups will receive intravenous infusions once weekly for 8 weeks (a total of 8 doses).
• Другое control group
  active-comparator control：（n=16）The control group will receive an equal volume of the same buffer solution without exosomes (.Subjects in this groups will receive intravenous infusions once weekly for 8 weeks (a total of 8 doses)

Первичные конечные точки

• Change in Glycated Hemoglobin (HbA1c) [Срок оценки: Baseline, Week 8]

Вторичные конечные точки (12)

• Glycated Hemoglobin (HbA1c) Response Rate [Срок оценки: Week 24, Week 52]
• Pancreatic Islet Function: Insulin (INS) [Срок оценки: Baseline, Week 4, Week 8，Week 12, Week 24, Week 52]
• Pancreatic Islet Function: C-peptide [Срок оценки: Baseline, Week 4, Week 8, Week 12, Week 24, Week 52]
• Changes in Blood Glucose during MMTT [Срок оценки: Baseline, Week 8, Week 52]
• Anthropometric and Metabolic Parameters [Срок оценки: Baseline, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 52]
• Soluble Adhesion Molecule (sICAM-1) [Срок оценки: Baseline, Week 4, Week 8, Week 12, Week 24, Week 52]
• Ophthalmologic Assessment [Срок оценки: Baseline, Week 24]
• Change in Central Obesity: Waist-to-Hip Ratio (WHR) [Срок оценки: Baseline, Week 4, Week 8, Week 12, Week 24, Week 52]
• Body fat percentage (%) [Срок оценки: Baseline, Week 8, Week 24, Week 52]
• C-Reactive Protein (CRP) [Срок оценки: Baseline, Week 4, Week 8, Week 12, Week 24, Week 52]
• Time in Range (TIR, 3.9-10.0 mmol/L) [Срок оценки: baseline to week 8]
• Mean glucose level [Срок оценки: baseline to week 8]

Критерии участия

Критерии включения

• Age 18-65 years, diagnosed with T2DM for ≥5 years (<20 years).
• Regular diet and exercise control combined with the following intensive treatment regimens, with screening HbA1c remaining between 7.5% and 9.0% (FBG <13.9mmol/L):Stable dose of insulin (basal or premixed) (<1.5u/kg·d) combined with ≥1 oral antidiabetic drug (OAD) (e.g., Metformin, SGLT2i) for ≥3 months; or Insulin combined with a GLP-1RA (maximum tolerated dose, ≤1mg Qw); or GLP-1RA (maximum tolerated dose, ≤1mg Qw) combined with ≥1 OAD for ≥3 months; or Stable dose of ≥3 OADs (must include Metformin) for ≥3 months.
• Fasting C-peptide ≥1.0 ng/mL.
• Glutamic acid decarboxylase (GAD) antibody and Islet cell antibody (IAA) negative.
• Body Mass Index (BMI) 18.5-35 kg/m ².
• Subjects voluntarily participate in the study and sign the informed consent form.
• Female subjects of childbearing potential and non-sterilized male subjects must agree to use highly effective contraceptive methods during the study period and for 1 year following the final infusion.

Критерии исключения

• Type 1 diabetes or other specific types of diabetes.
• Occurrence of severe hypoglycemia or diabetic ketoacidosis within the past 6 months.
• Severe cardiovascular/cerebrovascular diseases, hepatic or renal insufficiency (e.g., eGFR <30 mL/min·1.73m²).
• Active malignancy, coagulation disorders, immune system diseases.
• Pregnant or lactating women.
• Individuals with allergic constitution.
• Exclusion of subjects currently participating in other clinical trials.
• Subjects deemed unsuitable for participation by the investigator.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Peking University first Hospital — Пекин

Идентификаторы

NCT: NCT07693036 · 2026R0184-0002

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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