Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: hUC-MSC, Placebo control drug.
- Кому может быть актуально
- Состояния в реестре: Alport Syndrome. Базовые параметры: 3 лет — 12 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Список центров уточняется — проверьте первичный протокол.
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial
Обзор
The goal of this clinical trial is to evaluate the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in the treatment of Alport syndrome (AS) in a randomized, single-blind, placebo-controlled trial, to provide a clinical basis for the development of stem cell products for the treatment of AS, and to further clarify the therapeutic effect of hUC-MSC in the treatment of AS.
Вмешательства
- Биопрепарат hUC-MSC
This group of patients received a total of 2 peripheral intravenous Human umbilical cord mesenchymal stem cells (hUC-MSC) infusions during the treatment period on the basis of conventional symptomatic and supportive treatment. The interval between the two infusions was 14 days, and the single infusion dose was approximately 2×1000,000 cells/Kg. Conventional clinical treatment refers to symptomatic treatment with ACEI and ARB drugs. - Препарат Placebo control drug
This group of patients received a total of 2 intravenous infusions of normal saline (the same volume, specifications and batches used in the experimental group) as a placebo control during the treatment period on the basis of conventional symptomatic and supportive treatment. The interval between the two infusions was 14 days. Conventional clinical treatment refers to symptomatic treatment with ACEI and ARB drugs.
Первичные конечные точки
- Urine protein remission rate [Срок оценки: 2 weeks to 12 months after treatment]
Вторичные конечные точки (2)
- Hematuria remission rate [Срок оценки: 2 weeks to 12 months after treatment]
- Renal function improvement rate [Срок оценки: 2 weeks to 12 months after treatment]
Критерии участия
Критерии включения
- 3 years old ≤Age ≤ 12 years old;
- Meet the diagnostic criteria of Alport syndrome;
- Positive proteinuria or combined hematuria;
- Chronic kidney disease (CKD) stage: I-III stage children, that is, glomerular filtration rate greater than 60 ml/min.1.73m2;
- No history of infectious diseases within 1 week before treatment;
- Negative infectious disease screening;
- No allergic state and related clinical manifestations;
- Signed informed consent (children or their families).
Критерии исключения
- Age <3 years or >12 years old;
- Alport syndrome patients with only microscopic hematuria and normal glomerular filtration rate;
- Patients with significantly reduced renal function, chronic kidney disease stage IV or V, Alport syndrome;
- Patients with other renal diseases;
- Have a history of severe allergic reactions or be allergic to 2 or more foods or drugs;
- Known allergy to stem cells or stem cell-derived products or ingredients in stem cell preparations;
- Have severe heart, liver, lung and other organ dysfunction or have tumors;
- Those with developmental malformations of the urinary system;
- Those with autoimmune diseases and regular use of immunosuppressants;
- Those with serious infectious diseases that are not under control;
- Those with a history of infectious diseases such as HBV, HCV, HIV, syphilis;
- History of surgery or acute trauma or blood loss exceeding 200ml within 3 months;
- Participated in other clinical studies within 3 months;
- Have received any cell product or derivative product treatment within 12 months;
- Other circumstances that the researcher deems inappropriate for inclusion.
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
- Простое слепое
- Основная цель
- Лечение
Центры проведения
Список центров уточняется — проверьте первичный протокол.
Идентификаторы
NCT: NCT06731192 · [2022]00100-3 · MR-44-24-042448