Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome

Phase 3Not Yet RecruitingNCT06731192

Guangzhou Women and Children's Medical Center40 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Alport Syndrome

Interventions

hUC-MSCBIOLOGICAL

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.

Placebo control drugDRUG

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.

Eligibility

Sex: ALLMin age: 3 YearsMax age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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). Exclusion Criteria: * 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.

Outcomes

Primary Outcomes

Urine protein remission rate

Follow-up was performed at 2 weeks, 4 weeks, 6 weeks, 8 weeks, 3 months, 4 months, 5 months, 6 months, 8 months, 10 months, and 12 months after the last hUC-MSC infusion. Urinalysis, 24-hour urine protein quantification, and urine protein/creatinine ratio were tested to evaluate the remission of urine protein after treatment.

Time frame: 2 weeks to 12 months after treatment

Secondary Outcomes

Hematuria remission rate

Follow-up was performed at 2 weeks, 4 weeks, 6 weeks, 8 weeks, 3 months, 4 months, 5 months, 6 months, 8 months, 10 months, and 12 months after the last hUC-MSC infusion. The urine abnormal red blood cell count of the children was tested to evaluate the remission of hematuria after treatment.

Time frame: 2 weeks to 12 months after treatment

Renal function improvement rate

Follow-up was performed at 2 weeks, 4 weeks, 6 weeks, 8 weeks, 3 months, 4 months, 5 months, 6 months, 8 months, 10 months, and 12 months after the last hUCMSC infusion to measure the serum creatinine, blood urea nitrogen, serum cystatin C, glomerular filtration rate and other indicators of the children to evaluate the improvement of renal function after treatment.

Time frame: 2 weeks to 12 months after treatment

Central Contacts

Xia Gao

CONTACT

86-020-81330569 gaoxiagz@vip.163.com

Data from ClinicalTrials.gov

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