Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome

Phase 2

Not yet recruiting

• Conditions: Alport Syndrome
• Interventions: Biological: hUC-MSC; Drug: Placebo control drug

• Registration Number: NCT06731192
• Lead Sponsor: Guangzhou Women and Children's Medical Center

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-09
• Study First Submitted QC: 2024-12-09
• Study First Posted: 2024-12-12
• Last Update Submitted: 2024-12-29
• Last Update Posted: 2024-12-31
• Study Start: 2025-01-01
• Primary Completion: 2026-06-30
• Study Completion: 2028-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MSC Theatment	hUC-MSC	Conventional symptomatic supportive treatment and human umbilical cord mesenchymal stem cells treatment
Placebo Control	Placebo control drug	Conventional symptomatic supportive treatment and normal saline as a placebo control

Primary Outcome Measures

Name	Time	Method
Urine protein remission rate	2 weeks to 12 months after treatment	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.

Secondary Outcome Measures

Name	Time	Method
Hematuria remission rate	2 weeks to 12 months after treatment	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.
Renal function improvement rate	2 weeks to 12 months after treatment	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.