Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome (HUCMSC)

Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial

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 Overview

• Status: Not yet recruiting
• Conditions: Alport Syndrome
• Intervention / Treatment: Biological: hUC-MSC; Drug: Placebo control drug

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Xia Gao; Phone Number: 86-020-81330569; Email: gaoxiagz@vip.163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MSC Theatment; Conventional symptomatic supportive treatment and human umbilical cord mesenchymal stem cells treatment	Biological: 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.
Placebo Comparator: Placebo Control; Conventional symptomatic supportive treatment and normal saline as a placebo control	Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	2 weeks to 12 months after treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	2 weeks to 12 months after treatment

Collaborators and Investigators

• Sponsor: Guangzhou Women and Children's Medical Center

Study record dates

Study Major Dates

• Study Start (Estimated): January 1, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: December 9, 2024
• First Submitted That Met QC Criteria: December 9, 2024
• First Posted (Actual): December 12, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 29, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Keywords: Randomized Clinical Trial; Alport syndrome; Human Umbilical Cord Mesenchymal Stem Cells
• Additional Relevant MeSH Terms: Urogenital Diseases; Pathologic Processes; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Connective Tissue Diseases; Disease; Congenital Abnormalities; Urogenital Abnormalities; Nephritis; Collagen Diseases; Syndrome; Nephritis, Hereditary
• Other Study ID Numbers: [2022]00100-3; MR-44-24-042448 (Other Grant/Funding Number: National Health Commission of the People's Republic of China)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No