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

Guangzhou Women and Children's Medical Center

Status and phase: Not yet enrolling; Phase 3; Phase 2

Conditions: Alport Syndrome

Treatments: Biological: hUC-MSC; Drug: Placebo control drug

Study type: Interventional

Funder types: Other

Identifiers

NCT06731192

MR-44-24-042448 (Other Grant/Funding Number)

[2022]00100-3

Details and patient eligibility

About

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.

Enrollment: 40 estimated patients

Sex: All

Ages: 3 to 12 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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40 participants in 2 patient groups, including a placebo group

MSC Theatment

Experimental group

Description:

Conventional symptomatic supportive treatment and human umbilical cord mesenchymal stem cells treatment

Treatment:

Biological: hUC-MSC

Placebo Control

Placebo Comparator group

Description:

Conventional symptomatic supportive treatment and normal saline as a placebo control

Treatment:

Drug: Placebo control drug