Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome

NCT06731192 | Not Yet Recruiting Phase 2 DMC

• 2 other identifiers: [2022]00100-3MR-44-24-042448
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Alport Syndrome

Keywords

Alport syndrome; Human Umbilical Cord Mesenchymal Stem Cells; Randomized Clinical Trial

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Hematuria remission rate

  2 weeks to 12 months after treatment

• Renal function improvement rate

  2 weeks to 12 months after treatment

Study Arms (2)

MSC Theatment

EXPERIMENTAL

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

Biological: hUC-MSC

Placebo Control

PLACEBO COMPARATOR

Conventional symptomatic supportive treatment and normal saline as a placebo control

Drug: Placebo control drug

Interventions

hUC-MSC BIOLOGICAL

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.

MSC Theatment

Placebo control drug 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.

Placebo Control

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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;

Sponsors & Collaborators

• Guangzhou Women and Children's Medical Center: lead

MeSH Terms

Conditions

Nephritis, Hereditary

Condition Hierarchy (Ancestors)

Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Nephritis; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Central Study Contacts

Xia Gao

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 9, 2024
• First Posted: December 12, 2024
• Study Start: January 1, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2028
• Last Updated: December 31, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share