Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: hUC-MSC, Placebo control drug.
- Who it may be relevant to
- Registry conditions: Alport Syndrome. Basic parameters: 3 years — 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial
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.
Interventions
- 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. - 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.
Primary outcome measures
- Urine protein remission rate [Time frame: 2 weeks to 12 months after treatment]
Secondary outcome measures (2)
- Hematuria remission rate [Time frame: 2 weeks to 12 months after treatment]
- Renal function improvement rate [Time frame: 2 weeks to 12 months after treatment]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Single blind
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06731192 · [2022]00100-3 · MR-44-24-042448