Clinical trial NCT06731192: Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome
Official title: Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Alport Syndrome
Keywords: Alport syndrome
Human Umbilical Cord Mesenchymal Stem Cells
Randomized Clinical Trial
Lead sponsor: Guangzhou Women and Children's Medical Center
Sponsor class: OTHER
First posted: 2024-12-12
Enrollment: 40
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Urine protein remission rate
timeFrame: 2 weeks to 12 months after treatment
Secondary outcomes:
measure: Hematuria remission rate
timeFrame: 2 weeks to 12 months after treatment
measure: Renal function improvement rate
timeFrame: 2 weeks to 12 months after treatment
Other outcomes:
Locations:
Study officials:
References: