Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome

NCT ID: NCT06731192

Last Updated: 2024-12-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-01-01

Study Completion Date

2028-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Alport Syndrome

Keywords

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Alport syndrome Human Umbilical Cord Mesenchymal Stem Cells Randomized Clinical Trial

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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MSC Theatment

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

Group Type EXPERIMENTAL

hUC-MSC

Intervention Type 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.

Placebo Control

Conventional symptomatic supportive treatment and normal saline as a placebo control

Group Type PLACEBO_COMPARATOR

Placebo control drug

Intervention Type 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.

Interventions

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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.

Intervention Type BIOLOGICAL

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.

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

3 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Guangzhou Women and Children's Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Central Contacts

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Xia Gao

Role: CONTACT

Phone: 86-020-81330569

Email: [email protected]

Other Identifiers

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MR-44-24-042448

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

[2022]00100-3

Identifier Type: -

Identifier Source: org_study_id