Alport SyndromePhase 2/3HUCMSCNCT06731192
Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome
A Phase 2/3 study testing hUC-MSC in people with Alport Syndrome, run by Guangzhou Women and Children's Medical Center.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Alport Syndrome
- What is being tested?
- hUC-MSC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2025to 31 Dec 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- 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;
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- 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 locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial
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
- Being tested
- hUC-MSC, Placebo control drug
- Keywords
- Alport syndrome, Human Umbilical Cord Mesenchymal Stem Cells, Randomized Clinical Trial
- Medical subject headings
- Nephritis, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hUC-MSCBiological
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 drugDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| MSC Theatment | Experimental | Conventional symptomatic supportive treatment and human umbilical cord mesenchymal stem cells treatment Receives: Biological: hUC-MSC |
| Placebo Control | Placebo comparator | Conventional symptomatic supportive treatment and normal saline as a placebo control Receives: Drug: Placebo control drug |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 weeks to 12 months after treatment
Secondary outcomes (2)
Hematuria 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. The urine abnormal red blood cell count of the children was tested to evaluate the remission of hematuria after treatment.
Measured: 2 weeks to 12 months after treatment
Renal function improvement 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 hUCMSC infusion to measure the serum creatinine, blood urea nitrogen, serum cystatin C, glomerular filtration rate and other indicators of the children to evaluate the improvement of renal function after treatment.
Measured: 2 weeks to 12 months after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2024
- Study start1 Jan 2025 (estimated)
- Primary completion30 Jun 2026 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alport SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangzhou Women and Children's Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06731192
- Sponsor's protocol ID
- [2022]00100-3
- Other IDs
- MR-44-24-042448
- Acronym
- HUCMSC
- Status last verified
- Dec 2024
- First submitted
- 9 Dec 2024
- Last updated
- 31 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial
- ClinicalTrials.gov ID
- NCT06731192
- Acronym / sponsor ID
- HUCMSC / [2022]00100-3
- Conditions
- Alport Syndrome
- Keywords
- Alport syndrome, Human Umbilical Cord Mesenchymal Stem Cells, Randomized Clinical Trial
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Guangzhou Women and Children's Medical CenterUniversity, hospital or other
- Study start
- 1 Jan 2025 (estimated)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 12 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MSC Theatment | Experimental | Biological: hUC-MSCConventional symptomatic supportive treatment and human umbilical cord mesenchymal stem cells treatment |
| Placebo Control | Placebo comparator | Drug: Placebo control drugConventional symptomatic supportive treatment and normal saline as a placebo control |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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;
Key exclusion criteria (15 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hUC-MSCThis 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. | Biological | MSC Theatment |
| Placebo control drugThis 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. | Drug | Placebo Control |
Primary outcome
Urine protein remission rate
Time frame: 2 weeks to 12 months after treatment
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 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing hUC-MSC in people with Alport Syndrome, run by Guangzhou Women and Children's Medical Center.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 31 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xia Gao (86-020-81330569, gaoxiagz@vip.163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Urine protein remission rate (time frame: 2 weeks to 12 months after treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Dec 2024.