Stem cell therapy for renal dysfunction
| ISRCTN | ISRCTN62094626 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN62094626 |
- Submission date
- 29/12/2022
- Registration date
- 07/01/2023
- Last edited
- 18/03/2024
- Recruitment status
- Recruiting
- Overall study status
- Ongoing
- Condition category
- Urological and Genital Diseases
Plain English summary of protocol
Background and study aims
Alport syndrome (AS) is one of the most common human fatal hereditary renal (kidney) diseases and is characterized by hematuria (blood in the urine), albuminuria (too much protein in the urine), and a progressive decline of kidney function. The risk is high for male patients with COL4A5 deficiency to develop into an end-stage renal disease without effective treatments. The main aim of this study is to find out whether intravenous infusion (into a vein) of human umbilical cord mesenchymal stem cells (hUC-MSCs) is safe and feasible with good tolerance for patients with AS.
Who can participate?
Patients aged 3-18 years with AS
What does the study involve?
Participants will receive four doses of hUC-MSC as scheduled. Safety and effectiveness evaluations are performed at baseline (Day 0), weekly in the treatment phase (Day 7, Day 14, Day 21), and monthly in a follow-up phase (Months 1-12 after treatment). The study lasts for 1 year in total.
What are the possible benefits and risks of participating?
The treatment could reverse albuminuria based on pre-clinical studies in mice. No serious adverse events were reported in previous studies in children with cerebral palsy. The main risk is that patients might experience a transient fever of no more than 38.5°C based on other studies of stem cell transfusion.
Where is the study run from?
Affiliated Taihe Hospital of Hubei University of Medicine (China)
When is the study starting and how long is it expected to run for?
January 2015 to December 2026
Who is funding the study?
1. Shenzhen Key Medical Discipline Construction Fund (China)
2. Science and Technology Research Project of Hubei Province (China)
Who is the main contact?
Prof. Che Zhang, prof.zh@163.com
Contact information
Principal Investigator
No. 32 Southen Renmin Road
Shiyan
422000
China
 ORCID ID |
0000-0001-6757-4306 |
|---|---|
| Phone | +86 (0)719 8801691 |
| prof.zh@163.com |
Study information
| Study design | Multicenter interventional single-arm trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised study |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Scientific title | The safety and efficacy of human umbilical cord mesenchymal stem cell transfusion in patients with Alport syndrome |
| Study objectives | The intravenous infusion of human umbilical cord mesenchymal stem cell (hUC-MSC) is safe and feasible with good tolerance for patients with Alport syndrome |
| Ethics approval(s) | Approved 08/03/2015, Institutional Review Board of the Affiliated Taihe Hospital of Hubei University of Medicine (No. 32 Southen Renmin Road, Shiyan, Hubei 422000, China; +86 (0)719 8801691; thyyllwyh@126.com), ref: 20150303 |
| Health condition(s) or problem(s) studied | Albuminuria in the patients with Alport syndrome |
| Intervention | hUC-MSCs will be injected intravenously at a dose of 20 × 10e6 cells. The duration of infusion will last for 20 - 30 min. Patients will receive four doses of hUC-MSC at an interval of 7 days. |
| Intervention type | Biological/Vaccine |
| Pharmaceutical study type(s) | |
| Phase | Phase I |
| Drug / device / biological / vaccine name(s) | hUC-MSCs |
| Primary outcome measure | 1. Adverse event incidence is calculated at baseline (Day 0), weekly in the treatment phase (Day 7, Day 14, Day 21), and monthly in a follow-up phase (Month 1-12 post-treatment) 2. Urine protein is tested in the laboratory at baseline (Day 0), weekly in the treatment phase (Day 7, Day 14, Day 21), and monthly in a follow-up phase (Month 1-12 post-treatment) |
| Secondary outcome measures | 1. Hematuria is tested in the laboratory at baseline (Day 0), weekly in the treatment phase (Day 7, Day 14, Day 21), and monthly in a follow-up phase (Month 1-12 post-treatment) 2. Creatinine clearance rate is tested in the laboratory at baseline (Day 0), and monthly in a follow-up phase (Month 1-12 post-treatment) |
| Overall study start date | 08/01/2015 |
| Completion date | 31/12/2026 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 3 Years |
| Upper age limit | 18 Years |
| Sex | Both |
| Target number of participants | 12 |
| Key inclusion criteria | 1. Diagnosed with Alport syndrome (AS) according to the diagnosis criteria 2. Aged 3-18 years 3. Hematuria or combination with albuminuria persisted without remission after routine treatments for 1 year 4. Chronic kidney disease stage I-III, glomerular filtration rate >60 ml/min.1.73 m² 5. Written informed consent is obtained before study specific procedure |
| Key exclusion criteria | 1. Aged >18 years 2. Creatinine clearance rate <30 ml/min, or chronic kidney disease stage IV-V 3. With one of the disease histories: immunological disease or autoimmune diseases; serious hematologic or coagulation disorder; urogenital abnormalities; malignancy history; congenital heart disease or serious cardiovascular, liver, or pulmonary dysfunction 4. Uncontrolled endocrine diseases (e.g. diabetes, hyperthyroidism) 5. Serious allergy history or known allergy to more than two kinds of foods or medications 6. Active systemic infection 7. Any other concerns that hampered compliance or safety as judged by the investigator |
| Date of first enrolment | 15/01/2023 |
| Date of final enrolment | 31/12/2025 |
Locations
Countries of recruitment
- China
Study participating centres
Shiyan
422000
China
Shenzhen
518133
China
Haikou
571199
China
Sponsor information
Government
No. 52 Nanyuan Road
Wuhan
430064
China
| Phone | +86 (0)27 87135893 |
|---|---|
| 305265139@qq.com | |
| Website | http://www.hbstd.gov.cn/ |
"ROR" |
https://ror.org/00tbh0t11 |
Funders
Funder type
Government
No information available
No information available
Results and Publications
| Intention to publish date | 31/12/2027 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal. |
| IPD sharing plan | The datasets generated during the current study will be available upon request from the principal investigator Prof. Che Zhang (prof.zh@163.com). The type of data that will be shared: de-identified demographic data, clinical characteristics, and outcomes of participants. Dates of availability: currently unknown. Whether consent from participants was required and obtained: written consent forms will be obtained before any study-specific procedure, and will be kept as the source documents at the investigational sites. Comments on data anonymization: clinical data are de-identified before being collected for scientific research purposes to protect the privacy of patients. Any ethical or legal restrictions: the current study has been approved by the ethical committee and follows the guidelines for Good Clinical Practice, as well as the requirements of the local Institution for New Drug Clinical Trials. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Protocol article | 15/03/2024 | 18/03/2024 | Yes | No |
Editorial Notes
18/03/2024: Publication reference added.
06/01/2023: Trial's existence confirmed by the Institutional Review Board of the Affiliated Taihe Hospital of Hubei University of Medicine.
