Nephrotic SyndromeNCT02602873
Study of Tacrolimus Used for Pediatric Patients With Nephrotic Syndrome Based on Pharmacogenomics and Metabonomics
An observational study of Nephrotic Syndrome, run by Guangzhou Women and Children's Medical Center at 1 site in China.
Trial summary
Tacrolimus is recommended to be the first line therapeutic medication within the several immunosuppressive agents when treating refractory pediatric nephrotic syndrome, because of its definite efficacy and low toxicity. Read more about this study
- Who is it for?
- Ages 1 month to 14 years
- Conditions the study is for
- Nephrotic Syndrome
- What is being tested?
- therapeutic drug monitoring
- How many people
- 150 participants
- Where
- 1 siteChina
- Study dates
- Aug 2015to Aug 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 month to 14 years
- All sexes
- No healthy volunteers
- Child
Study population: pediatric patients with nephrotic syndrome
Who may be able to join
- patients with refractory nephrotic syndrome;
- patients age ≤14y.
Who cannot join
- patients are sensitive to steroid;
- combined therapy with other immunosuppressive agent;
- combined using drugs which maybe interact the concentration of tacrolimus;
- with other malignant disease, such as tumor.
Trial locations and contacts
1 site in 1 country.
China1 site
Guangzhou women and children's medical center
Guangzhou, Guangdong, 510623
About this study
Official title: Guangzhou Women and Children's Medical Center
Tacrolimus is recommended to be the first line therapeutic medication within the several immunosuppressive agents when treating refractory pediatric nephrotic syndrome, because of its definite efficacy and low toxicity. But there are still some key problems which hinder the using of tacrolimus in clinic, such as its narrow therapeutic widow, great individual difference of pharmacokinetics. Routine therapeutic drug monitoring(TDM) is needed in practice. But the disadvantage of TDM is hysteresis, which could lead to treatment failure or toxicity. To find out the reasons of great pharmacokinetic difference between patients and find out the individual proper dosage before administration are important for the clinical using of tacrolimus.
It is hot in research of tacrolimus in organ transplant field, such as the association between gene polymorphisms of cytochrome P-450 3A4, 3A5 and multiple drug resistant gene(MDR1) and concentration of tacrolimus. However, there is few study about pharmacogenomics and metabonomics of tacrolimus in patients of nephrotic syndrome.
The aim is to study the relationships between pharmacogenomics, metabonomics of tacrolimus and its efficacy, toxicity and blood concentration in patients of nephrotic syndrome, to find out the exact dosage before administration, to provide reference to individual drug administration.
Read the detailed description
Investigators will collect data about efficacy and adverse drug reaction(ADR) such as time of efficacy when evaluated, how to evaluate ADR, data of demography, etc.
- Conditions
- Nephrotic Syndrome
- Being tested
- therapeutic drug monitoring
- Medical subject headings
- Nephrotic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
therapeutic drug monitoringOther
with therapeutic drug monitoring , dose of tacrolimus can be adjusted by therapeutic drug monitoring.
| Study group | Role | What they receive |
|---|---|---|
| good efficacy | using therapeutic drug monitoring to adjust the dose of tacrolimus. patients can reach effective outcome. Receives: Other: therapeutic drug monitoring | |
| poor efficacy | using therapeutic drug monitoring to adjust the dose of tacrolimus. patients can not reach effective outcome. Receives: Other: therapeutic drug monitoring |
What is being measured
The primary outcome is the main question the study is designed to answer.
"Genotypes as measured by polymerase chain reaction-restriction fragment length polymorphism"
genotype are collected from hospital system.
Measured: 1 week
"Concentration as measured by liquid chromatography mass spectrometry"
concentration of tacrolimus are collected from hospital system.
Measured: 1 week
"Relationship between genotypes and concentration as analyzed at 1 week"
using Statistic Package for Social Science 21.0 software to analyze the relationship between gene polymorphism and concentration.
Measured: 1 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2015 (estimated)
- Study startAug 2015
- Primary completionJul 2017 (estimated)
- Study completionAug 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Nephrotic 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
- Observational
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangzhou Women and Children's Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Sun Yat-sen University
- Responsible party
- Principal investigator, Mo Xiaolan, Guangzhou Women and Children's Medical Center
- Investigators
- Min Huang, Doctor, Study director, Sun Yat-sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02602873
- Sponsor's protocol ID
- 683292136
- Status last verified
- Aug 2016
- First submitted
- 9 Sep 2015
- Last updated
- 12 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No: we are carrying out experiment now. Data is not ready to publish for not completing the study. We will share data when we complete our study.
Publications
Mo X, Li J, Liu Y, Liao X, Tan M, Chen Y, He F, He Y, Li Y, Huang M. Kidney podocyte-associated gene polymorphisms affect tacrolimus concentration in pediatric patients with refractory nephrotic syndrome. Pharmacogenomics J. 2020 Aug;20(4):543-552. doi: 10.1038/s41397-019-0141-x. Epub 2020 Jan 6.
PubMed 31902946
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Guangzhou Women and Children's Medical Center
- ClinicalTrials.gov ID
- NCT02602873
- Acronym / sponsor ID
- 683292136
- Conditions
- Nephrotic Syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Guangzhou Women and Children's Medical CenterUniversity, hospital or other
- Collaborators
- Sun Yat-sen University
- Study start
- Aug 2015
- Primary completion
- Jul 2017 (estimated)
- Study completion
- Aug 2020 (estimated)
- First posted
- 11 Nov 2015 (estimated)
- 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.
Tacrolimus is recommended to be the first line therapeutic medication within the several immunosuppressive agents when treating refractory pediatric nephrotic syndrome, because of its definite efficacy and low toxicity. But there are still some key problems which hinder the using of tacrolimus in clinic, such as its narrow therapeutic widow, great individual difference of pharmacokinetics. Routine therapeutic drug monitoring(TDM) is needed in practice. But the disadvantage of TDM is hysteresis, which could lead to treatment failure or toxicity. To find out the reasons of great pharmacokinetic difference between patients and find out the individual proper dosage before administration are important for the clinical using of tacrolimus.
It is hot in research of tacrolimus in organ transplant field, such as the association between gene polymorphisms of cytochrome P-450 3A4, 3A5 and multiple drug resistant gene(MDR1) and concentration of tacrolimus. However, there is few study about pharmacogenomics and metabonomics of tacrolimus in patients of nephrotic syndrome.
The aim is to study the relationships between pharmacogenomics, metabonomics of tacrolimus and its efficacy, toxicity and blood concentration in patients of nephrotic syndrome, to find out the exact dosage before administration, to provide reference to individual drug administration.
Detailed description
Investigators will collect data about efficacy and adverse drug reaction(ADR) such as time of efficacy when evaluated, how to evaluate ADR, data of demography, etc.
Study design
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| good efficacy | – | Other: therapeutic drug monitoringusing therapeutic drug monitoring to adjust the dose of tacrolimus. patients can reach effective outcome. |
| poor efficacy | – | Other: therapeutic drug monitoringusing therapeutic drug monitoring to adjust the dose of tacrolimus. patients can not reach effective outcome. |
Who may qualify
- Ages
- 1 month to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- pediatric patients with nephrotic syndrome
Key inclusion criteria (2 listed)
- patients with refractory nephrotic syndrome;
- patients age ≤14y.
Key exclusion criteria (4 listed)
- patients are sensitive to steroid;
- combined therapy with other immunosuppressive agent;
- combined using drugs which maybe interact the concentration of tacrolimus;
- with other malignant disease, such as tumor.
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 |
|---|---|---|
| therapeutic drug monitoringwith therapeutic drug monitoring , dose of tacrolimus can be adjusted by therapeutic drug monitoring. | Other | good efficacy, poor efficacy |
Primary outcome
"Genotypes as measured by polymerase chain reaction-restriction fragment length polymorphism"
Time frame: 1 week
genotype are collected from hospital system.
"Concentration as measured by liquid chromatography mass spectrometry"
Time frame: 1 week
concentration of tacrolimus are collected from hospital system.
"Relationship between genotypes and concentration as analyzed at 1 week"
Time frame: 1 week
using Statistic Package for Social Science 21.0 software to analyze the relationship between gene polymorphism and concentration.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2016.
- Derived from this study: PubMed 31902946Mo X, Li J, Liu Y, Liao X, Tan M, Chen Y, He F, He Y, Li Y, Huang M. Kidney podocyte-associated gene polymorphisms affect tacrolimus concentration in pediatric patients with refractory nephrotic syndrome. Pharmacogenomics J. 2020 Aug;20(4):543-552. doi: 10.1038/s41397-019-0141-x. Epub 2020 Jan 6.
- Individual participant dataSharing planned: No. we are carrying out experiment now. Data is not ready to publish for not completing the study. We will share data when we complete our study.
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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. This study is not recruiting at the moment.
Min Huang, Doctor study director
Sun Yat-sen University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Nephrotic Syndrome, run by Guangzhou Women and Children's Medical Center at 1 site in China.
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 2 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 12 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
"Genotypes as measured by polymerase chain reaction-restriction fragment length polymorphism" (time frame: 1 week). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02602873, which also lists 1 linked publication.
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 12 Aug 2016.