Steroid Dependent and Steroid Resistent Nephrotic SyndromeNCT03219684
Cyclosporin A Therapy in Childhood Nephrotic Syndrome
An observational study of Steroid Dependent and Steroid Resistent Nephrotic Syndrome, run by Assiut University.
Trial summary
Nephrotic syndrome (NS) is among the most common pediatric kidney diseases and is defined as massive proteinuria (>40 mg/m2/h or urine protein to creatinine ratio >2 g/g) leading to hypoalbuminemia (<2.5 g/dL), edema, and hyperlipidemia. 60-70 % of patients present prior to age of 6 years Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Steroid Dependent and Steroid Resistent Nephrotic Syndrome
- What is being tested?
- serum protein ,kidney function ,liver function.,serum cholesterol
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2018to 1 Aug 2019
- 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 year to 18 years
- All sexes
- Child, Adult
Study population: children from the age of one year to the age of 18 year who are diagnosed as steroid resistent and steroid dependent nephrotic syndrome and on cyclosporin therapy
Who may be able to join
- The study will include all children (on cyclosporine therapy for treatment of Steroid dependent or Steroid resistant nephrotic syndrome ) who present to Nephrology Unit , and clinic at Assiut University Children Hospital during one year duration.
Who cannot join
- children with adequate response to steroid therapy and without relapses or resistance to steroid therapy
Trial locations and contacts
No sites are listed yet.
About this study
Official title: A Descriptive Study on the Effectiveness and Safety of Cyclosporin A Therapy in Steroid Dependent and Steroid Resistant Childhood Nephrotic Syndrome
Nephrotic syndrome (NS) is among the most common pediatric kidney diseases and is defined as massive proteinuria (>40 mg/m2/h or urine protein to creatinine ratio >2 g/g) leading to hypoalbuminemia (<2.5 g/dL), edema, and hyperlipidemia. 60-70 % of patients present prior to age of 6 years
Read the detailed description
. Most children with NS are treated initially with oral corticosteroids, and they can be clinically classified based on their ability to achieve remission (i.e., complete normalization of proteinuria). Approximately 85 % of children under the age of 6 years are steroid-sensitive, whereas the remainder have steroid-resistant disease. Older children are more likely to have steroid-resistant NS. Children with steroid-resistant disease may have an underlying genetic cause for NS, and providers should consider genetic testing in this population, depending on the age of the child . While inherited causes of NS are often resistant to all therapies, there are reports of complete or partial remission in some children .
For those children who respond to steroids, the majority will have one or more relapses and half will have frequently relapsing (≥4 relapses/year) or steroid-dependent (two consecutive relapses during steroid therapy or within 14 days of stopping steroids). NS Children with frequently relapsing NS and steroid-dependent NS may have significant side effects from cumulative corticosteroid therapy so treatment with other agents is often required .
Cyclosporine and tacrolimus are calcineurin inhibitors that are commonly used as immunosuppressive agents in solid organ transplantation. CNIs are recommended as first-line therapy for children with steroid-resistant NS and as steroid-sparing agents for children with frequently relapsing or steroid-dependent NS .Calcineurin inhibitors (CNIs) inhibit T-cell activation and may be exerting their effect in nephrotic syndrome through this mechanism.
Alternately, cyclosporine has been shown to directly target the podocyte and stabilize the actin cytoskeleton responsible for maintaining cell shape(5) .. Although the majority of studies in nephrotic syndrome have been performed with cyclosporine, tacrolimus appears to be equally efficacious.
Cyclosporin A therapy is well recognised regarding its steroid sparing effect in steroid dependant patients and is responsible for maintaining remission in more than 75% of patients with Steroid dependent nephrotic syndrome even after discontinuation of steroids Furthermore, it has been shown to be effective in inducing remission in steroid resistant nephrotic syndrome. However ,Cyclosporin A is associated with a plethora of side effects such as hypertension, nephrotoxicity hypertrichosis, gum hyperplasia, gastrointestinal disturbances and tremor.
- Being tested
- serum protein ,kidney function ,liver function.,serum cholesterol
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
serum protein ,kidney function ,liver function.,serum cholesterolDiagnostic test
monthly review of serum protein kidney function ,liver function and serum cholesterol in all patients
What is being measured
The primary outcome is the main question the study is designed to answer.
evaluation of nephrotic state of each patient for evaluation of efficacy of cyclosporin A therapy
measurment of serum albumin ,serum cholesterol and urinary protien excretion
Measured: one year
evaluation of side effects of cyclosporin A
serial observation of common side effects of cyclosporin A as hypertension, nephrotoxicity hypertrichosis, gum hyperplasia, gastrointestinal disturbances and tremor.
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2017
- Study start1 Apr 2018 (estimated)
- Primary completion1 Apr 2019 (estimated)
- Study completion1 Aug 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Steroid Dependent and Steroid Resistent Nephrotic SyndromeStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- 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
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Abdullah Ahmed Abdel_Ghany, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03219684
- Sponsor's protocol ID
- DSESCSDSRNS
- Status last verified
- Jul 2017
- First submitted
- 11 Jul 2017
- Last updated
- 17 Jul 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Descriptive Study on the Effectiveness and Safety of Cyclosporin A Therapy in Steroid Dependent and Steroid Resistant Childhood Nephrotic Syndrome
- ClinicalTrials.gov ID
- NCT03219684
- Acronym / sponsor ID
- DSESCSDSRNS
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Apr 2018 (estimated)
- Primary completion
- 1 Apr 2019 (estimated)
- Study completion
- 1 Aug 2019 (estimated)
- First posted
- 17 Jul 2017 (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.
Nephrotic syndrome (NS) is among the most common pediatric kidney diseases and is defined as massive proteinuria (>40 mg/m2/h or urine protein to creatinine ratio >2 g/g) leading to hypoalbuminemia (<2.5 g/dL), edema, and hyperlipidemia. 60-70 % of patients present prior to age of 6 years
Detailed description
. Most children with NS are treated initially with oral corticosteroids, and they can be clinically classified based on their ability to achieve remission (i.e., complete normalization of proteinuria). Approximately 85 % of children under the age of 6 years are steroid-sensitive, whereas the remainder have steroid-resistant disease. Older children are more likely to have steroid-resistant NS. Children with steroid-resistant disease may have an underlying genetic cause for NS, and providers should consider genetic testing in this population, depending on the age of the child . While inherited causes of NS are often resistant to all therapies, there are reports of complete or partial remission in some children .
For those children who respond to steroids, the majority will have one or more relapses and half will have frequently relapsing (≥4 relapses/year) or steroid-dependent (two consecutive relapses during steroid therapy or within 14 days of stopping steroids). NS Children with frequently relapsing NS and steroid-dependent NS may have significant side effects from cumulative corticosteroid therapy so treatment with other agents is often required .
Cyclosporine and tacrolimus are calcineurin inhibitors that are commonly used as immunosuppressive agents in solid organ transplantation. CNIs are recommended as first-line therapy for children with steroid-resistant NS and as steroid-sparing agents for children with frequently relapsing or steroid-dependent NS .Calcineurin inhibitors (CNIs) inhibit T-cell activation and may be exerting their effect in nephrotic syndrome through this mechanism.
Alternately, cyclosporine has been shown to directly target the podocyte and stabilize the actin cytoskeleton responsible for maintaining cell shape(5) .. Although the majority of studies in nephrotic syndrome have been performed with cyclosporine, tacrolimus appears to be equally efficacious.
Cyclosporin A therapy is well recognised regarding its steroid sparing effect in steroid dependant patients and is responsible for maintaining remission in more than 75% of patients with Steroid dependent nephrotic syndrome even after discontinuation of steroids Furthermore, it has been shown to be effective in inducing remission in steroid resistant nephrotic syndrome. However ,Cyclosporin A is associated with a plethora of side effects such as hypertension, nephrotoxicity hypertrichosis, gum hyperplasia, gastrointestinal disturbances and tremor.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- children from the age of one year to the age of 18 year who are diagnosed as steroid resistent and steroid dependent nephrotic syndrome and on cyclosporin therapy
Key inclusion criteria (1 listed)
- The study will include all children (on cyclosporine therapy for treatment of Steroid dependent or Steroid resistant nephrotic syndrome ) who present to Nephrology Unit , and clinic at Assiut University Children Hospital during one year duration.
Key exclusion criteria (1 listed)
- children with adequate response to steroid therapy and without relapses or resistance to steroid therapy
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 |
|---|---|---|
| serum protein ,kidney function ,liver function.,serum cholesterolmonthly review of serum protein kidney function ,liver function and serum cholesterol in all patients | Diagnostic test | – |
Primary outcome
evaluation of nephrotic state of each patient for evaluation of efficacy of cyclosporin A therapy
Time frame: one year
measurment of serum albumin ,serum cholesterol and urinary protien excretion
evaluation of side effects of cyclosporin A
Time frame: one year
serial observation of common side effects of cyclosporin A as hypertension, nephrotoxicity hypertrichosis, gum hyperplasia, gastrointestinal disturbances and tremor.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2017.
- Individual participant dataSharing planned: Undecided
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.
Questions about this trial
What is this clinical trial studying?
An observational study of Steroid Dependent and Steroid Resistent Nephrotic Syndrome, run by Assiut University.
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 1 inclusion and 1 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 17 Jul 2017. 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. 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?
Contact Muhammed Mahrous, MD (01003486595, eltellawy270@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
evaluation of nephrotic state of each patient for evaluation of efficacy of cyclosporin A therapy (time frame: one year). The study lists 2 primary outcomes in all. 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 17 Jul 2017.