A Descriptive Study on the Effectiveness and Safety of Cyclosporin A Therapy in Steroid Dependent and Steroid Resistant Childhood Nephrotic Syndrome

Cyclosporin A Therapy in Childhood Nephrotic Syndrome



Sponsors

Lead Sponsor



Source

Assiut University

Oversight Info

Has Dmc

No

Is Fda Regulated Drug

No


Brief 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

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.

Overall Status

Not yet recruiting

Start Date

2018-04-01

Completion Date

2019-08-01

Primary Completion Date

2019-04-01

Phase

N/A

Study Type

Observational

Primary Outcome

Measure

Time Frame

evaluation of nephrotic state of each patient for evaluation of efficacy of cyclosporin A therapy
one year
evaluation of side effects of cyclosporin A
one year

Number Of Groups

1

Enrollment

50

Condition


Intervention

Intervention Type

Diagnostic Test

Intervention Name


Description

monthly review of serum protein kidney function ,liver function and serum cholesterol in all patients


Eligibility

Study Pop

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

Sampling Method

Probability Sample

Criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- children with adequate response to steroid therapy and without relapses or resistance
to steroid therapy

Gender

All

Minimum Age

1 Year

Maximum Age

18 Years


Overall Contact

Last Name

Muhammed Mahrous, MD

Phone

01003486595

Email



Verification Date

2017-07-01

Lastchanged Date

2017-07-13

Firstreceived Date

2017-07-11

Responsible Party

Responsible Party Type

Principal Investigator

Investigator Affiliation

Assiut University

Investigator Full Name

Abdullah Ahmed Abdel_Ghany

Investigator Title

principal investigator


Has Expanded Access

No

Condition Browse


Intervention Browse

Mesh Term

Cyclosporins

Cyclosporine



Firstreceived Results Date

N/A

Overall Contact Backup

Last Name

Ahlam Badawy

Phone

01006807866

Email



Firstreceived Results Disposition Date

N/A

Study Design Info

Observational Model

Cohort

Time Perspective

Cross-Sectional



ClinicalTrials.gov processed this data on July 17, 2017

Conditions

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Interventions

Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied. Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase

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In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.



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