Idiopathic nephrotic syndrome in childhood MedDRA version: 21.1 Level: PT Classification code 10029164 Term: Nephrotic syndrome System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Trade Name: CellCept
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: Mycophenolatmofetile
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 35-
Sponsors
Ruprecht-Karls-University Heidelberg, Medical Faculty represented by Universitätsklinikum Heidelberg and its Commercial
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects meeting all of the following criteria will be considered for admission to the study: -First episode of steroid-sensitive nephrotic syndrome (SSNS) -in remission induced by daily glucocorticoids -male and female children aged = 1 year and = 10 years at beginning of study (typical age range of patients with SSNS -Ability of the persons having care and custody of the child to understand character and individual consequences of clinical study -Written informed consent of the persons having care and custody of the child (must be available before enrolment in the study) Are the trial subjects under 18? yes Number of subjects for this age range: 340 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects presenting with any of the following criteria will not be included in the study: -Secondary nephrotic syndrome -estimated glomerular filtration rate (eGFR) <90 ml/min x 1.73 m2 BSA -Ongoing treatment with systematically administered glucocorticoids or other immunosuppressive drugs at time of first episode of nephrotic syndrome. • Hemoglobin concentration of =9 g/dL • Leucocyte count of =2.500/µl Refusal of subject (please see also chapter 10.5)• Severe chronic gastrointestinal disease -History of hypersensitivity to mycophenolate mofetil or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of suspension of mycophenolate mofetil (CellCept suspension?) -Participation in other clinical studies or observation period of competing studies, respectively.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main purpose of the study is to show that MMF in the initial treatment of SSNS in children is not inferior regarding maintenance of initial remission and subsequent relapse rate compared to the standard high-dose prednisone regimen;Secondary Objective: Secondary endpoints are divided into five items: 1.Course of the disease as described by the following criteria a.Time from remission to first relapse b.Number of relapses during follow-up c.Mean relapse rate per patient and year d.Number of frequent relapsers e.Time from remission to intensification of immunosuppressive treatment with other drugs due to glucocorticoid-induced toxicity f.Rate of patients who require more intense immunosuppressive treatment 2.Glucocorticoid-associated toxicity 3.Mycophenolate mofetil-associated toxicity 4.Health-related quality of life, 5.Days missing school attendance and days of hospitalization ;Primary end point(s): Occurence of treated relapse within 24 months after end of Initial treatment;Timepoint(s) of evaluation of this end point: Visit 8, 27 month after day 1. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Key secondary endpoint(s): • Course of the disease: Time from remission to first relapse; number of relapses; mean relapse rate per patient and year; incidence of frequent relapsers • Prednisone-associated toxicity: Cumulative prednisone dose (mg/m² BSA); body mass index (standard deviation score); striae; hypertrichosis; acne; arterial hypertension; disturbances of carbohydrate and lipid metabolism; growth failure; cataract; glaucoma; psychological disturbances • MMF-associated toxicity: diarrhea; blood cell count disturbances, infections ;Timepoint(s) of evaluation of this end point: 27 month after day 1 | — |
Countries
Germany
Contacts
Public ContactDepartment of Pediatrics
University Hospital Köln
Outcome results
None listed