A Phase 4 interventional study of Tacrolimus and Mycophenolate Mofetil in Nephrotic Syndrome in Children, sponsored by The Children's Hospital of Zhejiang University School of Medicine. Completed at 12 sites in China. Open to participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2023-10-17.
Sponsored by The Children's Hospital of Zhejiang University School of Medicine · Phase 4, Interventional, and Treatment
Primary nephrotic syndrome accounts for approximately 90% of the total number of nephrotic syndrome in childhood and it is the most common glomerular disease in children. Although treatment with steroids is uesful for primary nephrotic syndrome, proning to cause frequent relapse/steroid-dependent nephrotic syndrome after treatment, and the usage of immunosuppressive agents has become a new choice for the treatment of such patients. This study is a prospective, randomized, multicenter, open, parallel controlled trial, evaluating the efficacy and safety of steroid combined with the immunosuppressive agents which are tacrolimus and mycophenolate mofetil to children who with frequently relapsing or steroid-dependent nephrotic syndrome, all we wish to obtain the proper drug choice and individualized treatment options for children with nephrotic syndrome.
Although steroids are recognized as first-line treatments for nephrotic syndrome, the vast majority of children relapse, and about half of them have frequent relapse or steroids dependence after treatment with steroids alone. Some children experienced steroids-resistance after multiple relapses, and eventually developed into chronic kidney dysfunction. Long-term or repeated application of large doses of steroids will lead to side effects such as obesity, growth retardation, and hypertension. Although the treatment of steroids with immunosuppressive agents is a new choice for the treatment of such patients, traditional immunosuppressive agents such as cyclophosphamide and cyclosporine A will bring some serious irreversible side effects, while immunosuppressive agents tacrolimus has the dual effects of immunosuppression and podocyte protection, and is more widely used in the department of nephrology, what's more, the other immunosuppressive agents mycophenolate mofetil has advantage of no kidney toxic, less adverse reactions and higher safety, which gradually being valued by nephrologists in recent years. This study mainly compares the efficacy and safety of tacrolimus and mycophenolate mofetil in the treatment of children with frequently relapsing or steroids-dependent nephrotic syndrome, in order to provide a more effective and safer treatment for children with nephrotic syndrome as well as the therapeutic medication options.
Exclusion Criteria:
Tacrolimus: 0.5mg and 1mg; Capsule; 0.05-0.10mg/kg/day，BID; Steroid: 5mg; Oral tablets; 1.0-1.5 mg/kg, qod or 0.5-0.75 mg/kg/day, qd;
Drug: Tacrolimus
Mycophenolate Mofetil: 250mg; Dispersible tablets; 20\~30mg/kg/day，BID; Steroid: 5mg; Oral tablets; 1.0-1.5 mg/kg, qod or 0.5-0.75 mg/kg/day, qd;
Drug: Mycophenolate Mofetil
The patients will be divided into two groups randomly. Tacrolimus dose: 0.05-0.10 mg/kg/day, BID. The concentration for tacrolimus is 5-10 ng/ml，then reduce the dosage of drugs to maintian the concentration for tacrolimus is \< 5ng/ml. Total duration : 1 year. Steroid dose: 1.0-1.5 mg/kg, qod or 0.5-0.75 mg/kg/day, qd， then gradually taper the steroid to 5mg/day.
Also known as: Tacrolimus capsules（CYONSE®）
The patients will be divided into two groups randomly. Mycophenolate Mofetil dose: 20\~30mg/kg/day，BID. The concentration for MPA-AUC is 30\~50 μg.h/ml，then reduce the dosage of drugs to maintian the concentration for MPA-AUC is ≤40 μg.h/ml. Total duration : 1 year. Steroid dose: 1.0-1.5 mg/kg, qod or 0.5-0.75 mg/kg/day, qd， then gradually taper the steroid to 5mg/day.
Also known as: Mycophenolate Mofetil Dispersible tablets（CYCOPIN®）
1-year relapse-free survival rate
The rate of no relapse within 1 year
Time frame: 1-year period after randomization
Relapse of nephrotic syndrome during 12 months after randomization
Proportion of patients with one or more relapse(s) of nephrotic syndrome
Time frame: 1-year period after randomization
Number of relapses during 12 months follow up
Number of nephrotic syndrome relapses per patient year during the 12 months period after randomization
Time frame: 1-year period after randomization
The first time to relapse
The first time to relapse after patients taking part in this study
Time frame: 1-year period after randomization
Cumulative prednisone dosage (milligrams per kilogram per year)
The total dosage of prednisones from the beginning to the end of the trial
Time frame: 1-year period after randomization
Change in serum cholesterol, hemoglobin and blood albumin of the patients
The changes of serum cholesterol, hemoglobin and blood albumin in each follow-up during the study
Time frame: 1-year period after randomization
Change in renal function of the patients
The change for renal function was judged by the changes of serum creatinine and estimated glomerular filtration rate in each follow-up during the study
Time frame: 1-year period after randomization
Change in anthropometry and growth velocity during 12-month period after randomization
Changes in standard deviation scores for weight, height and body mass index during 12-month period after randomization
Time frame: 1-year period after randomization
Adverse event
The number of harmful reactions and the types of adverse events during the study
Time frame: 1-year period after randomization
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Plan to share: No — We could not share data without patients' agreement.
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The Children's Hospital of Zhejiang University School of Medicine