Study of Tacrolimus vs Mycophenolate Mofetil in Pediatric Patients With Nephrotic Syndrome (STAMP)

Zhejiang University

Status and phase: Completed; Phase 4

Conditions: Nephrotic Syndrome in Children

Treatments: Drug: Mycophenolate Mofetil; Drug: Tacrolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT04048161

STAMP

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 270 patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Sensitive but frequent relapses or steroids dependence nephrotic syndrome
• Age: 2 to 18 years old
• Normal renal function: estimated glomerular filtration rate ≥90ml/min/1.73m2
• Morning urine protein <1+ or urine protein-creatinine ratio <0.2g/g (<20 mg/mmol) for 3 consecutive days and above when in enroll
• No tacrolimus, mycophenolate mofetil, cyclosporine A, rituximab or cyclophosphamide was used within 2 years prior to the enrollment

Exclusion criteria

• steroids-resistant nephrotic syndrome
• Family history of nephrotic syndrome, chronic glomerulonephritis or uremia
• Leukopenia (White Blood Cells ≤ 3.0 * 10^9 / L)
• Moderate to severe anemia (hemoglobin <9.0 g/dL)
• Thrombocytopenia (platelet count <100*10^12/L)
• Positive Hepatitis B virus serological indicators (Hepatitis B surface antigen or / and Hepatitis B virus e antigen or / and Hepatitis B core antibody), Hepatitis C virus-positive or patients with abnormal liver function (2 or more times of alamine aminotransferase or total bilirubin was exceeded the normal value, and continued to rise for 2 weeks)
• There are chronic active infections such as Epstein-Barrvirus, cytomegalovirus or Mycobacterium tuberculosis, and the usage of steroids and immunosuppressive agents may aggravate the state of an illness
• Secondary nephrotic syndrome (such as purpuric nephritis, lupus nephritis, etc.)
• Those who with hematological or endocrine system diseases as well as serious organs illness such as heart, liver or kidney
• Those who with other autoimmune diseases or primary immunodeficiencies or tumors
• Those who was known to be sensitized to tacrolimus, mycophenolate mofetil, glucocorticoids, or any of the above drugs
• Those who have participated in other clinical trials within three months prior to the enrollment
• Those who was not suitable for participating this study judged by investigator

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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270 participants in 2 patient groups

Tacrolimus(Group A)

Experimental group

Description:

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;

Treatment:

Drug: Tacrolimus

Mycophenolate Mofetil(Group B)

Active Comparator group

Description:

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;

Treatment:

Drug: Mycophenolate Mofetil