STERN: Study of the Telitacicept in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome

Sponsor

The Children's Hospital of Zhejiang University School of Medicine (Other)

Overall Status

Recruiting

CT.gov ID

NCT06125405

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The main objective is to evaluate the effectiveness of telitacicept in pediatric patients with frequently relapsing or steroid dependent nephrotic syndrome within the 52-week follow-up.

Condition or Disease	Intervention/Treatment	Phase
Nephrotic Syndrome in Children Telitacicept	Drug: Telitacicept	Phase 3

Detailed Description

Nephrotic syndrome(NS) is the most common glomerular disease in children. Approximately 45-50% of patients with nephrotic syndrome exhibit frequent relapses or are dependent on steroid therapy. Frequent relapses or steroid dependence in nephrotic syndrome have been challenging issues for clinicians. Long-term, repeated, and high-dose oral steroid use can lead to side effects such as obesity, delayed development, hypertension, diabetes, glaucoma, osteoporosis, and increased susceptibility to infections. The addition of traditional immunosuppressants such as cyclophosphamide and tacrolimus can cause severe and irreversible side effects. Therefore, exploring new drugs and their application protocols is particularly important. Telitacicept has a unique dual-target mechanism that can inhibit B cell maturation and differentiation at multiple stages, thereby inhibiting B cell activity. Clinical studies have confirmed its significant efficacy in various kidney diseases, such as lupus nephritis, IgA nephropathy, and adult recurrent minimal change nephrotic syndrome; moreover, it has good safety profiles.

Therefore, through this prospective, single-center, open-label clinical trial, we aim to evaluate whether telitacicept provides superior efficacy compared to existing conventional treatment regimens for childhood frequent relapse (FR) or steroid-dependent (SD) nephrotic syndrome (NS), and assess its safety profile. Our goal is to provide an optimized treatment plan for childhood FRNS or SDNS.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Study of the Telitacicept in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome

Anticipated Study Start Date :

Nov 24, 2023

Anticipated Primary Completion Date :

Oct 24, 2026

Anticipated Study Completion Date :

Oct 24, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Telitacicept group Weekly administration (administration time can be within 1 week + 3 days). Body weight and dosage: for subjects with body weight greater than 10kg and less than or equal to 20kg, the dose of Telitacicept is 40mg; for subjects with body weight greater than 20kg and less than or equal to 40kg, the dose of Telitacicept is 80mg; for subjects with body weight greater than 40kg and less than or equal to 60kg, the dose of Telitacicept is 120mg; for subjects with body weight greater than or equal to 60kg, the dose of Telitacicept is 160mg. Treatment duration: 52 weeks.	Drug: Telitacicept The study duration was 52 weeks, with the experimental group receiving subcutaneous injections of Telitacicept once weekly for a total of 52 weeks.

Arm

Intervention/Treatment

Experimental: Telitacicept group

Weekly administration (administration time can be within 1 week + 3 days). Body weight and dosage: for subjects with body weight greater than 10kg and less than or equal to 20kg, the dose of Telitacicept is 40mg; for subjects with body weight greater than 20kg and less than or equal to 40kg, the dose of Telitacicept is 80mg; for subjects with body weight greater than 40kg and less than or equal to 60kg, the dose of Telitacicept is 120mg; for subjects with body weight greater than or equal to 60kg, the dose of Telitacicept is 160mg. Treatment duration: 52 weeks.

Drug: Telitacicept

The study duration was 52 weeks, with the experimental group receiving subcutaneous injections of Telitacicept once weekly for a total of 52 weeks.

Outcome Measures

Primary Outcome Measures

1-year relapse-free survival rate [1-year period after enrollment]
The rate of no relapse within 1 year

Secondary Outcome Measures

Relapse of nephrotic syndrome during 12 months after enrollment [1-year period after enrollment]
Proportion of patients with one or more relapse(s) of nephrotic syndrome
Number of relapses during 12 months follow up [1-year period after enrollment]
Number of nephrotic syndrome relapses per patient year during the 12 months period after enrollment
The first time to relapse [1-year period after enrollment]
The first time to relapse after patients taking part in this study
Cumulative prednisone dosage (milligrams per kilogram per year) [1-year period after enrollment]
The total dosage of prednisones from the beginning to the end of the trial
Change in hemoglobin of the patients [1-year period after enrollment]
The changes of hemoglobin (g/L) in each follow-up during the study
Change in blood albumin of the patients [1-year period after enrollment]
The changes of blood albumin (g/L）in each follow-up during the study
Change in renal function of the patients [1-year period after enrollment]
The change for renal function was judged by the changes of estimated glomerular filtration rate (eGFR in ml/min/1.73m^2) in each follow-up during the study
Change in mass index (BMI) during 12-month period after enrollment [1-year period after enrollment]
Changes in standard deviation scores for weight (Wt in kilograms), height (Ht in meters) will be combined to report body mass index (BMI in kg/m^2) during 12-month period after enrollment

Other Outcome Measures

Adverse event [1-year period after enrollment]
The number of harmful reactions and the types of adverse events during the study

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 18 Years

Sexes Eligible for Study:

All

Accepts 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 rituximab was used within 6 months, no tacrolimus, mycophenolate mofetil, cyclosporine A, or cyclophosphamide was used within 3 months, no ACTH was used within 3 months prior to the enrollment

Exclusion Criteria:

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 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