Study of Initial Steroid Treatment in Young Children With Nephrotic Syndrome

• ClinicalTrials.gov Identifier: NCT04536181
• Recruitment Status: Withdrawn
• First Posted: September 2, 2020
• Last Update Posted: December 17, 2020
• Sponsor: Children's Hospital of Fudan University
• Information provided by (Responsible Party): Children's Hospital of Fudan University

Study Description

Brief Summary:

This study is a multicentric, prospective, randomized, double blind, placebo controlled trial compared the efficacy of 6 months vs. 3 months of initial prednisolone therapy in decreasing the incidence of FRNS with steroid-sensitive nephrotic syndrome in children age 1 year up to 6 years. Three months tapering prednisolone or placebo are administrated follow by open-label 3 months standard prednisolone therapy.The cumulative incidence of FRNS and adverse events for one year were evaluated in the 3-month and 6-month groups.

Condition or disease	Intervention/treatment	Phase
Steroid-Sensitive Nephrotic Syndrome	Drug: Prednisolone; Drug: Placebo	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 0 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Comparative Study of 3 or 6 Months Initial Steroid Treatment in Children Under 6 Years of Age With Steroid Sensitive Nephrotic Syndrome：a Randomized, Double-blind, Placebo-controlled Study
• Estimated Study Start Date: January 1, 2021
• Estimated Primary Completion Date: September 1, 2022
• Estimated Study Completion Date: October 1, 2022

Arms and Interventions

Arm	Intervention/treatment
Placebo Comparator: 3 months group; Subjects will receive 12-weeks of placebo following randomization	Drug: Placebo; 12 weeks simulated tablets
Experimental: 6 months group; 12 Weeks of Prednisolone Therapy Subjects will add an additional 12 weeks of Prednisolone to follow pre-randomization standard of care prednisolone.	Drug: Prednisolone; 12 weeks prednisolone treatment; Other Name: prednisone

Outcome Measures

Primary Outcome Measures :

1. Occurrence of frequent relapses of nephrotic syndrome at 12th month after 3 month standard treatment [ Time Frame: 12 month period after 3 month standard treatment ]
   Proportion of patients with frequent relapses at 12th month after 3 month standard treatment.Frequent relapses nephrotic syndrome was defined as two or more relapses within 6 months after completing initial treatment, or four relapses within any period of 12 months, including relapses during initial treatment.

Secondary Outcome Measures :

1. Number of relapses during 12 months follow up after 3 month standard treatment [ Time Frame: 12 month period after 3 month standard treatment ]
   Number of nephrotic syndrome relapses per patient year during the 12-month period after 3 month standard treatment
2. Time to first relapse (days) [ Time Frame: 12 month period after 3 month standard treatment ]
   Number of days from randomization to occurrence of first relapse
3. Cumulative prednisolone [or corticosteroid equivalent] received during 12 month period after 3 month standard treatment [ Time Frame: 12 month period after 3 month standard treatment ]
   Total amount of prednisolone [or corticosteroid equivalent] received, as mg/kg/day or mg/m2/day as intervention and for treatment of relapses, during 12 months follow up after 3 month standard treatment
4. The use of steroid-sparing medications [ Time Frame: 12 month period after 3 month standard treatment ]
   The proportion of patients in each study arm treated with steroid-sparing strategies or medications.it is a binary varibale (1/0). The variable wolud be sette into "1", if the patients use the steroid-sparing medications such as cyclophosphamide, levamisole, mycophenolate mofetil，rituximab and so on.
5. Adverse events during 12-month period after 3 month standard treatment [ Time Frame: 12 month period after 3 month standard treatment ]
   Number of adverse events experienced, related or unrelated to corticosteroid use
6. Change in height during 12-month period after 3 month standard treatment [ Time Frame: 12 month period after 3 month standard treatment ]
   The standard deviation scores (SDS) for height will be measured at 12th month and randomization. Change in height is SDS at 12 month minus that of randomization.
7. Occurrence of steroid-dependent nephrotic syndrome at 12th month after 3 month standard treatment [ Time Frame: 12 month period after 3 month standard treatment ]
   Proportion of patients with steroid-dependent nephrotic syndrome at 12th month after 3 month standard treatment.Steroid-dependent nephrotic syndrome was defined as steroid-sensitive nephrotic syndrome with 2 or more consecutive relapses during tapering or within 14 days of stopping steroids.

Eligibility Criteria

• Ages Eligible for Study: 1 Year to 6 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Idiopathic, steroid-sensitive, first episode of nephrotic syndrome
• Age 12 months up to 6 years
• Written informed consent

Exclusion Criteria:

• Nephrotic syndrome known to be secondary to a systemic disorder, e.g., Immunoglobulin A (IgA) nephropathy, systemic lupus erythematosus, Henoch Schonlein purpura, vasculitis, , hepatitis B or Alport syndrome.
• Therapy with prednisolone for prior episodes of nephrotic syndrome
• Persistent estimated glomerular filtration rate (GFR) <75 ml/min/1.73 m2
• Patients who show relapse during the first 3 months of pre-randomization corticosteroid therapy for nephrotic syndrome
• Patients with initial steroid resistance
• Patients who are allergic to glucocorticoids
• The compliance of patients or their guardians is poor

Contacts and Locations

Locations

China, Shanghai

Children's hospital of Fudan university

Shanghai, Shanghai, China, 200000

China

Shanghai Children's Hospital

Shanghai, China

Shanghai Children's Medical Center

Shanghai, China

Xinhua Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, China

Sponsors and Collaborators

Children's Hospital of Fudan University

Investigators

• Principal Investigator: Hong Xu, PhD.MD.; Children's Hospital of Fudan University

More Information

• Responsible Party: Children's Hospital of Fudan University
• ClinicalTrials.gov Identifier: NCT04536181
• Other Study ID Numbers: CSICS
• First Posted: September 2, 2020
• Last Update Posted: December 17, 2020
• Last Verified: December 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Nephrotic Syndrome; Nephrosis; Hypersensitivity; Syndrome; Disease; Pathologic Processes; Immune System Diseases; Kidney Diseases; Urologic Diseases; Prednisone; Prednisolone; Anti-Inflammatory Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Antineoplastic Agents, Hormonal; Antineoplastic Agents