Prednisolone Trial in Children Younger Than 4 Years

NCT03141970 | Completed Phase 3 DMC FDA Drug

• 2 other identifiers: v1.0CTRI/2015/06/005939
• Study Type: interventional
• Target: 170
• Locations: 2 countries
• Sites: 5

Brief Summary

This study is a multicentric, randomized, parallel group, open label controlled trial of children age 1 year up to 4 years with new onset, idiopathic nephrotic syndrome. It is designed to test the initial duration of steroid therapy of either 3 month or 6 month total duration. Participants will be randomized to either extend their pre-trial 3 months (12 weeks) of standard of care corticosteroid therapy to add an additional 12 weeks of therapy or to stop therapy. Pre-trial standard of care corticosteroids will include 60 mg/m2/day for 6 weeks followed by 40 mg/m2/day every other day for 6 weeks of prednisolone or equivalent. The trial intervention will therefore be an additional 12 vs 0 weeks of corticosteroids in these children with idiopathic nephrotic syndrome.

Conditions

Nephrotic Syndrome

Keywords

Proteinuria Hypoproteinemia Edema Hypoalbuminemia Prednisone

Outcome Measures

Primary Outcomes (1)

• Relapse of nephrotic syndrome during 12 months after randomization

  Proportion of patients with one or more relapse(s) of nephrotic syndrome

  12 month period following randomization

Secondary Outcomes (7)

• Number of relapses during 12 months follow up

  12 month period following randomization

• Time to first relapse (days)

  12 month period following randomization

• Occurrence of frequent relapses of nephrotic syndrome during 12 months from randomization

  12 month period following randomization

• Cumulative prednisolone [or corticosteroid equivalent] received during 12 month period from randomization

  12 month period following randomization

• The use of steroid-sparing medications

  12 month period following randomization

Other Outcomes (21)

• In a subgroup of 20 patients, the proportions of the following cell subsets, at baseline and at 6 and 12 months after randomization and at first relapse

  12 month period following randomization

• Relapse of nephrotic syndrome during 24 months after randomization

  24 month period following randomization

• Number of relapses during 24 months follow up

  24 month period following randomization

Study Arms (2)

Intervention: Prednisolone

EXPERIMENTAL

Drug: 12- Weeks of Prednisolone Therapy Subjects will add an additional 12 weeks of Prednisolone to follow pre-randomization standard of care prednisolone. Post randomization Prednisolone therapy of 30 mg/m2 on alternate days for 4 weeks, 20 mg/m2 on alternate days for 4 weeks, and 10 mg/m2 on alternate days for 4 weeks

Drug: Prednisolone

No intervention

NO INTERVENTION

Subjects will NOT receive 12-weeks of additional Prednisolone therapy following randomization

Interventions

Prednisolone DRUG

Prednisolone for 12 weeks as follows 30 mg/m2 on alternate days for 4 weeks 20 mg/m2 on alternate days for 4 weeks 10 mg/m2 on alternate days for 4 weeks

Also known as: Prednisone

Intervention: Prednisolone

Eligibility Criteria

• Age: 1 Year - 4 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Idiopathic, steroid-sensitive, first episode of nephrotic syndrome
• Age 12 months up to 48 months
• Written informed consent

You may not qualify if:

• 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.
• Persistent estimated glomerular filtration rate (GFR) \<75 ml/min/1.73 m2,
• Therapy with prednisolone for prior episodes of nephrotic syndrome,
• Therapy with corticosteroids in the past 3 months, in a dose more than 1 mg/kg for \>14 days for any other reason,
• Corticosteroid therapy for initial episode of nephrotic syndrome prior to randomization varying from pre-specified protocol on more than 14 days,
• Patients who show relapse during the first 3 months of pre-randomization corticosteroid therapy for nephrotic syndrome,
• Unclear treatment history,
• Gross hematuria,
• Patients with initial steroid resistance,
• Participation in any other drug study during the course of this study.
• Participation in more than one study without approval from the researchers involved in each study,

Sponsors & Collaborators

• All India Institute of Medical Sciences: lead
• NephCure Accelerating Cures Institute: collaborator
• University of Michigan: collaborator
• Department of Biotechnology, Government of India (funding agency): collaborator

Study Sites (5)

Cedars-Sinai Medical Center, Pediatric IBD & Pediatric Nephrology

Los Angeles, California, 90048, United States

Location

Stanford University Medical Center, Department of Pediatrics, Division of Nephrology

Stanford, California, 94305, United States

Location

University of Michigan Department of Pedatric Nephrology

Ann Arbor, Michigan, 48109, United States

Location

Levine's Children/Carolinas HealthCare System

Charlotte, North Carolina, 28207, United States

Location

All India Institute of Medical Sciences

New Delhi, National Capital Territory of Delhi, 110029, India

Location

Related Publications (1)

• Hahn D, Samuel SM, Willis NS, Craig JC, Hodson EM. Corticosteroid therapy for nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Aug 22;8(8):CD001533. doi: 10.1002/14651858.CD001533.pub7.

  PMID: 39171624 DERIVED

MeSH Terms

Conditions

Nephrotic Syndrome

Interventions

Prednisolone; Prednisone

Condition Hierarchy (Ancestors)

Nephrosis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Pregnadienediols

Study Officials

• Arvind Bagga, MD

  All India Institute of Medical Sciences, New Delhi, India

  PRINCIPAL INVESTIGATOR

• Debbie Gipson, MD

  University of Michigan

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor, Department of Pediatrics

Model Details: Multicentric, Parallel group, Open label randomized controlled trial

Study Record Dates

• First Submitted: March 17, 2017
• First Posted: May 5, 2017
• Study Start: July 1, 2015
• Primary Completion: October 31, 2020
• Study Completion: October 31, 2021
• Last Updated: January 30, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP

Locations

US (4); IN (1)