Randomized Trial Evaluating Mycophenolate Mofetil in Children With Nephrotic Syndrome After Rituximab Treatment

NCT04531865 | Withdrawn Phase 3

• 1 other identifier: FRSDRM
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 4

Brief Summary

The aim of this study is to evaluate the efficacy and safety of maintenance Mycophenolate Mofetil following single course of Rituximab in maintaining remission over 12 months among Children with frequently-relapsing or steroid-dependent nephrotic syndrome

Conditions

Frequently Relapsing Nephrotic Syndrome; Steroid-Dependent Nephrotic Syndrome

Outcome Measures

Primary Outcomes (1)

• 1-year relapse-free survival rate

  The rate of no relapse within 1 year

  1-year period after randomization

Secondary Outcomes (6)

• The concentration for MPA-area under curve(AUC)

  At 48 weeks

• Proportion of patients with a relapse

  6 months period after randomization

• Time to relapse (days)

  1-year period after randomization

• B-Cell Recovery Time

  1-year period after randomization

• Change in growth velocity

  1-year period after randomization

Study Arms (2)

Rituximab and Mycophenolate Mofetil

EXPERIMENTAL

First course Course Rituximab at Randomization. Addition of Maintenance Mycophenolate Mofetil from 4 Month onwards.

Drug: Rituximab; Drug: Mycophenolate Mofetil

Rituximab Only

PLACEBO COMPARATOR

First course Course Rituximab at Randomization. Addition of Maintenance Placebo tablets matching Mycophenolate mofetil from 4 Month onwards.

Drug: Rituximab; Drug: Placebo tablets matching Mycophenolate Mofetil

Interventions

Rituximab DRUG

Rituximab: 375 mg/m2 intravenously on day 0 and day 7

Also known as: Rituximab Biosimilar HLX01

Rituximab Only; Rituximab and Mycophenolate Mofetil

Mycophenolate Mofetil DRUG

Addition of Maintenance Mycophenolate Mofetil from 4 Month onwards. Dose: 20\~30mg/kg/day，BID. Total duration : 8 months.

Also known as: Mycophenolate Mofetil Dispersible tablets（CYCOPIN®）

Rituximab and Mycophenolate Mofetil

Placebo tablets matching Mycophenolate Mofetil DRUG

Addition of Maintenance Placebo tablets matching Mycophenolate Mofetil from 4 Month onwards. Dose: 20\~30mg/kg/day，BID. Total duration : 8 months.

Rituximab Only

Eligibility Criteria

• Age: 1 Year - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children between 1 and 16 years with Frequently-relapsing or Steroid-dependent Nephrotic Syndrome
• Estimated glomerular filtration rate (eGFR) ≥90 ml/min per 1.73 m2 at study entry.
• Remission at study entry
• Patients in whom ≥5 CD20-positive cells/μL are observed in the peripheral blood.
• Parents willing to give informed written and audiovisual consent.

You may not qualify if:

• Patients who have been diagnosed with nephritic- NS, such as immunoglobulin A(IgA) nephropathy, prior to assignment or in whom secondary NS is suspected.
• Patients showing one of the following abnormal clinical laboratory values:
• \) Leukocytes \< 3000/μL. 2) Neutrophils \< 1500/μL. 3) Platelets \< 50,000/μL. 4) Alanine aminotransferase (ALT) \> 2.5× upper limit of normal value. 5) Aspartate aminotransferase (AST) \> 2.5× upper limit of normal value. 6) Positive for hepatitis B surface (HBs) antigen, HBs antibody, hepatitis B core (HBc) antibody, or hepatitis C virus (HCV) antibody. 7) Positive for HIV antibody.
• \. Patients meeting one of the following infection criteria:
• \) Presence or history of severe infections within 6 months prior to assignment.2) Presence or history of opportunistic infections within 6 months prior to assignment.3) Presence of active tuberculosis.4) Patients with a history of tuberculosis or in whom tuberculosis is suspected.5) Presence or history of active hepatitis B or hepatitis C or hepatitis B virus carrier.6) Presence of human immunodeficiency virus (HIV) infection.
• \. Presence or history of angina pectoris, cardiac failure, myocardial infarction, or serious arrhythmia (findings observed under Grade 4 of the Common Terminology Criteria for Adverse Events (CTCAE)).
• \. Presence or history of autoimmune diseases or vascular purpura.
• \. Presence or history of malignant tumor.
• \. History of organ transplantation.
• \. History of drug allergies to methylprednisolone, acetaminophen, cetirizine, mycophenolate mofetil,rituximab, or any of the above drugs
• \. Uncontrollable hypertension.
• \. Having received a live vaccine within 4 weeks prior to enrollment.
• \. Patients who do not agree with contraception during the study period.
• \. Judged inappropriate for this study by the treating or study physicians.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead
• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine: collaborator
• Shanghai Children's Medical Center: collaborator
• Shanghai Children's Hospital: collaborator

Study Sites (4)

Children's hospital of Fudan university

Shanghai, Shanghai Municipality, 200000, China

Location

Shanghai Children's Hospital

Shanghai, China

Location

Shanghai Children's Medical Center

Shanghai, China

Location

Xinhua Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, China

Location

MeSH Terms

Conditions

Nephrotic Syndrome

Interventions

Rituximab; Mycophenolic Acid

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)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Xu Hong, PhD.MD.

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 26, 2020
• First Posted: August 31, 2020
• Study Start: January 1, 2021
• Primary Completion: September 1, 2022
• Study Completion: October 1, 2022
• Last Updated: December 29, 2020

Record last verified: 2020-12

Locations

CN (4)