A Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Participants With Childhood Onset Idiopathic Nephrotic Syndrome

Running, not enrolling · Phase 3

Conditions studied: Childhood Idiopathic Nephrotic Syndrome

In brief

This open-label, randomized multicenter study is to assess the efficacy, safety, and pharmacokinetics (PK)/pharmacodynamics (PD) of obinutuzumab compared with mycophenolate mofetil (MMF) in children and young adults (aged \>= 2-25 years) with frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS).

Key facts

Study ID: NCT05627557
Run by: Hoffmann-La Roche
People needed: 85
Starts: 2023-03-29
Expected to finish: 2026-09-04
Last updated by the study team: 2025-12-15

Who can join

Age: 2 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Diagnosis of frequently relapsing nephrotic syndrome (FRNS) or steroid dependent nephrotic syndrome (SDNS) before the age of 18 years
Must be in complete remission defined by the absence of edema, UPCR <= 0.2 g/g at screening and have three consecutive daily urine dipstick readings of trace or negative for protein within the week prior to randomization
Must have had at least one relapse in the 6 months prior to screening, after discontinuation of or while receiving oral corticosteroids and/or immunosuppressive therapy to prevent relapses
Participants having received cyclophosphamide in the 6 months prior to randomization must have experienced at least 1 relapse subsequent to cyclophosphamide discontinuation
Estimated glomerular filtration rate (eGFR) within normal range for age
For females of childbearing potential: participants who agree to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraception, during the treatment period and for 18 months after the final dose of obinutuzumab and for 6 weeks after the final dose of MMF
For males: participants who agree to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods, and agree to refrain from donating sperm during the treatment period and for 90 days after the final dose of MMF

You may not qualify if…

Secondary nephrotic syndrome
History of steroid resistant nephrotic syndrome
History of genetic defects known to directly cause nephrotic syndrome
Treatment with other immunosuppressive medications to prevent relapse, other than MMF or oral corticosteroids within 2 months prior to randomization
Pregnancy or breastfeeding or intending to become pregnant during the study or within 18 months after the final dose of obinutuzumab, or within 6 weeks after the final dose of MMF
Females of childbearing potential, including those who have had a tubal ligation, must have a negative serum pregnancy test result within 28 days prior to initiation of study treatment and a negative urine pregnancy test at Day 1, prior to randomization
History of organ or bone marrow transplant
Participation in another therapeutic trial within 30 days of enrollment or 5 half-lives of the investigational drug
Intolerance or contraindication to study therapies
Participants demonstrating prior treatment failure to MMF as defined by two or more relapses in any 6-month period of time while receiving MMF for at least a 6-month duration
Participants in the judgment of the investigator likely to require systemic corticosteroids for reasons other than idiopathic nephrotic syndrome during the study
Active infection of any kind or any major episode of infection requiring hospitalization or treatment with IV anti-infective medications within 4 weeks prior to screening, or completion of oral anti-infectives within 2 weeks prior to randomization
History of or currently active primary or secondary immunodeficiency, including known history of human immunodeficiency virus (HIV) infection and other severe Immunodeficiency blood disorders
History of progressive multifocal leukoencephalopathy
History of or current cancer, including solid tumors, hematological malignancies, and carcinoma in situ within the past 5 years
Major surgery requiring hospitalization during the 4 weeks prior to screening or during screening
High risk for clinically significant bleeding or any condition requiring plasmapheresis, intravenous immunoglobulin, or acute blood product transfusions
Evidence of any significant or uncontrolled concomitant disease that, in the investigator's judgment, would preclude participant's participation, including but not limited to nervous system, respiratory, cardiac, hepatic, endocrine, malignant, or gastrointestinal disorders
Currently active alcohol or drug abuse or history of alcohol or drug abuse

Where it is running

Lucile Packard Children's Hospital - Stanford — Palo Alto, California, United States
Memorial Healthcare System — Hollywood, Florida, United States
Nicklaus Children's Hospital — Miami, Florida, United States
Nemours Children's Hospital — Orlando, Florida, United States
University of South Florida — Tampa, Florida, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics — Atlanta, Georgia, United States
Hackensack University Medical Center — Hackensack, New Jersey, United States
Levine Children's Hospital — Charlotte, North Carolina, United States
University of Utah - Primary Children's Hospital - PPDS — Salt Lake City, Utah, United States
University of Virginia Health System — Charlottesville, Virginia, United States
UZ Gent — Ghent, Belgium
Irmandade Da Santa Casa de Misericordia de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil
Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS — São José do Rio Preto, São Paulo, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo — São Paulo, São Paulo, Brazil
Peking University First Hospital — Beijing, China
The First Affiliated Hospital of Sun Yat-sen University — Guangzhou, China
The children's hospital , Zhejiang university school of medicine — Hangzhou, China
Tongji Hospital Tongji Medical College Huazhong University of Science and Technology — Wuhan, China
Xi'an Children's Hospital — Xi'an, China
Henan Children's Hospital Zhengzhou Children's Hospital — Zhengzhou, China
Chu Toulouse — Toulouse, Haute-Garonne, France
Hopital Femme Mere Enfants — Bron, France
Hopital Henri Mondor — Créteil, France
Hopital Robert Debre — Paris, France
Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN — Genoa, Liguria, Italy

Full record on ClinicalTrials.gov

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