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

Last updated: April 2, 2025
Sponsor: Hoffmann-La Roche
Overall Status: Active - Not Recruiting

Phase

3

Condition

Kidney Disease

Kidney Failure (Pediatric)

Nephrotic Syndrome

Treatment

Diphenhydramine Hydrochloride

MMF

Prednisone

Clinical Study ID

NCT05627557
WA43380
2023-505140-19-00
2022-000369-42
  • Ages 2-25
  • All Genders

Study Summary

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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Diagnosis of frequently relapsing nephrotic syndrome (FRNS) or steroid dependentnephrotic syndrome (SDNS) before the age of 18 years

  • Must be in complete remission defined by the absence of edema, UPCR <= 0.2 g/g atscreening and have three consecutive daily urine dipstick readings of trace ornegative for protein within the week prior to randomization

  • Must have had at least one relapse in the 6 months prior to screening, afterdiscontinuation of or while receiving oral corticosteroids and/or immunosuppressivetherapy to prevent relapses

  • Participants having received cyclophosphamide in the 6 months prior to randomizationmust have experienced at least 1 relapse subsequent to cyclophosphamidediscontinuation

  • 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 obinutuzumaband for 6 weeks after the final dose of MMF

  • For males: participants who agree to remain abstinent (refrain from heterosexualintercourse) or use contraceptive methods, and agree to refrain from donating spermduring the treatment period and for 90 days after the final dose of MMF

Exclusion

Exclusion Criteria:

  • 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 thanMMF or oral corticosteroids within 2 months prior to randomization

  • Pregnancy or breastfeeding or intending to become pregnant during the study orwithin 18 months after the final dose of obinutuzumab, or within 6 weeks after thefinal 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 initiationof study treatment and a negative urine pregnancy test at Day 1, prior torandomization

  • History of organ or bone marrow transplant

  • Participation in another therapeutic trial within 30 days of enrollment or 5half-lives of the investigational drug

  • Intolerance or contraindication to study therapies

  • Participants demonstrating prior treatment failure to MMF as defined by two or morerelapses in any 6-month period of time while receiving MMF for at least a 6-monthduration

  • Participants in the judgment of the investigator likely to require systemiccorticosteroids for reasons other than idiopathic nephrotic syndrome during thestudy

  • Active infection of any kind or any major episode of infection requiringhospitalization or treatment with IV anti-infective medications within 4 weeks priorto screening, or completion of oral anti-infectives within 2 weeks prior torandomization

  • History of or currently active primary or secondary immunodeficiency, includingknown history of human immunodeficiency virus (HIV) infection and other severeImmunodeficiency 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 orduring screening

  • High risk for clinically significant bleeding or any condition requiringplasmapheresis, intravenous immunoglobulin, or acute blood product transfusions

  • Evidence of any significant or uncontrolled concomitant disease that, in theinvestigator's judgment, would preclude participant's participation, including butnot 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

Study Design

Total Participants: 80
Treatment Group(s): 6
Primary Treatment: Diphenhydramine Hydrochloride
Phase: 3
Study Start date:
March 29, 2023
Estimated Completion Date:
September 15, 2026

Connect with a study center

  • Hôpital Universitaire des Enfants Reine Fabiola

    Bruxelles, 1090
    Belgium

    Site Not Available

  • UZ Gent

    Gent, 9000
    Belgium

    Site Not Available

  • Instituto Méderi de Pesquisa e Saúde

    Passo Fundo, Rio Grande Do Sul 99010-120
    Brazil

    Site Not Available

  • Irmandade Da Santa Casa de Misericordia de Porto Alegre

    Porto Alegre, Rio Grande Do Sul 90020-090
    Brazil

    Site Not Available

  • Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS

    Sao Jose Do Rio Preto, São Paulo 15090-000
    Brazil

    Site Not Available

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

    Sao Paulo, São Paulo 05403-000
    Brazil

    Site Not Available

  • Peking University First Hospital

    Beijing City, 100034
    China

    Site Not Available

  • The First Affiliated Hospital of Sun Yat-sen University

    Guangzhou City, 510080
    China

    Site Not Available

  • The children's hospital , Zhejiang university school of medicine

    Hangzhou, 310003
    China

    Site Not Available

  • The children's hospital , Zhejiang university school of medicine

    Hangzhou City, 310003
    China

    Site Not Available

  • Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech

    Wuhan, 430030
    China

    Site Not Available

  • Xi'an Children's Hospital

    Xian, 710003
    China

    Site Not Available

  • Henan Children's Hospital Zhengzhou Children's Hospital

    Zhengzhou, 450018
    China

    Site Not Available

  • CHU de Nice

    Nice, Alpes-Maritimes 06000
    France

    Site Not Available

  • Chu Toulouse

    Bron, 69500
    France

    Site Not Available

  • Hopital Femme Mere Enfants

    Bron, 69500
    France

    Site Not Available

  • Hopital Henri Mondor

    Creteil, 94010
    France

    Site Not Available

  • CHU Montpellier- Hopital Arnaud de VIlleneuve

    Montpellier, 34295
    France

    Site Not Available

  • CHU de Nice

    Nice Cedex 1, 06001
    France

    Site Not Available

  • CHU de Nice; Service Système Nerveux Périphérique

    Nice Cedex 1, 06001
    France

    Site Not Available

  • Hopital Necker - Enfants Malades

    Paris, 75015
    France

    Site Not Available

  • Hopital Robert Debre

    Paris, 75019
    France

    Site Not Available

  • Chu Toulouse

    Toulouse, 31059
    France

    Site Not Available

  • Universitätsklinikum Essen

    Essen, 45147
    Germany

    Site Not Available

  • Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

    Genova, Liguria 16147
    Italy

    Site Not Available

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico - Clinica De Marchi - INCIPIT - PIN

    Milano, Lombardia 20122
    Italy

    Site Not Available

  • Ospedale Infantile Regina Margherita - INCIPIT - PIN

    Torino, Piemonte 10126
    Italy

    Site Not Available

  • Hokkaido University Hospital

    Hokkaido, 060-8648
    Japan

    Site Not Available

  • Kobe University Hospital

    Hyogo, 650-0017
    Japan

    Site Not Available

  • Hyogo prefectural Kobe Children's Hospital

    Hyogoken, 6500047
    Japan

    Site Not Available

  • Kitasato University Hospital

    Kanagawa, 252-0375
    Japan

    Site Not Available

  • Yokohama City University Medical Center

    Kanagawa, 232-0024
    Japan

    Site Not Available

  • Dokkyo Medical University Hospital

    Mibu-Machi, 321-0293
    Japan

    Site Not Available

  • Shiga University Of Medical Science Hospital

    Shiga, 520-2192
    Japan

    Site Not Available

  • National Center for Child Health and Development

    Tokyo, 157-8535
    Japan

    Site Not Available

  • Tokyo Metropolitan Children's Medical Center

    Tokyo, 1838561
    Japan

    Site Not Available

  • Uniwersytecki Szpital Kliniczny w Bialymstoku

    Bia?ystok, 15-274
    Poland

    Site Not Available

  • In-VIVO Osrodek Badan Klinicznych

    Bydgoszcz, 85-048
    Poland

    Site Not Available

  • Uniwersyteckie Centrum Kliniczne

    Gdansk,
    Poland

    Site Not Available

  • Dzieciecy Szpital Kliniczny UCK WUM

    Warszawa, 02-091
    Poland

    Site Not Available

  • Hospital Universitario Marques de Valdecilla

    Santander, Cantabria 39008
    Spain

    Site Not Available

  • Hospital Universitario Cruces

    Barakaldo, Vizcaya 48903
    Spain

    Site Not Available

  • Hospital Sant Joan de Deu - PIN

    Barcelona, 08950
    Spain

    Site Not Available

  • Baskent Universitesi - Ankara Hastanesi - Bahcelie

    Bahcelievler, 06490
    Turkey

    Site Not Available

  • Celal Bayar University Medical Faculty

    Manisa, 45010
    Turkey

    Site Not Available

  • Cukurova Universitesi Tip Fakultesi Balcali Hastanesi

    Saricam, 01250
    Turkey

    Site Not Available

  • Cedars Sinai Medical Center

    Los Angeles, California 90048
    United States

    Site Not Available

  • Lucile Packard Children's Hospital - Stanford

    Palo Alto, California 94304
    United States

    Site Not Available

  • University of California Benioff Children's Hospital

    San Francisco, California 94143-2350
    United States

    Site Not Available

  • Children's National Hospital

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Memorial Healthcare System

    Hollywood, Florida 33021
    United States

    Site Not Available

  • Nicklaus Children's Hospital

    Miami, Florida 33155-3009
    United States

    Site Not Available

  • Nemours Children's Hospital

    Orlando, Florida 32827
    United States

    Site Not Available

  • University of South Florida

    Tampa, Florida 33612
    United States

    Site Not Available

  • Children's Healthcare of Atlanta Center for Advanced Pediatrics

    Atlanta, Georgia 30329-2309
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109-0934
    United States

    Site Not Available

  • Children's Mercy Hospital

    Kansas City, Missouri 64108-4619
    United States

    Site Not Available

  • Hackensack University Medical Center

    Hackensack, New Jersey 07601
    United States

    Site Not Available

  • UNC Hospitals Outpatient Center at Eastowne

    Chapel Hill, North Carolina 27514-2286
    United States

    Site Not Available

  • Levine Children's Hospital

    Charlotte, North Carolina 28203-5866
    United States

    Site Not Available

  • Duke University Health Systems

    Durham, North Carolina 27710
    United States

    Site Not Available

  • University of Utah - Primary Children's Hospital - PPDS

    Salt Lake City, Utah 84113-1103
    United States

    Site Not Available

  • University of Virginia Health System

    Charlottesville, Virginia 22903
    United States

    Site Not Available

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