Ireland
--:--IST
Active, not recruiting Phase 3

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

NCT05627557 · tracked via the Priya Life Science Italy tracker
Phase
Phase 3
Started
2023-03-29
Last updated
2025-12-15

Condition(s) studied

Childhood Idiopathic Nephrotic Syndrome

Investigational drug(s) / intervention(s)

Obinutuzumab →MMFPrednisone →Methylprednisolone →Acetaminophen/ Paracetamol →Diphenhydramine Hydrochloride →

Obinutuzumab: Obinutuzumab will be administered as per schedule specified in the respective arm.

MMF: MMF will be administered as per schedule specified in the respective arm.

Prednisone: Participants taking prednisone or equivalent at randomization will follow a guided tapering schedule to reach the goal of 0mg/day by Weeks 4-6 (and no later than Week 8 following randomization and continue without prednisone through Week 52.

Methylprednisolone: Methylprednisolone 80 mg (or 1.5 mg/kg if \</=45 kg) IV will be administered as premedication prior to infusions.

Acetaminophen/ Paracetamol: Acetaminophen 15 mg/kg (maximum dose 1000 mg) will be administered PO as premedication prior to infusions.

Diphenhydramine Hydrochloride: Diphenhydramine HCl 0.5-1 mg/kg (maximum dose 50 mg) will be administered PO or IV as premedication prior to infusions.

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

Sex
ALL
Min age
2 Years
Max age
25 Years
Healthy volunteers
No
Inclusion Criteria: * 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 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 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

Primary outcome measure(s)

  • Percentage of Participants with Sustained Complete Remission at 1 year — At Week 52

Trial sites (39)

FacilityCityRegionStatus
Lucile Packard Children's Hospital - Stanford Palo Alto California
Memorial Healthcare System Hollywood Florida
Nicklaus Children's Hospital Miami Florida
Nemours Children's Hospital Orlando Florida
University of South Florida Tampa Florida
Children's Healthcare of Atlanta Center for Advanced Pediatrics Atlanta Georgia
Hackensack University Medical Center Hackensack New Jersey
Levine Children's Hospital Charlotte North Carolina
University of Utah - Primary Children's Hospital - PPDS Salt Lake City Utah
University of Virginia Health System Charlottesville Virginia
UZ Gent Ghent Belgium
Irmandade Da Santa Casa de Misericordia de Porto Alegre Porto Alegre Rio Grande do Sul
Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS São José do Rio Preto São Paulo
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo São Paulo São Paulo
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
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
Ospedale Infantile Regina Margherita - INCIPIT - PIN Turin Piedmont
Tokyo Metropolitan Children's Medical Center Fuchu-Shi Japan
Hokkaido University Hospital Hokkaido Japan
Hyogo prefectural Kobe Children's Hospital Hyogoken Japan
Kobe University Hospital Kobe Japan
Shiga University Of Medical Science Hospital Ōtsu Japan
Kitasato University Hospital Sagamihara-Shi Japan
Dokkyo Medical University Hospital Shimotsuga-Gun Japan
National Center for Child Health and Development Tokyo Japan
Yokohama City University Medical Center Yokohama Japan
Uniwersyteckie Centrum Kliniczne Gdansk Poland
Dzieciecy Szpital Kliniczny UCK WUM Warsaw Poland
Hospital Sant Joan de Deu - PIN Espluges de Llobregat Barcelona
Hospital Universitario Cruces Barakaldo Vizcaya
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05627557 on ClinicalTrials.gov ↗ ← All trials in Italy