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

NCT ID: NCT05627557

Last Updated: 2025-12-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

85 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-29

Study Completion Date

2026-09-04

Brief Summary

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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).

Detailed Description

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Conditions

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Childhood Idiopathic Nephrotic Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Obinutuzumab (Group A)

Participants in Group A will receive obinutuzumab 1000 milligrams (mg) (or 20 mg/ kilogram \[kg\] for participants \<45 kg) administered by intravenous (IV) infusion on Days 1, 15, 168 (Week 24), and 182 (Week 26).

Group Type EXPERIMENTAL

Prednisone

Intervention Type DRUG

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

Intervention Type DRUG

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

Acetaminophen/ Paracetamol

Intervention Type DRUG

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

Diphenhydramine Hydrochloride

Intervention Type DRUG

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

Obinutuzumab

Intervention Type DRUG

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

MMF (Group B)

Participants in Group B will receive oral MMF 600 mg/m\^2 twice a day (BID) (target 1200 mg/m2/day in divided doses, maximum 2 g/day) to Week 52.

Group Type ACTIVE_COMPARATOR

Prednisone

Intervention Type DRUG

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.

MMF

Intervention Type DRUG

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

Interventions

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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.

Intervention Type DRUG

Methylprednisolone

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

Intervention Type DRUG

Acetaminophen/ Paracetamol

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

Intervention Type DRUG

Diphenhydramine Hydrochloride

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

Intervention Type DRUG

Obinutuzumab

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

Intervention Type DRUG

MMF

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

Intervention Type DRUG

Other Intervention Names

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Non-Investigational Medicinal Product Non-Investigational Medicinal Product Non-Investigational Medicinal Product Non-Investigational Medicinal Product

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Lucile Packard Children's Hospital - Stanford

Palo Alto, California, United States

Site Status

Memorial Healthcare System

Hollywood, Florida, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Nemours Children's Hospital

Orlando, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Children's Healthcare of Atlanta Center for Advanced Pediatrics

Atlanta, Georgia, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status

University of Utah - Primary Children's Hospital - PPDS

Salt Lake City, Utah, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

UZ Gent

Ghent, , Belgium

Site Status

Irmandade Da Santa Casa de Misericordia de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Site Status

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

São José do Rio Preto, São Paulo, Brazil

Site Status

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

São Paulo, São Paulo, Brazil

Site Status

Peking University First Hospital

Beijing, , China

Site Status

The First Affiliated Hospital of Sun Yat-sen University

Guangzhou, , China

Site Status

The children's hospital , Zhejiang university school of medicine

Hangzhou, , China

Site Status

Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, , China

Site Status

Xi'an Children's Hospital

Xi'an, , China

Site Status

Henan Children's Hospital Zhengzhou Children's Hospital

Zhengzhou, , China

Site Status

Chu Toulouse

Toulouse, Haute-Garonne, France

Site Status

Hopital Femme Mere Enfants

Bron, , France

Site Status

Hopital Henri Mondor

Créteil, , France

Site Status

Hopital Robert Debre

Paris, , France

Site Status

Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

Genoa, Liguria, Italy

Site Status

Ospedale Infantile Regina Margherita - INCIPIT - PIN

Turin, Piedmont, Italy

Site Status

Tokyo Metropolitan Children's Medical Center

Fuchu-Shi, , Japan

Site Status

Hokkaido University Hospital

Hokkaido, , Japan

Site Status

Hyogo prefectural Kobe Children's Hospital

Hyogoken, , Japan

Site Status

Kobe University Hospital

Kobe, , Japan

Site Status

Shiga University Of Medical Science Hospital

Ōtsu, , Japan

Site Status

Kitasato University Hospital

Sagamihara-Shi, , Japan

Site Status

Dokkyo Medical University Hospital

Shimotsuga-Gun, , Japan

Site Status

National Center for Child Health and Development

Tokyo, , Japan

Site Status

Yokohama City University Medical Center

Yokohama, , Japan

Site Status

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Site Status

Dzieciecy Szpital Kliniczny UCK WUM

Warsaw, , Poland

Site Status

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, Spain

Site Status

Hospital Universitario Cruces

Barakaldo, Vizcaya, Spain

Site Status

Countries

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United States Belgium Brazil China France Italy Japan Poland Spain

References

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Larkins NG, Hahn D, Liu ID, Willis NS, Craig JC, Hodson EM. Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6.

Reference Type DERIVED
PMID: 39513526 (View on PubMed)

Dossier C, Bonneric S, Baudouin V, Kwon T, Prim B, Cambier A, Couderc A, Moreau C, Deschenes G, Hogan J. Obinutuzumab in Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome in Children. Clin J Am Soc Nephrol. 2023 Dec 1;18(12):1555-1562. doi: 10.2215/CJN.0000000000000288. Epub 2023 Sep 6.

Reference Type DERIVED
PMID: 37678236 (View on PubMed)

Other Identifiers

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2022-000369-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-505140-19-00

Identifier Type: OTHER

Identifier Source: secondary_id

WA43380

Identifier Type: -

Identifier Source: org_study_id

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