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

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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).

Conditions:

Childhood Idiopathic Nephrotic Syndrome

Study Start (Actual) 2023-03-29

Primary Completion (Estimated) 2026-08-15

Study Completion (Estimated) 2026-08-15

Enrollment (Estimated) 80

Study Type INTERVENTIONAL

Phase PHASE3

Locations:

📍 Los Angeles, California, United States

📍 Palo Alto, California, United States

📍 San Francisco, California, United States

📍 Washington, District of Columbia, United States

📍 Hollywood, Florida, United States

📍 Miami, Florida, United States

📍 Orlando, Florida, United States

📍 Tampa, Florida, United States

📍 Atlanta, Georgia, United States

📍 Ann Arbor, Michigan, United States

📍 Kansas City, Missouri, United States

📍 Hackensack, New Jersey, United States

📍 Chapel Hill, North Carolina, United States

📍 Charlotte, North Carolina, United States

📍 Durham, North Carolina, United States

📍 Salt Lake City, Utah, United States

📍 Charlottesville, Virginia, United States

📍 Bruxelles, Belgium

📍 Gent, Belgium

📍 Passo Fundo, RS, Brazil

📍 Porto Alegre, RS, Brazil

📍 Sao Jose Do Rio Preto, SP, Brazil

📍 Sao Paulo, SP, Brazil

📍 Beijing City, China

📍 Guangzhou City, China

📍 Hangzhou City, China

📍 Wuhan, China

📍 Xian, China

📍 Zhengzhou, China

📍 Bron, France

📍 Creteil, France

📍 Montpellier, France

📍 Nice Cedex 1, France

📍 Paris, France

📍 Genova, Liguria, Italy

📍 Milano, Lombardia, Italy

📍 Torino, Piemonte, Italy

📍 Hokkaido, Japan

📍 Hyogoken, Japan

📍 Hyogo, Japan

📍 Kanagawa, Japan

📍 Mibu-Machi, Japan

📍 Shiga, Japan

📍 Tokyo, Japan

📍 Bia?ystok, Poland

📍 Bydgoszcz, Poland

📍 Gdansk, Poland

📍 Warszawa, Poland

📍 Santander, Cantabria, Spain

📍 Barakaldo, Vizcaya, Spain

📍 Barcelona, Spain

📍 Bahcelievler, Turkey

📍 Manisa, Turkey

📍 Saricam, Turkey

More locations available...

Eligibility Criteria

Description

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

Ages Eligible for Study: 2 Years to 25 Years (CHILD, ADULT)

Sexes Eligible for Study: ALL

Accepts Healthy Volunteers: No

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted2022-11-04
First Submitted that Met QC Criteria2022-11-23
First Posted2022-11-25

Study Record Updates

Last Update Submitted that Met QC Criteria2024-07-11
Last Update Posted2024-07-12
Last Verified2024-07