A phase III trial of RO5072759 in childhood onset idiopathic nephrotic syndrome patients

JRCT ID: jRCT2011220036

Registered date:22/01/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Childhood Nephrotic Syndrome
• Date of first enrollment: 29/03/2023
• Target sample size: 80
• Countries of recruitment: Belgium,Japan,Brazil,Japan,China,Japan,France,Japan,Germany,Japan,Italy,Japan,Poland,Japan,Spain,Japan,Turkey,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Obinutuzumab: Participants in Obinutuzumab (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).
Mycophenolate Mofetil: MMF (Group B) will be titrated by Week 4 to a target dose of 1200 mg/m2/day (maximum 2 g/day), given in two divided doses (600 mg/m2 BID).

Outcome(s)

Primary Outcome

Efficacy
Percentage of Participants with Sustained Complete Remission at 1 year

Secondary Outcome

Safety, Efficacy, Phamacokinetics, Phamacodynamics
-Overall Relapse Free Survival (RFS) [Time Frame: At Week 52]
-Probability of RFS at Week 52 [Time Frame: At Week 52]
-Cumulative corticosteroid dose (prednisone or equivalent adjusted for time on study)[Time Frame: At Week 52]
-Number of relapses on randomized study treatment[Time Frame: At Week 52]

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: <= 25age old
• Gender: Both

Include criteria

-Diagnosis of FRNS or 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
-Estimated glomerular filtration rate (eGFR) within normal range for age

Exclude 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
-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

Related Information

• Primary Sponsor: Will Pendergraft, M.D., Ph.D.
• Secondary ID(s): NCT05627557