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MEmbranous Nephropathy Trial Of Rituximab

Phase 2
Completed
Conditions
Idiopathic Membranous Nephropathy
Interventions
Registration Number
NCT01180036
Lead Sponsor
Mayo Clinic
Brief Summary

The primary outcome of this study is to determine whether or not the B cell targeting with Rituximab is non-inferior or more effective than Cyclosporine in inducing long term remission of proteinuria.

Detailed Description

In IMN, experimental data suggests that B cells are involved in the pathogenesis of the disease. To date, the best proven therapy for patients with MN consists of the combined use of corticosteroids and cyclophosphamide (CYC). Since the mechanism of action of CYC includes suppression of various stages of the B cell cycle including B cell activation, proliferation, and differentiation and inhibition of immunoglobulin (IgG) secretion, it lends credence to the hypothesis that B cells abnormalities are involved in the pathogenesis of MN. Given the key role of IgG antibodies in MN, it is reasonable to postulate that suppression of antibody production by depleting B cells may improve or even resolve the glomerular pathology and be reflected by a reduction in proteinuria. Thus, a case could be made for using an agent capable of selectively depleting B cells, and therefore halting the production of immunoglobulins against antigens potentially present in the glomeruli. This approach could stop the initiating sequence of pathogenic events and result in resolution of the. The P.I. believes that the application of selective B cell targeting with Rituximab (RTX) will prove at least equal, or even superior, both in the production of short term and long term control of the nephrotic syndrome (NS) and be safer than any current therapeutic regimen used to treat MN.

Based on this rationale, the investigators conducted a pilot trial in 15 newly-biopsied patients (\<3 years) with IMN and proteinuria \>5g/24h despite ACEi/ARB use for \>3months and systolic BP \<130 millimeter of mercury (mmHg). Mean baseline creatinine was 1.4 mg/dl. Thirteen males and 2 females, median age 47 (range 33-63), were treated with RTX (1g) on days 1 and 15. At six months, patients who remained with proteinuria \>3g/24 received a second identical course of RTX. Baseline proteinuria of 13.0±5.7g/24h (range 8.4-23.5) decreased to 6.0±7.0 g/24h (range 0.2-20) at 12 months (mean ± SD). In the fourteen patients who completed a 12 months follow-up complete remission (proteinuria \<0.3g/24h) was achieved in 2 patients and partial remission (\<3g/24h) in 7 patients. In 5 of these 7 patients, proteinuria was \<1.5g/24h and follow up at 18 months showed that 3 of these 7 patients on PR achieved CR of proteinuria. Five patients did not respond. The mean drop in proteinuria from baseline to 12 months was 6.2± 5.1g (p=.002, paired t-test). There were a limited number of minor side-effects. Initial cluster of differentiation 20 (CD20)+ B cell depletion was seen in all patients. However, at 3 months, CD20+ B cells were starting to recover with five patients \>35 cells/µl (range 35-152).(50) These data contrasts with previous work by Ruggenenti et al. using RTX given weekly (375 mg/m2) for 4 weeks. Pharmacokinetic (PK) analysis showed that RTX levels in this 2-dose regimen were 50% lower compared to non-proteinuric patients, which could potentially result in undertreatment.

Based on these results, the investigators recently conducted a study postulating that in patients with MN, 4 weekly doses of RTX would result in more effective B cell depletion, a higher remission rate and maintaining of the same safety profile compared to patients treated with RTX dosed at 1g x 2.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
130
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Rituximab Treatment ArmRituximabPatients randomized to the RTX arm will receive 1000 mg IV on Days 1 and 15. Patients who achieve complete remission at 6 months will not be retreated. A second course of RTX 1000 mg IV will be administered at study month 6 for individuals who have not achieved a complete remission, but have achieved a minimum of \>25% reduction in Time 0 proteinuria. Dosing at study month 6 will be independent of cluster of differentiation (CD) 19+ B cell count.
Cyclosporine Treatment ArmCyclosporinePatients randomized to the Cyclosporine arm will be started at a dose of CsA = 3.5 mg/kg/day p.o. divided into 2 equal doses given at 12 hour intervals. Target trough CsA blood levels are 125 to 175 ng/ml. Patients will have their doses adjusted according to their blood levels of CSA as monitored every 2 weeks until the target trough level is reached. If a complete remission is achieved by 6 months, CSA will be tapered and discontinued over a three-month period. If after 6 months there has not been a reduction in proteinuria of at least 25% of baseline values, the drug will also be discontinued. If there has been a \>25% reduction in baseline proteinuria (but not complete remission) the CSA will be continued for an additional 6 months.
Primary Outcome Measures
NameTimeMethod
Remission Status24 months after randomization

The number of subjects to reach the composite of maintaining complete remission or partial remission at 24 months after randomization will be the primary endpoint.

Secondary Outcome Measures
NameTimeMethod
Remission Status12 months after randomization

The number of subjects to reach either complete remission or partial remission at 12 months after randomization.

Trial Locations

Locations (21)

Toronto General Hospital

🇨🇦

Toronto, Ontario, Canada

Ohio State University

🇺🇸

Columbus, Ohio, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Mayo Clinic Jacksonville

🇺🇸

Jacksonville, Florida, United States

Mayo Clinic Scottsdale

🇺🇸

Scottsdale, Arizona, United States

MetroHealth System (Case Western Reserve University)

🇺🇸

Cleveland, Ohio, United States

University of Arizona, Tucson

🇺🇸

Tucson, Arizona, United States

Mayo Clinic Rochester

🇺🇸

Rochester, Minnesota, United States

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

University of Miami Hospital and Clinics

🇺🇸

Miami, Florida, United States

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

New York University

🇺🇸

New York, New York, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

University of Washington Medical Center

🇺🇸

Seattle, Washington, United States

Medical College of Wisconsin, Froedtert Hospital

🇺🇸

Milwaukee, Wisconsin, United States

Centre hospitalier universitaire de Quebec - Hotel-Dieu de Quebec

🇨🇦

Quebec, Canada

St. Paul's Hospital, Providence Health Care

🇨🇦

Vancouver, British Columbia, Canada

Stanford University

🇺🇸

San Francisco, California, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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