Active Studies

Matthew Weir

This Phase 3, randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of mezagitamab in adults with primary IgAN receiving stable background therapy. The study assesses the impact of mezagitamab on proteinuria reduction, measured by UPCR, and its ability to maintain kidney function over time. Participants are randomized 2:1 to receive either mezagitamab or placebo in two 1-year cycles, with an additional open-label cohort for patients with lower eGFR or prior mezagitamab exposure. Secondary endpoints include safety, tolerability, and long-term changes in eGFR to determine the drug's potential in mitigating immune complex-mediated kidney damage.

Matthew Weir

This Phase 2a, multicenter, randomized, double-blind, placebo-controlled umbrella study evaluates the efficacy and safety of three investigational agents—frexalimab, brivekimig, and rilzabrutinib—in patients aged 16 to 75 with primary FSGS or MCD. The primary objective is to assess changes in proteinuria and nephrotic syndrome remission rates over a 24-week treatment period. Participants are randomized across six treatment arms to compare these novel therapies against placebo. Key clinical outcomes focus on reductions in UPCR and the achievement of complete or partial clinical remission. The total study duration lasts up to 76 weeks, including a long-term follow-up phase to monitor safety and durability of response.

Susan Huang

This Phase III, multicenter, randomized, double-blind trial evaluates the efficacy and safety of sefaxersen (RO7434656) in patients with primary IgA nephropathy at high risk of disease progression. The study population includes participants who demonstrate persistent risk despite receiving optimized supportive care. Sefaxersen is a novel antisense oligonucleotide designed to inhibit the production of complement factor B, targeting the alternative complement pathway. Investigators assess the drug’s impact on proteinuria, typically measured by UPCR or UACR, and its effect on stabilizing eGFR over time. The trial aims to determine if this targeted molecular approach reduces the risk of progressive kidney function decline compared to placebo.

Matthew Weir

This Phase 3, randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of ianalumab as an add-on to standard-of-care therapy in adults with active lupus nephritis. c. c. q12w, or placebo. The primary objective is to assess the impact of B-cell depletion via ianalumab on clinical response rates, typically measured by improvements in UPCR and stabilization of eGFR.

Matthew Weir

This Phase 3, multicenter, randomized, double-blind trial evaluates the efficacy and safety of iptacopan, an oral complement alternative pathway inhibitor, in adult and adolescent patients with native C3 glomerulopathy. Participants are randomized to receive either iptacopan or placebo in addition to standard of care to assess the drug's impact on proteinuria and renal function. The primary endpoints include the reduction in UPCR and stabilization of eGFR compared to placebo at the end of the treatment period. Secondary objectives utilize serum C3 levels and repeat kidney biopsies to evaluate the inhibition of the alternative pathway and associated histopathological improvements.

Susan Huang

This Phase 3, randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of ravulizumab in approximately 510 adults with IgAN at high risk of disease progression. Participants must be on a stable standard of care regimen for at least three months prior to screening and are randomized 1:1 to receive weight-based IV infusions of ravulizumab or placebo. The primary endpoints are the reduction in proteinuria at Week 34 and the change in eGFR at Week 106. The study includes a specific cohort for patients with advanced kidney disease (eGFR 20–29 mL/min/1.73m²) to assess outcomes in late-stage progression.

Susan Huang

This Phase 3b, open-label continuation study evaluates the long-term safety and efficacy of TAK-755 (rADAMTS13) in patients with severe congenital thrombotic thrombocytopenic purpura (cTTP). The study enrolls both treatment-naïve patients and those transitioning from previous TAK-755 trials to receive either prophylactic or on-demand enzyme replacement therapy. Participants in the prophylactic cohort receive weekly or biweekly intravenous infusions to prevent acute TTP events, while the on-demand cohort receives daily dosing for the management of acute flares. The primary objective is to characterize the long-term safety profile and the ability of rADAMTS13 to maintain ADAMTS13 activity levels and prevent thrombotic microangiopathy. Treatment duration extends up to three years, with options for home-based self-infusion or caregiver administration in the prophylactic arm.

Arsh Jain

This multicenter, randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of BI 764198, an oral TRPC6 inhibitor, in adults and adolescents with primary FSGS or genetic FSGS related to TRPC6 variants. Participants are randomized to receive either BI 764198 or placebo once daily for 104 weeks as an adjunct to standard of care. The study assesses the drug's impact on disease progression and kidney function through regular monitoring of UPCR and adverse events. The primary objective is to determine if TRPC6 inhibition reduces proteinuria and stabilizes renal function over a long-term treatment period.

Susan Huang

This Phase 3, open-label, randomized trial evaluates the efficacy and safety of felzartamab compared to tacrolimus in patients with primary membranous nephropathy (PMN). The study population consists of adults with PMN at risk of progressive kidney disease, characterized by persistent proteinuria and the presence of autoantibodies. The primary endpoint is the proportion of participants achieving a complete response, defined by a significant reduction in UPCR and stable eGFR, at 104 weeks. Secondary objectives include assessing the time to disease progression, duration of remission, safety profiles, and the impact of felzartamab on anti-PLA2R antibody titers. Participants receive either intravenous felzartamab or oral tacrolimus, with provisions for rescue therapy in cases of treatment failure or disease relapse.

Susan Huang

This Phase 2 study evaluates the safety, tolerability, and efficacy of atacicept in adult and adolescent patients aged 10 and older with autoimmune glomerular diseases. The target population includes patients diagnosed with IgAN, pMN, MCD, or FSGS. Participants receive weekly subcutaneous injections of atacicept to determine its impact on reducing UPCR and preserving renal function. Primary endpoints focus on the drug's safety profile and its ability to achieve clinical remission across these specific nephropathies.

Matthew Weir

This Phase III multicenter, randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of iptacopan, an oral proximal complement inhibitor, in adults and adolescents with idiopathic IC-MPGN. Participants receive either iptacopan or placebo in addition to standard of care to assess the drug's impact on alternative complement pathway dysregulation. The primary objectives are to demonstrate a reduction in proteinuria, measured by UPCR, and stabilization or improvement in eGFR. Secondary endpoints include changes in patient-reported fatigue and long-term safety profiles. Following the double-blind period, eligible participants may transition to an open-label extension study.

Matthew Weir

The BEYOND study evaluates the safety and efficacy of BION-1301, a humanized monoclonal antibody, in adults with biopsy-proven IgA nephropathy. Participants are randomized to receive 600 mg of BION-1301 or placebo via subcutaneous injection every two weeks for 104 weeks. The primary analysis population includes patients with an eGFR ≥ 30 mL/min/1.73m², while an exploratory cohort assesses patients with an eGFR between 20 and 30 mL/min/1.73m². The primary objective is to determine the impact of BION-1301 on proteinuria reduction compared to placebo. Secondary outcomes include long-term safety and the preservation of renal function over a 2.5-year study period.

Matthew Weir

The ALIGN study is a Phase 3, randomized, double-blind trial evaluating the efficacy and safety of atrasentan, a selective endothelin A receptor antagonist, in patients with biopsy-proven IgA nephropathy at risk of progressive renal function loss. Participants receive 0.75 mg of atrasentan or placebo daily for 132 weeks while maintained on a maximally tolerated, stable dose of a RAS inhibitor. The study population includes a primary cohort of approximately 320 patients and a separate stratum for those on stable SGLT2i therapy. The primary endpoint is the change in proteinuria as measured by UPCR, with secondary outcomes assessing kidney function via eGFR slope and overall safety.

Matthew Weir

This randomized, controlled trial evaluates the efficacy and safety of iptacopan, an oral factor B inhibitor, for inducing and maintaining remission in patients with newly diagnosed or relapsed active GPA or MPA. Participants receive iptacopan or placebo in combination with a rituximab induction regimen. The study assesses primary outcomes related to clinical remission and secondary endpoints including disease relapse rates, GC-related toxicity, and changes in eGFR and UPCR. The target population includes patients with active ANCA-associated vasculitis requiring induction therapy.

Andrew House

This Phase III, randomized, multicentre, double-blind study evaluates the efficacy, safety, and tolerability of a zibotentan/dapagliflozin combination compared to dapagliflozin monotherapy. The study population includes patients with CKD and high proteinuria, specifically targeting those who remain at risk despite standard care. The primary objective is to determine if the addition of zibotentan, a selective endothelin A receptor antagonist, to the SGLT2 inhibitor dapagliflozin further reduces albuminuria. Researchers monitor changes in UACR and eGFR as key indicators of renal function and treatment response.