Active Studies

Pavel Roshanov

This Phase 1a/1b randomized, double-blind, placebo-controlled trial evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of GSK4771261. Part A assesses single ascending doses in healthy participants to determine the drug's safety profile and systemic impact. Part B investigates the intervention in patients with autosomal dominant polycystic kidney disease (ADPKD) to establish initial clinical data in the target population. The study monitors standard safety parameters and physiological responses to inform future dosing strategies for chronic kidney disease management.

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.

Andrew House

The purpose of this study is the evaluate the clinical characteristics of adults with tuberous sclerosis complex and to provide multidisciplinary care.

Dervla Connaughton

This multi-center study evaluates the clinical and economic impact of genetic testing in patients with CKD who meet Ontario Health's Provincial Genetic Program criteria for suspected genetic kidney disease. Participants receive either genome-wide sequencing or standard genetic testing based on the timing of their initial diagnosis. The study assesses patient and caregiver quality of life, healthcare resource utilization via ICES data linkage, and the perceived clinical utility of testing as reported by referring physicians. Researchers also perform an economic analysis to compare genome-wide sequencing against standard testing protocols to determine the optimal timing and method for genomic assessment in CKD management.

Dervla Connaughton

The purpose of the study is to identify underlying genetic causes of kidney disease using advanced genetic analysis techniques such as whole exome and genome sequencing.

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.

Dervla Connaughton

This Phase 2a randomized, double-blind, placebo-controlled study evaluates the efficacy and safety of BAY 3401016 in adults aged 18 to 45 with rapidly progressing Alport Syndrome. The intervention is a monoclonal antibody that targets Semaphorin 3A (Sema3A), a protein implicated in the pathogenesis of glomerular damage and progressive renal decline. The primary objective is to determine if Sema3A inhibition reduces proteinuria and slows the loss of kidney function in this population. Investigators monitor changes in UACR and eGFR to assess the drug's impact on disease progression. The trial includes an initial treatment phase followed by an extension phase to evaluate long-term safety and clinical outcomes.