A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of the NOX1/4 Inhibitor Setanaxib in Patients with Alport Syndrome

Conditions

Alport syndrome

Brief summary

Percentage of patients with SAEs; and Percentage of patients with treatment-emergent AESIs, ie, anemia.

Detailed description

Percentage of patients with clinically significant changes in heart rate and blood pressure., Percentage of patients with clinically significant changes in 12-lead ECG., Percentage of patients with clinically significant changes in physical examination., Percentage of patients with clinically significant changes in hematology, serum chemistry, urinalysis, and thyroid function., Percentage of patients with clinically significant changes in hearing audiometric testing (bone- and air-conduction)., The ratio of UPCR at 24 weeks compared to baseline; and Percentage of patients with a 25% reduction in UPCR at 24 weeks, compared to baseline., The ratio of eGFR at 24 weeks compared to baseline., Pre-dose and post-dose plasma concentrations of setanaxib and GKT138184 at steady state. The following PK properties will be calculated: Area under the concentration-time curve over 24 hours at steady state (AUC0-24-ss); Minimum plasma concentration at steady state (Cmin-ss); and Maximum plasma concentration at steady state (Cmax-ss).

Related papers

No related papers found yet.

Interventions

DRUGSetanaxib

DRUGPlacebo tablets

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Percentage of patients with SAEs; and Percentage of patients with treatment-emergent AESIs, ie, anemia.	—

Secondary

Measure	Time frame
Percentage of patients with clinically significant changes in heart rate and blood pressure., Percentage of patients with clinically significant changes in 12-lead ECG., Percentage of patients with clinically significant changes in physical examination., Percentage of patients with clinically significant changes in hematology, serum chemistry, urinalysis, and thyroid function., Percentage of patients with clinically significant changes in hearing audiometric testing (bone- and air-conduction)., The ratio of UPCR at 24 weeks compared to baseline; and Percentage of patients with a 25% reduction in UPCR at 24 weeks, compared to baseline., The ratio of eGFR at 24 weeks compared to baseline., Pre-dose and post-dose plasma concentrations of setanaxib and GKT138184 at steady state. The following PK properties will be calculated: Area under the concentration-time curve over 24 hours at steady state (AUC0-24-ss); Minimum plasma concentration at steady state (Cmin-ss); and Maximum plasma conc	—

Measure

Time frame

Percentage of patients with clinically significant changes in heart rate and blood pressure., Percentage of patients with clinically significant changes in 12-lead ECG., Percentage of patients with clinically significant changes in physical examination., Percentage of patients with clinically significant changes in hematology, serum chemistry, urinalysis, and thyroid function., Percentage of patients with clinically significant changes in hearing audiometric testing (bone- and air-conduction)., The ratio of UPCR at 24 weeks compared to baseline; and Percentage of patients with a 25% reduction in UPCR at 24 weeks, compared to baseline., The ratio of eGFR at 24 weeks compared to baseline., Pre-dose and post-dose plasma concentrations of setanaxib and GKT138184 at steady state. The following PK properties will be calculated: Area under the concentration-time curve over 24 hours at steady state (AUC0-24-ss); Minimum plasma concentration at steady state (Cmin-ss); and Maximum plasma conc

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Countries

Austria, Czechia, France, Lithuania, Slovakia, Spain

Outcome results

None listed