The primary purpose of this proof-of-concept clinical study was to evaluate the efficacy and safety of the study drug, ACH-0144471 (also known as danicopan and ALXN2040), in participants with C3G who also had significant proteinuria attributable to C3G.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
13
Clinical Study Site
Aurora, Colorado, United States
Clinical Study Site
Lawrenceville, Georgia, United States
Clinical Study Site
Iowa City, Iowa, United States
Clinical Study Site
Baltimore, Maryland, United States
Change From Baseline In Composite Biopsy Score At Week 28
The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of 6 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
Time frame: Baseline, Week 28
Participants With Reduction In Proteinuria At Week 28
Proteinuria reduction was defined as ≥ 30% decrease from baseline based on 24-hour urine protein (mg/day).
Time frame: Week 28
Change From Baseline In Proteinuria At Week 28
Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28.
Time frame: Baseline, Week 28
Percent Change From Baseline In Proteinuria At Week 28
Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28.
Time frame: Baseline, Week 28
Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the 6-month blinded treatment period, with eGFR as the dependent variable and time as the independent variable.
Time frame: 6 months
Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values during the open-label extension period with eGFR as the dependent variable and time as the independent variable.
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Clinical Study Site
New York, New York, United States
Clinical Study Site
New York, New York, United States
Clinical Study Site
London, United Kingdom
Time frame: 12 months
Change From Baseline In eGFR At Week 28
Change from baseline in eGFR at Week 28 is presented.
Time frame: Baseline, Week 28
Participants With Significant Improvement In eGFR Relative To Baseline At Week 28
Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR.
Time frame: Baseline, Week 28
Participants With Significant Improvement In eGFR Relative To Baseline At Week 52
Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR.
Time frame: Baseline, Week 52