The primary purpose of this study was to evaluate the efficacy of 12 months of oral ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G or IC-MPGN based on histologic scoring and proteinuria.
This was an open-label study to evaluate the efficacy of treatment with danicopan in participants 12 years of age or older with biopsy-confirmed C3G or IC-MPGN who had not undergone renal transplantation. All participants were to receive active treatment with danicopan for approximately 40 months. The starting dosage was to be 100 mg TID, and after 2 weeks, the dosage was to be increased to 200 mg TID for participants with body weight ≥ 60 kg or 150 mg TID for participants with body weight \< 60 kg. Planned enrollment was approximately 20 participants.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
Danicopan was to be administered as an oral tablet.
Clinical Study Site
Birmingham, Alabama, United States
Clinical Study Site
Stanford, California, United States
Clinical Study Site
New Haven, Connecticut, United States
Change From Baseline In Composite Biopsy Score At End Of Initial 12-Month Treatment Period
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 the initial 12 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
Time frame: Baseline, end of initial 12-Month Treatment Period
Participants With Reduction In Proteinuria At End Of Initial 12-Month Treatment Period
Proteinuria reduction was defined as ≥30% decrease from baseline based on 24-hour urine protein (mg/day).
Time frame: Baseline, end of initial 12-Month Treatment Period
Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
Proteinuria was assessed based on 24-hour urine collections at baseline and end of the initial 12-month Treatment Period.
Time frame: Baseline, end of initial 12-Month Treatment Period
Percent Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
Proteinuria was assessed based on 24-hour urine collections at baseline and end of initial 12-month Treatment Period.
Time frame: Baseline, end of initial 12-Month Treatment Period
Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To End Of Initial 12-Month Treatment Period
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the Initial 12-Month Treatment Period, with eGFR as the dependent variable and time as the independent variable.
Time frame: End of initial 12-Month Treatment Period
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Clinical Study Site
Cincinnati, Ohio, United States
Clinical Study Site
Columbus, Ohio, United States
Clinical Study Site
Philadelphia, Pennsylvania, United States
Clinical Study Site
Sydney, New South Wales, Australia
Clinical Study Site
Brisbane, Queensland, Australia
Clinical Study Site
Melbourne, Victoria, Australia
Clinical Study Site
Antwerp, Belgium
...and 3 more locations
Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period
Change from baseline in eGFR at end of initial 12-Month Treatment Period is presented.
Time frame: Baseline, end of initial 12-Month Treatment Period
Participants With Significant Improvement In eGFR Relative To Baseline At End Of Initial 12-Month Treatment Period
Significant improvement relative to baseline was defined as a ≥ 25% increase from baseline in eGFR.
Time frame: Baseline, end of initial 12-Month Treatment Period
Change From Baseline in eGFR Over 12 Months of Treatment For Participants Meeting eGFR Inclusion Criteria
Participants were eligible for enrollment if inclusion criteria were met including having an eGFR \>=30 milliliters (mL)/minute (min)/1.73 square meter (m\^2) at the time of screening or at any time over the preceding 4 weeks. This Outcome Measure was registered in case there were participants who were enrolled and ended up not meeting the Eligibility Criteria and was intended to report data for change from baseline in eGFR for only the participants who met the eligibility criteria (that is, participants who did not meet the eligibility criteria would have been excluded from analysis for this Outcome Measure). Since all enrolled participants met the Eligibility Criteria, none of the participants were excluded from this analysis. Therefore, this data is the same data that is presented in Outcome Measure #6 "Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period". Change from baseline in eGFR at end of initial 12-Month Treatment Period is presented.
Time frame: End of initial 12-Month Treatment Period
Change From Baseline In Measured GFR At The End Of The Initial 12-Month Treatment Period
Data for this Outcome Measure was to be collected where available. None of the sites collected data for this Outcome Measure.
Time frame: End of initial 12-Month Treatment Period