To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH. Danicopan dose may be increased within each participant, to a maximum of 200 milligrams (mg) three times daily (TID) based on safety and efficacy at protocol-specified time points.
The purpose of this study is to determine the effectiveness of danicopan in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a drug commonly used for treatment of PNH) for 24 weeks in participants with PNH. The 24-week treatment period was followed by a long-term extension phase. In the extension phase, participants received the same danicopan dose plus eculizumab as they were receiving at the end of 24-week treatment phase. Results are reported for the 24-week treatment period.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Participants received a daily oral dose of danicopan TID during the treatment period.
Participants received intravenous eculizumab administered at the participant's usual dose and schedule.
Clinical Study Site
Baltimore, Maryland, United States
Clinical Study Site
Cleveland, Ohio, United States
Clinical Study Site
Florence, Italy
Clinical Study Site
Naples, Italy
Clinical Study Site
Change From Baseline In Hemoglobin At Week 24
Time frame: Baseline, Week 24
Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment
Time frame: Within 24 weeks prior to first dose and during 24-week treatment period
Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment
Time frame: Within 24 weeks prior to first dose and during 24-week treatment period
Change From Baseline In Lactate Dehydrogenase At Week 24
Time frame: Baseline, Week 24
Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Time frame: Day 1 (after dosing) through end of study (maximum exposure: 1631 days)
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