Clinical trial NCT04889690: Study of Multiple Doses of Danicopan in Healthy Participants
Official title: A Multiple Ascending Dose Study To Assess Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of ACH-0144471 In Healthy Volunteers
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy
Keywords: Danicopan
ALXN2040
ACH-0144471
Factor D Inhibitor
Pharmacokinetics
Pharmacodynamics
Ascending Dose
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Achillion, a wholly owned subsidiary of Alexion
First posted: 2021-05-17
Enrollment: 45
Sex: ALL
Minimum age (years): 25
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

Brief summary: This was a multiple ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants. Four different doses (75 milligrams \[mg\], 200 mg, 500 mg, 800 mg) and dose-matched placebo were administered under fasted conditions.


Eligibility criteria: Inclusion Criteria:

* Healthy was defined as having no clinically relevant abnormalities identified by a detailed medical history, physical exam, blood pressure and heart rate measurements, 12-lead electrocardiogram, and clinical laboratory tests.
* Body mass index of 18 to 30 kilograms (kg)/meter squared with a minimum body weight of 50 kg.

Exclusion Criteria:

* History or clinically relevant evidence of significant cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disease.
* Any condition possibly affecting drug absorption (including gastrectomy and cholecystectomy).
* Body temperature greater than or equal to 38°Celcius on Day -1 or Day 1, Hour 0; history of febrile illness or other evidence of infection within 14 days prior to first study drug administration.
* Current tobacco/nicotine user; consumption of any alcohol within 72 hours before first study drug administration or have a history of regular alcohol consumption exceeding 21 drinks/week within 6 months of screening; positive urine drug screen at screening or Day -1.
* Clinically significant laboratory abnormalities at either Screening or Day -1.

Primary outcomes:
measure: Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities
timeFrame: Day 1 through Day 42
Secondary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax) Of Danicopan
timeFrame: Up to 16 hours postdose
measure: Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan
timeFrame: Up to 16 hours postdose
measure: Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Danicopan
timeFrame: Up to 16 hours postdose
measure: Activity Of Danicopan As Measured By Alternative Pathway (AP) Wieslab Assay
timeFrame: Up to 16 hours postdose
measure: Relationship Between AP Inhibition And Danicopan Plasma Concentrations
timeFrame: Up to 16 hours postdose
Other outcomes:
Locations:
city: Auckland
country: New Zealand
facility: Clinical Trial Site
Study officials:
References: