Clinical trial NCT04889677: Study of a Single Dose of Danicopan in Healthy Participants
Official title: A Single Ascending Dose Study To Assess The 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: 44
Sex: ALL
Minimum age (years): 21
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 single ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants. Four different doses (200 milligrams \[mg\], 600 mg, 1200 mg, 2400 mg) and dose-matched placebo were administered under fasted and fed conditions.


Eligibility criteria: Inclusion Criteria:

* Healthy defined as having no clinically relevant abnormalities identified by a detailed medical history, physical examination, blood pressure and pulse 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 (for example, gastrectomy), cholecystectomy to be considered acceptable.
* Body temperature ≥ 38°Celcius on Day -1 or Day 1 Hour 0; history of febrile illness or other evidence of infection within 14 days prior to study drug administration.
* Current tobacco/nicotine users; consumption of any alcohol within 72 hours before study drug administration or 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 28
Secondary outcomes:
measure: Area Under The Plasma Concentration-time Curve Extrapolated To Infinity (AUC0-inf) Of Danicopan Under Both Fed And Fasted Conditions
timeFrame: Up to 144 hours postdose
measure: Maximal Plasma Concentration (Cmax) Of Danicopan Under Both Fed And Fasted Conditions
timeFrame: Up to 144 hours postdose
measure: Time To Reach The Maximal Plasma Concentration (Tmax) Of Danicopan Under Both Fed And Fasted Conditions
timeFrame: Up to 144 hours postdose
measure: Relationship Between Alternative Pathway Inhibition And Danicopan Plasma Concentrations
timeFrame: Up to 144 hours postdose
Other outcomes:
Locations:
city: Auckland
country: New Zealand
facility: Clinical Trial Site
Study officials:
References:
citation: Wiles JA, Galvan MD, Podos SD, Geffner M, Huang M. Discovery and Development of the Oral Complement Factor D Inhibitor Danicopan (ACH-4471). Curr Med Chem. 2020;27(25):4165-4180. doi: 10.2174/0929867326666191001130342.