Study of Multiple Doses of Danicopan in Healthy Participants

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT04889690

Collaborator

Achillion, a wholly owned subsidiary of Alexion (Industry)

Enrollment

Location

Arms

7.7

Actual Duration (Months)

5.8

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Healthy	Drug: Danicopan Drug: Placebo	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

45 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Basic Science

Official Title:

A Multiple Ascending Dose Study To Assess Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of ACH-0144471 In Healthy Volunteers

Actual Study Start Date :

May 21, 2016

Actual Primary Completion Date :

Jan 11, 2017

Actual Study Completion Date :

Jan 11, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1: 200 mg All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Cohort 2: 500 mg All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Cohort 3: 800 mg All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Cohort 4: 75 mg All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo

Outcome Measures

Primary Outcome Measures

Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities [Day 1 through Day 42]

Secondary Outcome Measures

Maximum Observed Plasma Concentration (Cmax) Of Danicopan [Up to 16 hours postdose]
Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan [Up to 16 hours postdose]
Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Danicopan [Up to 16 hours postdose]
Activity Of Danicopan As Measured By Alternative Pathway (AP) Wieslab Assay [Up to 16 hours postdose]
Relationship Between AP Inhibition And Danicopan Plasma Concentrations [Up to 16 hours postdose]

Eligibility Criteria

Criteria

Ages Eligible for Study:

25 Years to 55 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

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.