Study of a Single Dose of Danicopan in Healthy Participants

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT04889677

Collaborator

Achillion, a wholly owned subsidiary of Alexion (Industry)

Enrollment

Location

Arms

4.5

Actual Duration (Months)

9.7

Patients Per Site Per Month

Study Details

Study Description

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.

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

Study Design

Study Type:

Interventional

Actual Enrollment :

44 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Basic Science

Official Title:

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

Actual Study Start Date :

Feb 4, 2016

Actual Primary Completion Date :

Jun 21, 2016

Actual Study Completion Date :

Jun 21, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Group 1: 200 mg All participants (fasted) received either 200 mg of danicopan as a single oral dose or dose-matched placebo.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Group 2: 600 mg All participants (fasted) received either 600 mg of danicopan as a single oral dose or dose-matched placebo.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Group 3: 1200 mg All participants (fasted) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Group 4: 2400 mg All participants (fasted) received either 2400 mg of danicopan administered as 2 single doses of 1200 mg each, 12 hours apart, or dose-matched placebo.	Drug: Danicopan Other Names: ACH-0144471 (formerly) ALXN2040 ACH-4471 ACH4471 4471 Drug: Placebo
Experimental: Group 5: 1200 mg All participants (fed) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.	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 28]

Secondary Outcome Measures

Area Under The Plasma Concentration-time Curve Extrapolated To Infinity (AUC0-inf) Of Danicopan Under Both Fed And Fasted Conditions [Up to 144 hours postdose]
Maximal Plasma Concentration (Cmax) Of Danicopan Under Both Fed And Fasted Conditions [Up to 144 hours postdose]
Time To Reach The Maximal Plasma Concentration (Tmax) Of Danicopan Under Both Fed And Fasted Conditions [Up to 144 hours postdose]
Relationship Between Alternative Pathway Inhibition And Danicopan Plasma Concentrations [Up to 144 hours postdose]

Eligibility Criteria

Criteria

Ages Eligible for Study:

21 Years to 55 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

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.