Study of a Single Dose of Danicopan in Healthy Participants

Study Overview
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Brief Summary
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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.

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

Conditions
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Healthy

Intervention / Treatment
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* Drug: Danicopan
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 21 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group 1: 200 mg<br>All participants (fasted) received either 200 mg of danicopan as a single oral dose or dose-matched placebo. | Drug: Danicopan<br> <br> * Other names: 4471;Drug: Placebo<br> <br> |
| Experimental: Group 2: 600 mg<br>All participants (fasted) received either 600 mg of danicopan as a single oral dose or dose-matched placebo. | Drug: Danicopan<br> <br> * Other names: 4471;Drug: Placebo<br> <br> |
| Experimental: Group 3: 1200 mg<br>All participants (fasted) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo. | Drug: Danicopan<br> <br> * Other names: 4471;Drug: Placebo<br> <br> |
| Experimental: Group 4: 2400 mg<br>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<br> <br> * Other names: 4471;Drug: Placebo<br> <br> |
| Experimental: Group 5: 1200 mg<br>All participants (fed) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo. | Drug: Danicopan<br> <br> * Other names: 4471;Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Danicopan, ALXN2040, ACH-0144471, Factor D Inhibitor, Pharmacokinetics, Pharmacodynamics, Ascending Dose