Study of a Single Dose of Danicopan in Healthy Participants

Phase 1

Completed

• Conditions: Healthy
• Interventions: Drug: Danicopan; Drug: Placebo

• Registration Number: NCT04889677
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-02-04
• Primary Completion: 2016-06-21
• Study Completion: 2016-06-21
• Status Verified: 2021-05
• Study First Submitted: 2021-05-12
• Study First Submitted QC: 2021-05-12
• Last Update Submitted: 2021-05-12
• Study First Posted: 2021-05-17
• Last Update Posted: 2021-05-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 44

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group 1: 200 mg	Placebo	All participants (fasted) received either 200 mg of danicopan as a single oral dose or dose-matched placebo.
Group 2: 600 mg	Placebo	All participants (fasted) received either 600 mg of danicopan as a single oral dose or dose-matched placebo.
Group 3: 1200 mg	Placebo	All participants (fasted) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.
Group 4: 2400 mg	Placebo	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.
Group 5: 1200 mg	Placebo	All participants (fed) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.
Group 1: 200 mg	Danicopan	All participants (fasted) received either 200 mg of danicopan as a single oral dose or dose-matched placebo.
Group 2: 600 mg	Danicopan	All participants (fasted) received either 600 mg of danicopan as a single oral dose or dose-matched placebo.
Group 3: 1200 mg	Danicopan	All participants (fasted) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.
Group 4: 2400 mg	Danicopan	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.
Group 5: 1200 mg	Danicopan	All participants (fed) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Time To Reach The Maximal Plasma Concentration (Tmax) Of Danicopan Under Both Fed And Fasted Conditions	Up to 144 hours postdose
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
Relationship Between Alternative Pathway Inhibition And Danicopan Plasma Concentrations	Up to 144 hours postdose

Trial Locations

Locations (1)

Clinical Trial Site; 🇳🇿 Auckland, New Zealand