Study of Multiple Doses of Danicopan in Healthy Participants

Phase 1

Completed

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

• Registration Number: NCT04889690
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-05-21
• Primary Completion: 2017-01-11
• Study Completion: 2017-01-11
• 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: 45

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 2: 500 mg	Danicopan	All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.
Cohort 3: 800 mg	Danicopan	All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.
Cohort 4: 75 mg	Danicopan	All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period.
Cohort 1: 200 mg	Placebo	All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period.
Cohort 2: 500 mg	Placebo	All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.
Cohort 3: 800 mg	Placebo	All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.
Cohort 4: 75 mg	Placebo	All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period.
Cohort 1: 200 mg	Danicopan	All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period.

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 42

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Clinical Trial Site; 🇳🇿 Auckland, New Zealand