Study of Multiple Doses of Danicopan in Healthy Participants

Alexion Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Healthy

Treatments: Drug: Danicopan; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04889690

ACH471-002

Details and patient eligibility

About

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.

Enrollment: 45 patients

Sex: All

Ages: 25 to 55 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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45 participants in 4 patient groups

Cohort 1: 200 mg

Experimental group

Description:

All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period.

Treatment:

Drug: Placebo

Drug: Danicopan

Cohort 2: 500 mg

Experimental group

Description:

All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.

Treatment:

Drug: Placebo

Drug: Danicopan

Cohort 3: 800 mg

Experimental group

Description:

All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.

Treatment:

Drug: Placebo

Drug: Danicopan

Cohort 4: 75 mg

Experimental group

Description:

All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period.

Treatment:

Drug: Placebo

Drug: Danicopan