Study of Multiple Doses of Danicopan in Healthy Participants

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

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.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: Placebo; Drug: Danicopan

• Study Type: Interventional
• Enrollment (Actual): 45
• Phase: Phase 1

Contacts and Locations

Study Locations

• New Zealand
  • Auckland, New Zealand
    • Clinical Trial Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 25 years to 55 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: BASIC_SCIENCE
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Cohort 1: 200 mg; All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period.	Drug: Placebo; Drug: Danicopan; Other Names: ALXN2040; ACH-0144471 (formerly); ACH-4471; ACH4471; 4471
EXPERIMENTAL: Cohort 2: 500 mg; All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.	Drug: Placebo; Drug: Danicopan; Other Names: ALXN2040; ACH-0144471 (formerly); ACH-4471; ACH4471; 4471
EXPERIMENTAL: Cohort 3: 800 mg; All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.	Drug: Placebo; Drug: Danicopan; Other Names: ALXN2040; ACH-0144471 (formerly); ACH-4471; ACH4471; 4471
EXPERIMENTAL: Cohort 4: 75 mg; All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period.	Drug: Placebo; Drug: Danicopan; Other Names: ALXN2040; ACH-0144471 (formerly); ACH-4471; ACH4471; 4471

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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 42

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals
• Collaborators: Achillion, a wholly owned subsidiary of Alexion

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 21, 2016
• Primary Completion (ACTUAL): January 11, 2017
• Study Completion (ACTUAL): January 11, 2017

Study Registration Dates

• First Submitted: May 12, 2021
• First Submitted That Met QC Criteria: May 12, 2021
• First Posted (ACTUAL): May 17, 2021

Study Record Updates

• Last Update Posted (ACTUAL): May 17, 2021
• Last Update Submitted That Met QC Criteria: May 12, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Keywords: Pharmacokinetics; Pharmacodynamics; Danicopan; ALXN2040; ACH-0144471; Factor D Inhibitor; Ascending Dose
• Other Study ID Numbers: ACH471-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No