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CompletedNCT04889677Updated May 17, 2021

Study of a Single Dose of Danicopan in Healthy Participants

A Phase 1 interventional study of Danicopan and Placebo in Healthy, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 1 site in New Zealand. Open to participants aged 21 Years to 55 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2021-05-17.

Sponsored by Alexion Pharmaceuticals, Inc. · Phase 1, Interventional, and Basic science

From the registry’s dates

  • Registered 5 years 3 months after the study started (first participant enrolled Feb 2016, registered May 2021).
Phase
Phase 1
Study type
Interventional
Enrollment
44
Allocation
Randomized
Ages
21 Years to 55 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Healthy

Keywords

  • Danicopan
  • ALXN2040
  • ACH-0144471
  • Factor D Inhibitor
  • Pharmacokinetics
  • Pharmacodynamics
  • Ascending Dose
03

In context

Lead sponsor

Alexion Pharmaceuticals, Inc. is the lead sponsor of 249 studies on the registry; 25 are open to participants now.

Of its 98 completed or terminated interventional studies of FDA-regulated products, 70 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
21 Years to 55 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

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

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.
05

Study design

Phase
Phase 1
Primary purpose
Basic science
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
44 participants (actual)

Study arms

  • Experimental
    Group 1: 200 mg

    All participants (fasted) received either 200 mg of danicopan as a single oral dose or dose-matched placebo.

    Drug: Danicopan · Drug: Placebo

  • Experimental
    Group 2: 600 mg

    All participants (fasted) received either 600 mg of danicopan as a single oral dose or dose-matched placebo.

    Drug: Danicopan · Drug: Placebo

  • Experimental
    Group 3: 1200 mg

    All participants (fasted) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.

    Drug: Danicopan · Drug: Placebo

  • Experimental
    Group 4: 2400 mg

    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 · Drug: Placebo

  • Experimental
    Group 5: 1200 mg

    All participants (fed) received either 1200 mg of danicopan as a single oral dose or dose-matched placebo.

    Drug: Danicopan · Drug: Placebo

Interventions

  • DrugDanicopan

    Also known as: ACH-0144471 (formerly), ALXN2040, ACH-4471, ACH4471, 4471

  • DrugPlacebo
06

What researchers measure

Primary outcomes

  1. Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities

    Time frame: Day 1 through Day 28

Secondary outcomes

  1. Area Under The Plasma Concentration-time Curve Extrapolated To Infinity (AUC0-inf) Of Danicopan Under Both Fed And Fasted Conditions

    Time frame: Up to 144 hours postdose

  2. Maximal Plasma Concentration (Cmax) Of Danicopan Under Both Fed And Fasted Conditions

    Time frame: Up to 144 hours postdose

  3. Time To Reach The Maximal Plasma Concentration (Tmax) Of Danicopan Under Both Fed And Fasted Conditions

    Time frame: Up to 144 hours postdose

  4. Relationship Between Alternative Pathway Inhibition And Danicopan Plasma Concentrations

    Time frame: Up to 144 hours postdose

07

Study locations

1 site
  • Clinical Trial Site
    Auckland, New Zealand
08

References and documents

Publications

  • Wiles JA, Galvan MD, Podos SD, Geffner M, Huang M. Discovery and Development of the Oral Complement Factor D Inhibitor Danicopan (ACH-4471). Curr Med Chem. 2020;27(25):4165-4180. doi: 10.2174/0929867326666191001130342. PubMed 31573880 ↗

Individual participant data

Plan to share: No

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 17, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04889677
Lead sponsor
Alexion Pharmaceuticals, Inc.
Collaborators
Achillion, a wholly owned subsidiary of Alexion
Responsible party
Sponsor
First posted
May 17, 2021
Start date
Feb 4, 2016
Primary completion
Jun 21, 2016
Completion
Jun 21, 2016
Last update
May 17, 2021

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in May 2021. You cannot join it, but the record below documents what was studied.

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