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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04889690




Registration number
NCT04889690
Ethics application status
Date submitted
12/05/2021
Date registered
17/05/2021
Date last updated
17/05/2021

Titles & IDs
Public title
Study of Multiple Doses of Danicopan in Healthy Participants
Scientific title
A Multiple Ascending Dose Study To Assess Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of ACH-0144471 In Healthy Volunteers
Secondary ID [1] 0 0
ACH471-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Healthy 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Danicopan
Treatment: Drugs - Placebo

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.

Experimental: Cohort 2: 500 mg - All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.

Experimental: Cohort 3: 800 mg - All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.

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.


Treatment: Drugs: Danicopan


Treatment: Drugs: Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities
Timepoint [1] 0 0
Day 1 through Day 42
Secondary outcome [1] 0 0
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Timepoint [1] 0 0
Up to 16 hours postdose
Secondary outcome [2] 0 0
Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Timepoint [2] 0 0
Up to 16 hours postdose
Secondary outcome [3] 0 0
Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Danicopan
Timepoint [3] 0 0
Up to 16 hours postdose
Secondary outcome [4] 0 0
Activity Of Danicopan As Measured By Alternative Pathway (AP) Wieslab Assay
Timepoint [4] 0 0
Up to 16 hours postdose
Secondary outcome [5] 0 0
Relationship Between AP Inhibition And Danicopan Plasma Concentrations
Timepoint [5] 0 0
Up to 16 hours postdose

Eligibility
Key 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.
Minimum age
25 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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
Purpose of the study
Basic Science
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/05/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
11/01/2017
Sample size
Target
Accrual to date
Final
45
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Achillion, a wholly owned subsidiary of Alexion
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04889690
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries