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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/NCT04451434




Registration number
NCT04451434
Ethics application status
Date submitted
19/06/2020
Date registered
30/06/2020
Date last updated
15/02/2021

Titles & IDs
Public title
Study of Danicopan in Participants of Japanese Descent
Scientific title
A Phase 1 Study to Determine the Safety, Tolerability, and Pharmacokinetics of Danicopan After Administration as an Oral Tablet in the Fed and Fasted States in Participants of Japanese Descent
Secondary ID [1] 0 0
ALXN2040-HV-101
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

Experimental: Danicopan 200 mg Fasted - Fasting participants will receive a single dose of 200 mg danicopan.

Experimental: Danicopan 200 mg Fed - Fed participants will receive a single dose of 200 mg danicopan.

Experimental: Danicopan 400 mg Fed - Fed participants will receive a single dose of 400 mg danicopan.


Treatment: Drugs: Danicopan
Oral tablet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number Of Participants With Treatment-Emergent Adverse Events
Timepoint [1] 0 0
Day 1 (after first dose) through safety follow-up (10 +/- 2 days after last dose)
Primary outcome [2] 0 0
Area Under The Concentration Versus Time Curve (AUC) Of Danicopan In Both Fed And Fasted States
Timepoint [2] 0 0
up to 72 hours postdose
Primary outcome [3] 0 0
Maximum Observed Concentration (Cmax) Of Danicopan In Both Fed And Fasted States
Timepoint [3] 0 0
up to 72 hours postdose
Primary outcome [4] 0 0
Time To Maximum Observed Concentration (Tmax) Of Danicopan In Both Fed And Fasted States
Timepoint [4] 0 0
up to 72 hours postdose
Primary outcome [5] 0 0
Dose Proportionality Of Danicopan In Fed State Assessed by AUC
Timepoint [5] 0 0
up to 72 hours postdose
Primary outcome [6] 0 0
Dose Proportionality Of Danicopan In Fed State Assessed by Cmax
Timepoint [6] 0 0
up to 72 hours postdose
Secondary outcome [1] 0 0
Activity Of Danicopan As Measured By Alternative Pathway Wieslab Assay
Timepoint [1] 0 0
up to 72 hours postdose
Secondary outcome [2] 0 0
Complement Factor B Fraction b Levels
Timepoint [2] 0 0
up to 72 hours postdose

Eligibility
Key inclusion criteria
1. Participants who are of Japanese descent defined as:

- First generation (born to 2 Japanese parents and 4 Japanese grandparents)

- Born in Japan, and not have lived outside Japan for greater than 10 years

- Lifestyle, including diet, must not have significantly changed since leaving
Japan

2. No clinically significant history or presence of electrocardiogram findings as judged
by the Investigator at screening and prior to the first dose of study intervention in
Period 1.

3. Body mass index in the range of 18.0 to 32.0 kilograms (kg)/meters squared, inclusive,
with a minimum body weight of 50.0 kg at screening.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Evidence of any clinically significant deviation from normal in clinical laboratory
evaluations, as determined by the Investigator or designee.

2. History of any medical or psychiatric condition or disease that, in the opinion of the
Investigator or designee, might limit the participant's ability to complete or
participate in this clinical study, confound the results of the study, or pose an
additional risk to the participant by their participation in the study.

3. History or presence of clinically significant hypersensitivity or idiosyncratic
reaction to the study interventions or related compounds or commonly used
antibacterial agents.

4. History of meningococcal infection, or a first-degree relative with a history of
meningococcal infection.

5. History of febrile illness, or other evidence of infection, within 14 days prior to
the first dose of study intervention.

6. Any major surgery within 4 weeks of the first dose of study intervention.

7. Diagnosis or history of Gilbert's syndrome, in the opinion of the Investigator or
designee.

8. Unable to refrain from or anticipates the use of any drug.

9. Receipt of a vaccine within 30 days prior to the first dose of study intervention.

10. Receipt of blood products within 6 months prior to the first dose of study
intervention.

11. Participation in any other investigational study drug trial in which receipt of an
investigational study drug occurred within 5 half-lives (if known) or 30 days prior to
the first dose of study intervention, whichever is longer.

12. Seated blood pressure is less than 90/40 millimeters of mercury (mmHg) or greater than
140/90 mmHg at screening.

13. Participants who test positive for human immunodeficiency virus, hepatitis B virus,
and/or hepatitis C virus.

14. Current tobacco users and smokers or a positive cotinine test at screening.

15. Donation of whole blood from 3 months prior to the first dose of study intervention,
or of plasma from 30 days prior to the first dose of study intervention.

16. Positive drugs of abuse screen at screening or Day -1 of Period 1.

17. Positive results for alcohol screen at screening or Day -1 of Period 1.

18. Is a female with a positive pregnancy test at screening or Day -1 of Period 1 or who
is lactating, or who plan to become pregnant (within 5 months of screening).

Study design
Purpose of the study
Treatment
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Crossover
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
17/08/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/09/2020
Sample size
Target
Accrual to date
Final
9
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Study Site - Brisbane
Recruitment postcode(s) [1] 0 0
- Brisbane

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics
(PD) of danicopan after a single-dose oral administration under fed and fasting conditions to
participants of Japanese descent.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04451434
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04451434