Wilson Disease
Conditions
Brief summary
The main purpose of the study is to confirm how long ALXN1840 stays in the body of Japanese and non-Japanese healthy participants (that is, pharmacokinetic \[PK\] profile).
Interventions
DRUGALXN1840
Tablet for oral use.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
Key Inclusion Criteria: - Body weight ≤80 kilograms (kg) and body mass index (BMI) within the range 18-25 kg/m\^2, inclusive, at screening. - Negative serum pregnancy test. - Female participants of childbearing potential and male participants with a female spouse or partner of childbearing potential must be willing to follow protocol-specified contraception guidance starting at least one menstrual cycle before first study drug administration and continuing for up to 3 months after the end of systemic exposure of the study drug (that is, 3 months after end of study visit). Key
Exclusion criteria
- Current or recurrent/chronic disease (for example, cardiovascular, hematological, neurological, endocrine, immunological, rheumatological, renal, hepatic, or gastrointestinal (GI) or other conditions) that or could affect clinical assessments or clinical laboratory evaluations. - Current or relevant history of physical or psychiatric illness that are not stable or may require a change in treatment, use of prohibited therapies during the study or make the participant unlikely to fully comply with the requirements of the study or complete the study, or any condition that presents undue risk from the study drug or study procedures. - Any other significant disease or disorder which, in the opinion of the Investigator, may put the participant at risk. - History of significant allergic reaction (for example, anaphylaxis or angioedema) to any product (for example, food, pharmaceutical). - Use of prescription medications (excluding oral contraceptives) within 14 days prior to dosing on Day 1, except with prior approval of Alexion. - Use of non-prescription/ over-the-counter medications including vitamins and dietary or herbal supplements, within 7 days prior to dosing on Day 1. - Donated or lost 400 milliliters (mL) blood or more within the last 16 weeks preceding the first day of dosing.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose | Day 1 through Day 11 of Dosing Periods 1 and 2 |
Countries
United Kingdom
Participant flow
Recruitment details
For this study, Japanese participants were defined as those whose parents and grandparents were both Japanese and who had spent less than 5 years outside of Japan.
Pre-assignment details
A total of 24 participants were screened, all of whom were enrolled and received ALXN1840 in the study at 1 study site. Of the 24 enrolled, 12 were Japanese, therefore, they were allocated to Cohort 1, and 12 were non-Japanese, therefore, they were allocated to Cohort 2. All participants were included in all analysis sets.
Participants by arm
| Arm | Count |
|---|---|
| Cohort 1: Japanese Participants All Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2. | 12 |
| Cohort 2: Non-Japanese Participants All non-Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2. | 12 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Dosing Period 1 | Adverse Event | 1 | 0 |
Baseline characteristics
| Characteristic | Cohort 2: Non-Japanese Participants | Total | Cohort 1: Japanese Participants |
|---|---|---|---|
| Age, Continuous | 29.5 years STANDARD_DEVIATION 6.78 | 30.6 years STANDARD_DEVIATION 6.32 | 31.7 years STANDARD_DEVIATION 5.91 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 12 Participants | 24 Participants | 12 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 13 Participants | 12 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 11 Participants | 11 Participants | 0 Participants |
| Sex: Female, Male Female | 5 Participants | 10 Participants | 5 Participants |
| Sex: Female, Male Male | 7 Participants | 14 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 12 | 0 / 12 |
| other Total, other adverse events | 5 / 12 | 5 / 12 |
| serious Total, serious adverse events | 0 / 12 | 0 / 12 |
Outcome results
Primary
Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose
Time frame: Day 1 through Day 11 of Dosing Periods 1 and 2
Population: The PK population consisted of all participants who had sufficient plasma samples to enable the calculation of PK parameters of at least area under the curve (AUC) for at least one period.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1: Japanese Participants | Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose | Dosing Period 1: ALXN1840 15 mg | 10158.9753 hours* nanograms/milliliters | Standard Deviation 1427.41833 |
| Cohort 1: Japanese Participants | Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose | Dosing Period 2: ALXN1840 60 mg | 16178.7885 hours* nanograms/milliliters | Standard Deviation 6103.53245 |
| Cohort 2: Non-Japanese Participants | Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose | Dosing Period 1: ALXN1840 15 mg | 9221.0132 hours* nanograms/milliliters | Standard Deviation 3413.75213 |
| Cohort 2: Non-Japanese Participants | Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose | Dosing Period 2: ALXN1840 60 mg | 20911.3717 hours* nanograms/milliliters | Standard Deviation 11491.5869 |