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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-004483-57 | EudraCT Number | ||
| C18055 | Other Identifier | RPL study code |
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: Japanese Participants | Experimental | All Japanese participants will receive a single dose of ALXN1840 15 milligrams (mg) in Dosing Period 1 and will receive a single dose of ALXN1840 60 mg in Dosing Period 2. |
|
| Cohort 2: Non-Japanese Participants | Experimental | All non-Japanese participants will receive a single dose of ALXN1840 15 mg in Dosing Period 1 and will receive a single dose of ALXN1840 60 mg in Dosing Period 2. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALXN1840 | Drug | Tablet for oral use. |
|
| Measure | Description | 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 |
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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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Richmond Pharmacology Ltd., St George's University of London | London | United Kingdom |
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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: Japanese Participants | All Japanese participants received a single dose of ALXN1840 15 milligrams (mg) in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Dosing Period 1 |
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| Dosing Period 2 |
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The safety population consisted of all participants who received at least 1 dose of the study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | Cohort 2: Non-Japanese Participants |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | 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. | Posted | Mean | Standard Deviation | hours* nanograms/milliliters | Day 1 through Day 11 of Dosing Periods 1 and 2 |
|
Baseline up to end of study visit (up to Day 29)
The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dizziness | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | Alexion Pharmaceuticals Inc. | +1 855-752-2356 | clinicaltrials@alexion.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 5, 2019 | Dec 15, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 8, 2019 | Dec 15, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006527 | Hepatolenticular Degeneration |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
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| COMPLETED |
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| NOT COMPLETED |
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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.
| BG002 | Total | Total of all reporting groups |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | 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.
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| 0 |
| 12 |
| 0 |
| 12 |
| 5 |
| 12 |
| EG001 | Cohort 2: Non-Japanese Participants | All non-Japanese participants received a single dose of 15 mg ALXN1840 in Dosing Period 1 and a single dose of 60 mg ALXN1840 in Dosing Period 2. | 0 | 12 | 0 | 12 | 5 | 12 |
| Headache | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
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| Presyncope | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
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| Paraesthesia | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
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| Syncope | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
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| Haemorrhoids | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
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| Medical device site reaction | General disorders | MedDRA 21.1 | Systematic Assessment |
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| Non-cardiac chest pain | General disorders | MedDRA 21.1 | Systematic Assessment |
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| Oral herpes | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
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| Liver function test abnormal | Investigations | MedDRA 21.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
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| Vaginal discharge | Reproductive system and breast disorders | MedDRA 21.1 | Systematic Assessment | The N for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event. |
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| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
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| D002493 |
| Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008664 | Metal Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |