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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-001104-41 | EudraCT Number |
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This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALXN1840 | Experimental | Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALXN1840 | Drug | Administered orally as tablets. |
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| Measure | Description | Time Frame |
|---|---|---|
| Mean Daily Copper Balance: Day 1 Through Day 8 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) |
| Mean Daily Copper Balance: Day 31 Through Day 35 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Accumulation: Day 31 through Day 35 (ALXN1840 30 mg) |
| Mean Daily Copper Balance: Day 25 Through Day 28 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Accumulation: Day 25 through Day 28 (ALXN1840 15 mg) |
| Mean Daily Copper Balance: Day 36 Through Day 39 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Accumulation: Day 36 through Day 39 (ALXN1840 30 mg) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline In Mean Daily Copper Balance | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Eugene S. Swenson, MD, PhD | Alexion Pharmaceuticals, Inc. | Study Director |
| Peter Ksenuk, MD | Alexion Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | New Haven | Connecticut | 06510 | United States | ||
| Research Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 | Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. |
| FG001 | Cohort 2 | Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 milligrams/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 | Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. |
| BG001 | Cohort 2 |
| Units | Counts |
|---|---|
| Participants |
|
| 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 | Mean Daily Copper Balance: Day 1 Through Day 8 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams/day | Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) |
|
Baseline up to Day 56
Safety set included all participants who received at least 1 dose of ALXN1840.
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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 | Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 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 | Mar 18, 2022 | Jul 31, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 8, 2021 | Jul 31, 2023 | 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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| Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) | Copper was assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper. | Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg |
| Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State | The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure. | Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
| Change From Baseline In Total Molybdenum Excretion In Urine And Feces | Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
| Mean Daily Molybdenum Balance At ALXN1840 Steady State | Molybdenum balance at steady state was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum (out) equal to molybdenum (in). | Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
| Accumulation Of Molybdenum As Determined By Molybdenum Balance | Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). | Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg) |
| Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum | Day 1 up to Day 39 |
| Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum | Day 39 |
| Grafton |
| 1010 |
| New Zealand |
| Research Site | London | SE1 1YR | United Kingdom |
Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| OG001 |
| Cohort 2 |
Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. |
|
|
| Secondary | Change From Baseline In Mean Daily Copper Balance | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams/day | Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
|
|
|
| Secondary | Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) | Copper was assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams | Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg |
|
|
|
| Secondary | Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State | The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams | Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
|
|
|
| Secondary | Change From Baseline In Total Molybdenum Excretion In Urine And Feces | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams | Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
|
|
|
| Secondary | Mean Daily Molybdenum Balance At ALXN1840 Steady State | Molybdenum balance at steady state was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum (out) equal to molybdenum (in). | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams/day | Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) |
|
|
|
| Secondary | Accumulation Of Molybdenum As Determined By Molybdenum Balance | Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligrams | Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg) |
|
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| Primary | Mean Daily Copper Balance: Day 31 Through Day 35 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. | Posted | Mean | Standard Deviation | milligrams/day | Accumulation: Day 31 through Day 35 (ALXN1840 30 mg) |
|
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| Primary | Mean Daily Copper Balance: Day 25 Through Day 28 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. | Posted | Mean | Standard Deviation | milligrams/day | Accumulation: Day 25 through Day 28 (ALXN1840 15 mg) |
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| Primary | Mean Daily Copper Balance: Day 36 Through Day 39 | Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. | Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. | Posted | Mean | Standard Deviation | milligrams/day | Accumulation: Day 36 through Day 39 (ALXN1840 30 mg) |
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| Secondary | Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum | Pharmacokinetic (PK) analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK profiles. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanograms/milliliters | Day 1 up to Day 39 |
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| Secondary | Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum | Pharmacokinetic analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK profiles. Here, Number of participants analyzed signifies those who were evaluable for this outcome measure and number analyzed signifies those who were evaluable at specified time points. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours*nanograms/milliliters | Day 39 |
|
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|
| 0 |
| 8 |
| 0 |
| 8 |
| 6 |
| 8 |
| EG001 | Cohort 2 | Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39. | 0 | 1 | 0 | 1 | 1 | 1 |
| Abdominal distension | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Hordeolum | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Periodontitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Pulpitis dental | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
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| Nail injury | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
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| Liver function test abnormal | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Transaminases increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Migraine | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Sleep disorder | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Breast tenderness | Reproductive system and breast disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Phlebitis superficial | Vascular disorders | MedDRA v25.1 | Non-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 |
| Day 31 through Day 35 |
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| Day 36 through Day 39 |
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| Copper Food Content: Day 31 through Day 35 |
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| Copper Food Content: Day 36 through Day 39 |
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| Copper Drink Content: Day 1 through Day 8 |
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| Copper Drink Content: Day 25 through Day 28 |
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| Copper Drink Content: Day 31 through Day 35 |
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| Copper Drink Content: Day 36 through Day 39 |
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| Copper Feces Content: Day 1 through Day 8 |
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| Copper Feces Content: Day 25 through Day 28 |
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| Copper Feces Content: Day 31 through Day 35 |
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| Copper Feces Content: Day 36 through Day 39 |
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| Copper Urine Content: Day 1 through Day 8 |
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| Copper Urine Content: Day 25 through Day 28 |
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| Copper Urine Content: Day 31 through Day 35 |
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| Copper Urine Content: Day 36 through Day 39 |
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| Molybdenum Drink Content : Days 25 through 28 |
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| Molybdenum Drink Content : Days 36 through 39 |
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| Molybdenum Feces Content : Days 25 through 28 |
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| Molybdenum Feces Content : Days 36 through 39 |
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| Molybdenum Urine Content : Days 25 through 28 |
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| Molybdenum Urine Content : Days 36 through 39 |
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| Molybdenum Feces Content : Days 31 through 35 |
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| Molybdenum Feces Content : Days 36 through 39 |
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| Molybdenum Urine Content : Day 1 through 8 |
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| Molybdenum Urine Content : Days 25 through 28 |
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| Molybdenum Urine Content : Days 31 through 35 |
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| Molybdenum Urine Content : Days 36 through 39 |
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| Plasma ultrafiltrate molybdenum |
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