Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
Study Identifier:
ALXN1840-WD-302
ClinicalTrials.gov Identifier:
EudraCT Identifier:
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other
Available Documents
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: ALXN1840
- Drug: Standard of Care
Date
Sep 2021 - Jun 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 3 - 17 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Diagnosis of Wilson Disease by Leipzig Score ≥ 4.
- Adequate venous access to allow collection of required blood samples.
- Able to swallow intact ALXN1840 tablets or mini-tablets.
- Willing to avoid intake of foods and drinks with high contents of copper.
- Willing and able to follow protocol-specified contraception requirements.
Exclusion Criteria
- Decompensated hepatic cirrhosis or MELD score \> 13 (ages 12 to \<18) or PELD score \> 13 (ages 3 to \< 12).
- Modified Nazer score \> 7.
- Clinically significant gastrointestinal bleed within past 3 months.
- Alanine aminotransferase (ALT) \> 2 × upper limit of normal (ULN) for participants treated for \> 28 days with WD therapy or ALT \> 5 × ULN for treatment-naïve participants or participants who have been treated for ≤ 28 days.
- Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
- Hemoglobin less than lower limit of the reference range for age and sex.
- History of seizure activity within 6 months prior to informed consent/assent.
- Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or estimated glomerular filtration rate \< 30 milliliters/minute/1.73 meter squared.
Sex
Female & Male
Age
3 - 17 Years
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: ALXN1840
- Drug: Standard of Care
Date
Sep 2021 - Jun 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 3 - 17 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Diagnosis of Wilson Disease by Leipzig Score ≥ 4.
- Adequate venous access to allow collection of required blood samples.
- Able to swallow intact ALXN1840 tablets or mini-tablets.
- Willing to avoid intake of foods and drinks with high contents of copper.
- Willing and able to follow protocol-specified contraception requirements.
Exclusion Criteria
- Decompensated hepatic cirrhosis or MELD score \> 13 (ages 12 to \<18) or PELD score \> 13 (ages 3 to \< 12).
- Modified Nazer score \> 7.
- Clinically significant gastrointestinal bleed within past 3 months.
- Alanine aminotransferase (ALT) \> 2 × upper limit of normal (ULN) for participants treated for \> 28 days with WD therapy or ALT \> 5 × ULN for treatment-naïve participants or participants who have been treated for ≤ 28 days.
- Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
- Hemoglobin less than lower limit of the reference range for age and sex.
- History of seizure activity within 6 months prior to informed consent/assent.
- Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or estimated glomerular filtration rate \< 30 milliliters/minute/1.73 meter squared.
Protocol Summary
This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).
Trial Locations
Location
Status
Location
Research Site
Parkville, Australia, VIC 3052
Status
N/A
Location
Research Site
South Brisbane, Australia, 4101
Status
N/A
Location
Research Site
Lille, France, 59037
Status
N/A
Location
Research Site
Toulouse, France, 31059
Status
N/A
Location
Research Site
Hanover, Germany, 30625
Status
N/A
Location
Research Site
Tübingen, Germany, 72076
Status
N/A
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