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Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease With an Extension Period of up to 60 Months

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03403205
Recruitment Status : Active, not recruiting
First Posted : January 18, 2018
Last Update Posted : April 7, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the efficacy of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in WD patients aged 12 and older.

Condition or disease Intervention/treatment Phase
Wilson Disease Drug: ALXN1840 Drug: SoC Therapy Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 215 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Masking Description: This study is rater-blinded for the Unified Wilson Disease Rating Scale (UWDRS) assessment only.
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate the Efficacy and Safety of ALXN1840 Administered For 48 Weeks Versus Standard of Care in Patients With Wilson Disease Aged 12 Years and Older With an Extension Period of Up To 60 Months
Actual Study Start Date : February 15, 2018
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease

Arm Intervention/treatment
Experimental: ALXN1840 15-60 mg Drug: ALXN1840
ALXN1840 administered orally in 15 mg tablets
Other Name: Formerly named WTX101

Active Comparator: Standard of Care (SoC) Medication Drug: SoC Therapy
Depending on the site/region,subjects randomized to receive SoC treatment will receive trientine, penicillamine, Zinc, or a combination of these medicines, administered according to standard regimens.




Primary Outcome Measures :
  1. Evaluate the efficacy of ALXN1840 using standard of care as comparator and copper as the control assessed in terms of the percentage change from baseline (Day 1) to 48 weeks in non-ceruloplasmin-bound copper levels [ Time Frame: Change from baseline (Day 1) to 48 weeks ]

Secondary Outcome Measures :
  1. Safety and tolerability of individualized dosing of ALXN1840 by descriptive statistics [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  2. Effects of ALXN1840 on hepatic status [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  3. Effects of ALXN1840 on disability status [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  4. Effects of ALXN1840 on neurological status [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  5. Global effects of ALXN1840 on clinical symptoms as assessed by the Investigator on the Clinical Global Impression-Improvement Scale (CGI-I) and the Clinical Global Impression-Severity Scale (CGI-S) [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  6. Effects of ALXN1840 on NCC responder rate [ Time Frame: Change from baseline (Day 1) to 48 weeks ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion:

  • Established diagnosis of Wilson disease by Leipzig-Score = or > than 4
  • 12 years of age or older
  • Female patients of childbearing potential, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception starting at least 6 weeks before Day 1 visit and continuing through 28 days after the last dose of either ALXN1840 or SoC
  • Male patients, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception beginning at Day 1 and continuing through 90 days after last dose of either SoC or ALXN1840

Exclusion:

  • Decompensated hepatic cirrhosis
  • MELD score > 13
  • Modified Nazer score > 7
  • Clinically significant GI bleed within past 3 months
  • Alanine aminotransferase > 2 X upper limit of normal (ULN) for patients treated for > 28 days with WD therapy (Cohort 1)
  • Alanine aminotransferase > 5 X ULN for treatment naïve patients or patients who have been treated for < or = to 28 days (Cohort 2)
  • Marked neurological disease requiring either nasogastric feeding or intensive inpatient medical care
  • Hemoglobin < 9 g/dL
  • History of seizure activity within 6 months prior to informed consent
  • Pregnant (or women who are planning to become pregnant) or breastfeeding women
  • Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus or seropositivity for human immunodeficiency virus (HIV)
  • Previous treatment with tetrathiomolybdate
  • Patients with end-stage renal disease on dialysis (CKD 5) or creatinine clearance < 30 mL/min

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03403205


Locations
Show Show 87 study locations
Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
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Study Director: Eugene Swenson, MD, PhD Alexion Pharmaceuticals

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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03403205    
Other Study ID Numbers: WTX101-301
First Posted: January 18, 2018    Key Record Dates
Last Update Posted: April 7, 2020
Last Verified: April 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases