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

Learn more about:
Related Clinical Trial
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 A Phase I/II Study of VTX-801 in Adult Patients With Wilson’s Disease Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 Study of Retinal Vascular Parameters in Patients With Wilson’s Disease Evaluation of Patients With Liver Disease ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders The Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment Establishment of Human Cellular Disease Models for Wilson Disease Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson’s Disease in Japan. Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson’s Disease-related Liver Failure The Individual Therapy for Patients With Wilson’s Disease Study of Zinc for Wilson Disease Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson’s Disease Study of Tetrathiomolybdate in Patients With Wilson Disease 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 WILSTIM – DBS (WILson STIMulation – Deep Brain Stimulation) Efficacy and Safety Study of Zinc Acetate to Treat Wilson’s Disease in Japan. Efficacy of Invitro Expanded Bone Marrow Derived Allogeneic Mesenchymal Stem Cell Transplantation Via Portal Vein or Hepatic Artery or Peripheral Vein in Patients With Wilson Cirrhosis Inhibitory rTMS in Dystonic Wilson Patients Natural History of Wilson Disease A Registered Cohort Study on Wilson’s Disease sCD163 and sMR in Wilsons Disease – Associations With Disease Severity and Fibrosis Cohort Research on Wilson’s Disease Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson’s Disease Patients Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

Brief Title

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

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

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.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 Safety and tolerability of individualized dosing of ALXN1840 by descriptive statistics

Condition

Wilson Disease

Intervention

ALXN1840

Study Arms / Comparison Groups

 ALXN1840 15-60 mg
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

215

Start Date

February 15, 2018

Completion Date

February 2026

Primary Completion Date

February 2021

Eligibility 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
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Eugene Swenson, MD, PhD, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT03403205

Organization ID

WTX101-301


Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

Eugene Swenson, MD, PhD, Study Director, Alexion Pharmaceuticals


Verification Date

April 2020