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

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 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

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