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

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Brief Title

Efficacy and Safety of ALXN1840 (Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

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

Brief Summary

      The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101)
      administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD)
      participants aged 12 and older in the Primary Evaluation Period. In addition, efficacy and
      safety will be evaluated during an optional 60-month Extension Period.
    

Detailed Description

      The study consists of 2 cohorts. Cohort 1: Participants who have received SoC therapy for >
      28 days and Cohort 2: Participants who are treatment-naïve or who have received SoC therapy
      for ≤ 28 days.

      All enrolled participants were randomized by cohort in a 2:1 ratio to treatment with ALXN1840
      or SoC (either as continued therapy in Cohort 1 or as continued or initial therapy in Cohort
      2).
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Daily Mean Area Under The Effect-time Curve (AUEC) Of Directly Measured Non-ceruloplasmin-bound Copper (dNCC)

Secondary Outcome

 Change From Baseline In cNCC In Plasma.

Condition

Wilson Disease

Intervention

ALXN1840

Study Arms / Comparison Groups

 ALXN1840
Description:  ALXN1840 was administered orally for 48 weeks at doses ranging from 15 milligrams (mg) every other day (QOD) up to a titrated dose of 60 mg daily.
Participants who completed the Primary Evaluation Period had the option to participate in the up to 60-month Extension Period.

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 28, 2026

Primary Completion Date

February 24, 2021

Eligibility Criteria

        Key Inclusion Criteria:

          -  Established diagnosis of WD by Leipzig-Score ≥ than 4

          -  Female participants of childbearing potential, if heterosexually active, must be
             willing to follow protocol-specified guidance for highly effective contraception
             starting at least 6 weeks before the Day 1 visit and continuing through 28 days after
             the last dose of either ALXN1840 or SoC

          -  Male participants, if heterosexually active, must be willing to follow
             protocol-specified guidance for highly effective contraception beginning at Day 1
             visit and continuing through 90 days after last dose of either ALXN1840 or SoC

        Key Exclusion Criteria:

          -  Decompensated hepatic cirrhosis

          -  MELD score > 13

          -  Modified Nazer score > 7

          -  Clinically significant gastrointestinal bleed within past 3 months

          -  Alanine aminotransferase > 2 X upper limit of normal (ULN) for participants treated
             for > 28 days with WD therapy (Cohort 1)

          -  Alanine aminotransferase > 5 X ULN for treatment-naïve participants or participants
             who have been treated for ≤ 28 days (Cohort 2)

          -  Marked neurological disease requiring either nasogastric feeding or intensive
             inpatient medical care

          -  Hemoglobin < 9 grams/deciliter

          -  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

          -  Participants with end-stage renal disease on dialysis (chronic kidney disease stage 5)
             or creatinine clearance < 30 milliliter/minute
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT03403205

Organization ID

WTX101-301


Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

, , 


Verification Date

April 2022