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