CLINICAL TRIALS PROFILE FOR WTX101

Clinical Trials for WTX101

Trial ID	Title	Status	Sponsor	Phase	Summary
NCT02273596 ↗	Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients	Completed	Alexion Pharmaceuticals	Phase 2	The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly diagnosed with Wilson Disease (WD) who were aged 18 and older and who had NCC concentrations within or above the reference range at the time of enrollment in the study. The study consisted of a 24-week Treatment Period, followed by a planned 36-month Extension Period.
NCT02273596 ↗	Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients	Completed	Wilson Therapeutics AB	Phase 2	The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly diagnosed with Wilson Disease (WD) who were aged 18 and older and who had NCC concentrations within or above the reference range at the time of enrollment in the study. The study consisted of a 24-week Treatment Period, followed by a planned 36-month Extension Period.
NCT03403205 ↗	Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease With an Extension Period of up to 60 Months	Active, not recruiting	Alexion Pharmaceuticals	Phase 3	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.
NCT03403205 ↗	Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease With an Extension Period of up to 60 Months	Active, not recruiting	Wilson Therapeutics AB	Phase 3	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.
NCT04526197 ↗	Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.	Completed	Alexion Pharmaceuticals	Phase 1	This was a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of celecoxib, a sensitive cytochrome P450 2C9 (CYP2C9) substrate, in healthy male and female participants. The safety and tolerability of ALXN1840 were determined along with ALXN1840 pharmacokinetics (PK) in plasma as measured via total molybdenum with the coadministration of celecoxib.

Clinical Trial Conditions for WTX101

Condition Name

Condition Name
Intervention	Trials
Wilson Disease	6
Healthy	5

Condition MeSH

Condition MeSH
Intervention	Trials
Hepatolenticular Degeneration	6

Clinical Trial Locations for WTX101

Trials by Country

Trials by Country
Location	Trials
United States	19
United Kingdom	8
Japan	6
Poland	3
Australia	3

Trials by US State

Trials by US State
Location	Trials
Texas	4
Nebraska	3
Washington	2
Tennessee	2
Michigan	2

Clinical Trial Progress for WTX101

Clinical Trial Phase

Clinical Trial Phase
Clinical Trial Phase	Trials
Phase 3	2
Phase 2	2
Phase 1	7

Clinical Trial Status

Clinical Trial Status
Clinical Trial Phase	Trials
Completed	7
Recruiting	2
Active, not recruiting	1

Clinical Trial Sponsors for WTX101

Sponsor Name

Sponsor Name
Sponsor	Trials
Alexion Pharmaceuticals	11
Wilson Therapeutics AB	2
ERT: Clinical Trial Technology Solutions	1

Sponsor Type

Sponsor Type
Sponsor	Trials
Industry	14
Other	1