Protocol Explorer - ALXN1840-WD-204

This protocol was contributed by a third party and has not been verified. Provided for informational and standards-development purposes only — see the Disclaimer & Terms of Use.

ALXN1840-WD-204

Status & Metadata

Status : Draft

Source Method : Owner Publish

Generation Method : Unknown

Date Added: 22 Jul 2026

Contributor: polecat-divot.5k

View USDM Protocol Download JSON

Additional Resources

CDISC CORE Conformance

Conformance assessed

ISSUE_REPORTED 14

SUCCESS 177

SKIPPED 13

EXECUTION_ERROR 3

Download Excel report Download JSON report

Rationale

The principal aim of this exploratory study is to investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD). The study will specifically evaluate the effects of 2 different ALXN1840 doses as well as the duration of treatment on copper balance to further elucidate the dose response of “decoppering” versus “maintenance” dose in participants with WD.

The secondary aim of the study is to characterize the steady-state absorption, distribution, metabolism, and excretion (ADME; mass balance) of total molybdenum which is a surrogate measure of ALXN1840 disposition in participants with WD.

ALXN1840 contains the active anion tetrathiomolybdate and has been shown to cause negative copper balance when administered to healthy animals as well as decopper the liver in animal models of WD. In Study WTX101-201, which assessed the safety and efficacy of ALXN1840 in participants with WD, ALXN1840 demonstrated enhanced copper control as measured by reduced plasma non-ceruloplasmin-bound copper (NCC) corrected (NCCcorrected ) for the stable tetrathiomolybdate- copper -albumin tripartite complex (TPC).

Depletion of copper by ALXN1840 has been shown to occur primarily through fecal elimination in both healthy and WD animal models. In this study, copper balance will be calculated as the difference between measured copper input in food and drink and measured copper output in urine and feces. This method is classically considered the most objective measure of decoppering ability with a decoppering agent able to drive a net negative copper balance.

As part of the secondary objective, to confirm that ALXN1840 steady state is adequately characterized by the plasma pharmacokinetics (PK), a molybdenum mass balance assessment will be performed at predicted steady state for the 15 mg/day and 30 mg/day doses: if steady state is achieved, molybdenum(out) will equal molybdenum(in). This approach may also detect potential accumulation in the liver or other tissues of participants with WD.

Key Metrics

Study Type Interventional Study

Study Phase Phase II Trial

Protocol Version 2

Study ID N/A

Schedule of Activities: Main Design

Timeline: Main Timeline

Visit / Timing	Interval	Window	Activities
Screening TIM1	42 Days	0..33 Days	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Discontinue Zinc TIM2	21 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Check in TIM3	8 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day -7 TIM4	7 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day -6 & -5 Start TIM5	0 minutes	No window	Enrollment/inclusion Retained serum sample (safety) Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Concomitant medication and non-pharmacologic therapy/procedure
Day -6 & -5 TIM6	1 Day	No window	Concomitant medication and non-pharmacologic therapy/procedure
Day -6 & -5 Decision TIM7	0 minutes	No window	Adverse events Cu/Mo-controlled meals Light exercise regimen Concomitant medication and non-pharmacologic therapy/procedure
Day -4 TIM8	4 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day -3 TIM9	3 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day -2 TIM10	2 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day -1 TIM11	1 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 1 TIM12	1 Day	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 2 TIM13	1 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 3 TIM14	2 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 4 TIM15	3 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 5 TIM16	4 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 6 TIM17	5 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 7 TIM18	6 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 8 TIM19	7 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 9 TIM20	8 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 10 TIM21	9 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 11 TIM22	10 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 12 TIM23	11 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 13 TIM24	12 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 14 TIM25	13 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 15 TIM26	14 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 16 TIM27	15 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 17 TIM28	16 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 18 TIM29	17 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 19 TIM30	18 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 20 TIM31	19 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 21 TIM32	20 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 22 TIM33	21 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 23 TIM34	22 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 24 TIM35	23 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 25 TIM36	24 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 26 TIM37	25 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 27 TIM38	26 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 28 TIM39	27 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 29 TIM40	28 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 30 TIM41	29 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 31 TIM42	30 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 32 TIM43	31 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 33 TIM44	32 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 34 TIM45	33 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 35 TIM46	34 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 36 TIM47	35 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 37 TIM48	36 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 38 TIM49	37 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 39 TIM50	38 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
Day 40 TIM51	39 Days	No window	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure
End of Study TIM52	53 Days	2..2 Days	Inclusion/exclusion Prior WD treatment ALXN1840 15 mg/day Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Vitals sign measurements 12-lead ECG (triplicate) Adverse events Cu/Mo-controlled meals Light exercise regimen Urination and bowel movement monitoring, menstruation checks 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure

Timeline: Adverse Event Timeline

Visit / Timing	Interval	Window	Activities
Adverse event TIM64	0 minutes	No window	Adverse events

Timeline: Early Termination Timeline

Visit / Timing	Interval	Window	Activities
Early termination TIM65	0 minutes	No window	Discuss/document contraception Physical examination Chemistry, hematology, Coagulation Urinalysis Urine/serum pregnancy tests Vitals sign measurements 12-lead ECG (triplicate) Adverse events Concomitant medication and non-pharmacologic therapy/procedure

Timeline: PKPD Timeline

Visit / Timing	Interval	Window	Activities
-30 minutes TIM53	30 minutes	3..3 minutes	No activities recorded
Start TIM54	0 minutes	No window	No activities recorded
1 hours TIM55	1 Hours	No window	No activities recorded
2 hours TIM56	2 Hours	No window	No activities recorded
3 hours TIM57	3 Hours	No window	No activities recorded
4 hours TIM58	4 Hours	No window	No activities recorded
5 hours TIM59	5 Hours	No window	No activities recorded
6 hours TIM60	6 Hours	No window	No activities recorded
8 hours TIM61	8 Hours	No window	No activities recorded
12 hours TIM62	12 Hours	No window	No activities recorded
24 hours TIM63	24 Hours	No window	No activities recorded

Timeline: Unscheduled Timeline

Visit / Timing	Interval	Window	Activities
Unscheduled TIM66	0 minutes	No window	Alcohol test Urine drug screen HIV, hepatitis B and C screen Medical history/demographicsi WD history Prior WD treatment Physical examination Heightl , weight, and BMI Blood sampling for PK: Plasma total Mo and PUF-Mo PD: Plasma total and PUF-Cu, LBC, ceruloplasmin, ceruloplasmin-bound Cu Chemistry, hematology, Coagulation Urinalysis Urine/serum pregnancy tests Retained serum sample (safety) Vitals sign measurements 12-lead ECG (triplicate) Adverse events 24-hour urine for Cu and Mo Feces for Cu and Mo Concomitant medication and non-pharmacologic therapy/procedure