Table 1.

Visit schedule

Visit		1	2	3	4	5	6
Time and acceptable window (weeks)		-4	0*	13±1	26±2	39±2	52±2
Procedures		Screening and consent	Randomisation				End of treatment, end of trial	Unscheduled visit
Signed consent form		X
Demographics		X
Review of medical history		X
Assessment of eligibility criteria		X	X
Review of concomitant medications		X	X	X	X	X	X	X
Pulse and blood pressure		X		X	X	X	X	X†
Height and weight		X					X	X†
Give urine sample bottle to participant		X	X	X	X	X		X†
Clinical laboratory	Urine test‡		X	X	X	X	X
	Pregnancy test§	X	X¶	X	X	X	X	X†
	Blood tests (safety)**	X		X	X	X	X	X†
	High-sensitivity troponin blood test	X					X
	Haematocrit blood test††		X
	Sample handling/processing for central analysis	X	X	X	X	X	X	X†
	Specimen dispatch by post/courier	X	X	X	X	X	X	X†
Special procedure	ECG	X‡‡		X	X	X	X	X†
	CMR scan		X				X
	24-hour heart monitor	X					X
	Cardiopulmonary exercise test		X				X
	Phosphorus spectroscopy (subgroup)		X				X
Randomisation			X
Prescription of IMP			X	X	X	X
Pharmacy dispensing of IMP			X	X	X	X
Dispense participant diary and instruct			X
Pharmacy collection and recording of unused medication to assess compliance				X	X	X	X	X†
Review patient diary				X	X	X	X	X†
Review/reporting of AEs/SAEs				X	X	X	X	X
eCRF completion including data transfer and query resolution		X	X	X	X	X	X	X

Other abbreviations as per box 1.

*Visit 2 (randomisation) should take place within 28 days (4 weeks) of visit 1.

†Optional procedures performed at the investigator’s discretion

‡Urine test (efficacy): early morning urine collection for urine copper.

§Applies to females of childbearing capacity only. A serum pregnancy test will be performed at visit 1. Urine pregnancy tests will be performed at subsequent visits (serum testing may be performed if timely urine testing is not available).

¶At visit 2, female participants of childbearing capacity will be asked if there is a chance that they could have become pregnant since visit 1. If the participant confirms that there is a chance, then a urine pregnancy test will be performed.

**Blood tests (safety): blood count, renal function, serum iron, serum copper and serum caeruloplasmin.

††Haematocrit blood test (used as part of the CMR measurements)

‡‡May be performed at visit 1 or visit 2 but must be done before randomisation.

AE, adverse event; CMR, cardiovascular magnetic resonance; eCRF, electronic case report form; IMP, investigational medicinal product; SAE, serious adverse event.