A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (GATEWAY)

Main Inclusion Criteria:

• Male or female aged between 18 and 60 years
• Patient diagnosed with WD, as historically established and documented by Leipzig score ≥ 4, as per the 2012 EASL Clinical Practice Guidelines (EASL, 2012) and genetically confirmed
• Treated for WD according to international recommendations with no current evidence for inadequate treatment (EASL, 2012; Roberts et al, 2008)
• Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder, if present, as judged by a physician-expert(s) in assessing neurological and psychiatric manifestations of WD and (ii) Stable laboratory parameters used to assess copper metabolism including 24-hour urinary copper, non-ceruloplasmin-bound copper (NCC), as well as liver enzymes, hemoglobin, and white blood cell count

Main Exclusion Criteria:

• ALT level (mean of 2 measurements ≥ 1-week apart) > ULN or either of the 2 measurements > 1.5 x ULN
• Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin > ULN
• INR > ULN
• Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment visit
• Patient has moderate or severe renal impairment defined as eGFR CKD-EPI < 60 mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome
• Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection
• Any history or current evidence of hepatitis B infection
• Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative
• Positive QuantiFERON®-TB Gold tuberculosis test result
• Any concomitant disorder/condition - including hepatic disorders - or treatment possibly interfering with the conduct or evaluation of the study, according to the Investigator
• Pregnancy or breastfeeding
• Body Mass Index ≥ 30 kg/m2