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

Main Inclusion Criteria: * Male or female aged 18 and 65 years inclusive * Confirmed diagnosis of WD * Treated for WD according to international recommendations with no current evidence for inadequate treatment * Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder and (ii) Stable laboratory parameters used to assess copper metabolism Main Exclusion Criteria: * ALT level ≥ 2 ULN that is not readily explained by extrinsic factors * Total bilirubin \> 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin \> ULN * INR \> 1.2 * Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrollment 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 * Any history of diabetes * Pregnancy or breastfeeding * Body Mass Index ≥ 35 kg/m2 Othe…