A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
The objectives of this clinical trial are to assess, for up to 5 years, the safety,
 tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene
 therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and
 following background WD therapy withdrawal.
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,and (ii) stable laboratory parameters used
 to assess copper metabolism including 24-hour urinary copper, non-ceruloplasmin 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 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