Overview
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.
Eligibility
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
Other protocol defined Inclusion/ Exclusion criteria may apply