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Phase I-II

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

  • Study HIC#:2000028887
  • Last Updated:04/18/2024

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.

  • Age18 years - 60 years
  • GenderBoth

Contact Us

For more information about this study, including how to volunteer, contact:

Daksshi Hettiarachchi

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Eligibility Criteria

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

Principal Investigator

For more information about this study, including how to volunteer, contact: