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

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Brief Title

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

Official Title

A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease

Brief Summary

      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.

Study Phase

Phase 1/Phase 2

Study Type


Primary Outcome

Safety and tolerability profile (including treatment-emergent adverse events (TEAE))

Secondary Outcome

 Free serum Cu


Wilson's Disease



Study Arms / Comparison Groups



* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

January 2021

Completion Date

July 2027

Primary Completion Date

July 2023

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

          -  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

          -  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

          -  Pregnancy or breastfeeding

          -  Body Mass Index ≥ 30 kg/m2




18 Years - 60 Years

Accepts Healthy Volunteers



, +33 1 83 81 17 10, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Vivet Therapeutics SAS

Study Sponsor

, , 

Verification Date

November 2020