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

Interventional


Primary Outcome

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

Secondary Outcome

 Free serum Cu

Condition

Wilson's Disease

Intervention

VTX-801

Study Arms / Comparison Groups

 VTX-801
Description:  

Publications

* 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

Genetic

Estimated Enrollment

16

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).

          -  Male patients: willing to use two methods of contraception from signing informed
             consent through at least 6 months following VTX-801 infusion.

          -  Female patients: if of childbearing potential, willing to use two methods of
             contraception from signing informed consent through at least one week after W36.

          -  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: stable liver tests and no significant change in
             neurologic examination and in status of mood disorder, if present, as judged by the
             Investigator.

        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 severe renal impairment or patient has nephritis or nephrotic syndrome.

          -  Any history or current evidence of HIV-1, HIV-2, HTLV 1, HTLV-2 infection or 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.
      

Gender

All

Ages

18 Years - 60 Years

Accepts Healthy Volunteers

No

Contacts

, +33 7 63 59 96 54, [email protected]



Administrative Informations


NCT ID

NCT04537377

Organization ID

VTX-801_CLN_001


Responsible Party

Sponsor

Study Sponsor

Vivet Therapeutics SAS


Study Sponsor

, , 


Verification Date

August 2020