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

September 3, 2021

Completion Date

July 2027

Primary Completion Date

July 2023

Eligibility Criteria

        Main Inclusion Criteria:

          -  Male or female aged between 18 and 65 years

          -  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 > 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

          -  Any history of diabetes

          -  Pregnancy or breastfeeding

          -  Body Mass Index ≥ 35 kg/m2

        Other protocol defined Inclusion/ Exclusion criteria may apply
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

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

Location Countries

Denmark

Location Countries

Denmark

Administrative Informations


NCT ID

NCT04537377

Organization ID

VTX-801_CLN_001

Secondary IDs

2020-000963-22

Responsible Party

Sponsor

Study Sponsor

Vivet Therapeutics SAS


Study Sponsor

, , 


Verification Date

November 2022