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NCT Number: NCT04537377

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Wilson's Disease Basal Ganglia Diseases Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Hepatolenticular Degeneration Heredodegenerative Disorders, Nervous System Liver Diseases Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Movement Disorders Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Aarhus University Hospital, Aarhus, Denmark

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

VTX-801

Genetic

The investigational medicinal product (VTX-801) is a replication-deficient recombinant adeno-associated viral vector (rAAV) consisting of an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the ATP7B gene (ATP7B-minigene).

After reconstitution VTX-801 will be administered as a single dose intravenous (IV) administration per patient, at up to 3 different dose levels.

Primary outcomes

  1. Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants

    Time frame: through primary completion visit, an average of 1 year

    AEs will be summarized based on the date of onset for the event. Number of treatment-emergent AEs will be provided by SOC and PT, by dose cohort and overall.

Secondary outcomes

  1. Free Serum Cu

    Time frame: through primary completion visit, an average of 1 year

    Free serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.

  2. Total Serum Cu

    Time frame: through primary completion visit, an average of 1 year

    Total serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.

  3. 24-hour Urinary Cu

    Time frame: through primary completion visit, an average of 1 year

    24-hour urinary Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.

  4. Serum Ceruloplasmin Activity (Enzymatic Assay)

    Time frame: through primary completion visit, an average of 1 year

    Serum ceruloplasmin will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.

  5. VTX-801 Responder Status

    Time frame: At Week 12 and Week 36

    The number of Responders and Insufficient-Responders will be summarized by dose cohort and planned visit, with response to treatment. Responder status was assessed using radiocopper blood PK results and other cold copper parameters if needed.

Sponsors and collaborators

Lead sponsor

Vivet Therapeutics SAS

Industry

Registry information

Official study 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

Acronym: GATEWAY

Important dates

Study start
2021
Primary completion
2024
Study completion
2029
First posted
Sep 3, 2020
Registry last updated
Jan 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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