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

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

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 Overview

• Status: Active, not recruiting
• Conditions: Wilson's Disease
• Intervention / Treatment: Genetic: VTX-801

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Denmark
  • Aarhus, Denmark, 8200
    • Aarhus University Hospital
• Germany
  • Essen, Germany, 45147
    • University Hospital Essen
  • Tübingen, Germany, 72076
    • Universitätsklinikum Tübingen (UKT)
• United Kingdom
  • Surrey
    • Guildford, Surrey, United Kingdom, GU2 7XX
      • Royal Surrey County Hospital
• United States
  • California
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Yale University School of Medecine
  • Florida
    • Orlando, Florida, United States, 32803
      • Advent Health
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Health System
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157
      • Wake Forest School of Medicine
  • Texas
    • Dallas, Texas, United States, 75235
      • University of Texas Southwestern Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: VTX-801

Genetic: VTX-801; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants	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.	through primary completion visit, an average of 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Free Serum Cu	Free serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.	through primary completion visit, an average of 1 year
Total Serum Cu	Total serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.	through primary completion visit, an average of 1 year
24-hour Urinary Cu	24-hour urinary Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.	through primary completion visit, an average of 1 year
Serum Ceruloplasmin Activity (Enzymatic Assay)	Serum ceruloplasmin will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.	through primary completion visit, an average of 1 year
VTX-801 Responder Status	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.	At Week 12 and Week 36

Collaborators and Investigators

• Sponsor: Vivet Therapeutics SAS

Study record dates

Study Major Dates

• Study Start (Actual): September 3, 2021
• Primary Completion (Actual): December 17, 2024
• Study Completion (Estimated): June 18, 2029

Study Registration Dates

• First Submitted: August 19, 2020
• First Submitted That Met QC Criteria: August 28, 2020
• First Posted (Actual): September 3, 2020

Study Record Updates

• Last Update Posted (Actual): January 30, 2026
• Last Update Submitted That Met QC Criteria: January 29, 2026
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Digestive System Diseases; Neurodegenerative Diseases; Liver Diseases; Movement Disorders; Heredodegenerative Disorders, Nervous System; Basal Ganglia Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Metal Metabolism, Inborn Errors; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Hepatolenticular Degeneration
• Other Study ID Numbers: VTX-801_CLN_001; 2020-000963-22 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes