Wilson's DiseasePhase 1/2GATEWAYNCT04537377
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
A Phase 1/2 study testing VTX-801 in people with Wilson's Disease, run by Vivet Therapeutics SAS at 10 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Wilson's Disease
- What is being tested?
- VTX-801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 10 sitesin 4 countries
- Study dates
- 3 Sep 2021to 18 Jun 2029
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Trial locations and contacts
10 sites in 4 countries.
10 sites in 4 countries
United States6 sites
University of Michigan Health System
Ann Arbor, Michigan, 48109
University of Texas Southwestern Medical Center
Dallas, Texas, 75235
Yale University School of Medecine
New Haven, Connecticut, 06510
Advent Health
Orlando, Florida, 32803
UC Davis Medical Center
Sacramento, California, 95817
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157
Germany2 sites
University Hospital Essen
Essen, 45147
Universitätsklinikum Tübingen (UKT)
Tübingen, 72076
United Kingdom1 site
Royal Surrey County Hospital
Guildford, Surrey, GU2 7XX
About this study
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
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.
- Conditions
- Wilson's Disease
- Being tested
- VTX-801
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VTX-801Genetic
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.
| Study group | Role | What they receive |
|---|---|---|
| VTX-801 | Experimental | Not described Receives: Genetic: VTX-801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: through primary completion visit, an average of 1 year
Secondary outcomes (5)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: At Week 12 and Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2020
- Study start3 Sep 2021
- Primary completion17 Dec 2024
- Study completion18 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Wilson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Dose escalation study
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vivet Therapeutics SAS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04537377
- Sponsor's protocol ID
- VTX-801_CLN_001
- Other IDs
- 2020-000963-22
- Acronym
- GATEWAY
- Status last verified
- Dec 2025
- First submitted
- 19 Aug 2020
- Last updated
- 30 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04537377
- Acronym / sponsor ID
- GATEWAY / VTX-801_CLN_001
- Conditions
- Wilson's Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Vivet Therapeutics SASIndustry (pharma, biotech, devices)
- Study start
- 3 Sep 2021 (actual)
- Primary completion
- 17 Dec 2024 (actual)
- Study completion
- 18 Jun 2029 (estimated)
- First posted
- 3 Sep 2020 (actual)
- Results
- Posted 28 Jan 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Dose escalation study
| Arm | Type | Receives |
|---|---|---|
| VTX-801 | Experimental | Genetic: VTX-801 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VTX-801The 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. | Genetic | VTX-801 |
Primary outcome
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.
5 secondary outcomes
Free Serum Cu
Time frame: through primary completion visit, an average of 1 year
Total Serum Cu
Time frame: through primary completion visit, an average of 1 year
24-hour Urinary Cu
Time frame: through primary completion visit, an average of 1 year
Serum Ceruloplasmin Activity (Enzymatic Assay)
Time frame: through primary completion visit, an average of 1 year
VTX-801 Responder Status
Time frame: At Week 12 and Week 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VTX-801 in people with Wilson's Disease, run by Vivet Therapeutics SAS at 10 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Germany, Denmark, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Jan 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants (time frame: through primary completion visit, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2026.