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

NCT04537377 | Active Not Recruiting Phase 1 Results DMC FDA Drug

• 2 other identifiers: VTX-801_CLN_0012020-000963-22
• Study Type: interventional
• Target: 4
• Locations: 4 countries
• Sites: 10

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.

Conditions

Wilson's Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Free Serum Cu

  through primary completion visit, an average of 1 year

• Total Serum Cu

  through primary completion visit, an average of 1 year

• 24-hour Urinary Cu

  through primary completion visit, an average of 1 year

• Serum Ceruloplasmin Activity (Enzymatic Assay)

  through primary completion visit, an average of 1 year

• VTX-801 Responder Status

  At Week 12 and Week 36

Study Arms (1)

VTX-801

EXPERIMENTAL

Genetic: VTX-801

Interventions

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.

VTX-801

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vivet Therapeutics SAS: lead

Study Sites (10)

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Yale University School of Medecine

New Haven, Connecticut, 06510, United States

Location

Advent Health

Orlando, Florida, 32803, United States

Location

University of Michigan Health System

Ann Arbor, Michigan, 48109, United States

Location

Wake Forest School of Medicine

Winston-Salem, North Carolina, 27157, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

Location

Aarhus University Hospital

Aarhus, 8200, Denmark

Location

University Hospital Essen

Essen, 45147, Germany

Location

Universitätsklinikum Tübingen (UKT)

Tübingen, 72076, Germany

Location

Royal Surrey County Hospital

Guildford, Surrey, GU2 7XX, United Kingdom

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

The study was prematurely terminated due to futility and not for safety reasons, because the VTX-801 pharmacodynamics effects observed in the first 4 patients at the first 2 dose levels tested were insufficient to allow withdrawal of the SoC. Due to the early study termination and limited data, no conclusions can be drawn regarding the predefined objectives. The safety results were consistent with the expected safety profile for VTX-801. No new safety issues were observed.

Results Point of Contact

• Title: Head of Clinical Operations
• Organization: Vivet Therapeutics

svalero@vivet-therapeutics.com

+33763599654

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Dose escalation study

Study Record Dates

• First Submitted: August 19, 2020
• First Posted: September 3, 2020
• Study Start: September 3, 2021
• Primary Completion: December 17, 2024
• Study Completion (Estimated): June 18, 2029
• Last Updated: January 30, 2026
• Results First Posted: January 28, 2026

Record last verified: 2025-12

Locations

US (6); GB (1); DK (1); DE (2)