A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
- Conditions
- Wilson's Disease
- Interventions
- Genetic: VTX-801
- Registration Number
- NCT04537377
- Lead Sponsor
- Vivet Therapeutics SAS
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 4
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description VTX-801 VTX-801 -
- Primary Outcome Measures
Name Time Method Safety and tolerability profile (including treatment-emergent adverse events (TEAE)) at 1-Year post treatment 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 Outcome Measures
Name Time Method Free serum Cu at 1-Year post treatment Free serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
Total serum Cu at 1-Year post treatment Total serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
24-hour urinary Cu at 1-Year post treatment 24-hour urinary Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
VTX-801 Responder status 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.
Serum ceruloplasmin activity (enzymatic assay) at 1-Year post treatment Serum ceruloplasmin will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
Trial Locations
- Locations (10)
UC Davis Medical Center
🇺🇸Sacramento, California, United States
Yale University School of Medecine
🇺🇸New Haven, Connecticut, United States
Advent Health
🇺🇸Orlando, Florida, United States
University of Michigan Health System
🇺🇸Ann Arbor, Michigan, United States
Wake Forest School of Medicine
🇺🇸Winston-Salem, North Carolina, United States
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Aarhus University Hospital
🇩🇰Aarhus, Denmark
University Hospital Essen
🇩🇪Essen, Germany
Universitätsklinikum Tübingen (UKT)
🇩🇪Tübingen, Germany
Royal Surrey County Hospital
🇬🇧Guildford, Surrey, United Kingdom