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

Launched by VIVET THERAPEUTICS SAS · Aug 28, 2020

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment called VTX-801 for adults with Wilson's Disease, a condition that causes harmful amounts of copper to build up in the body. Researchers want to find out if VTX-801 is safe and how well it works over a period of up to 5 years. Participants will receive this gene therapy through an intravenous (IV) injection and will be monitored closely before and after stopping their current treatment for Wilson's Disease.

To be eligible for the trial, participants must be between 18 and 65 years old and have been diagnosed with Wilson's Disease. They should have been stable for at least a year, meaning their symptoms and lab results show no major changes. However, some people cannot participate, including those with certain liver or kidney issues, infections like hepatitis, or conditions that could interfere with the study. If you choose to take part, you can expect regular check-ups and tests to ensure your safety and to help the researchers learn more about how effective the treatment is.

Gender

ALL

Eligibility criteria

• 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