Clinical trial NCT04537377: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Wilson's Disease
Lead sponsor: Vivet Therapeutics SAS
Sponsor class: INDUSTRY
First posted: 2020-09-03
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: Dose escalation study
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Safety and tolerability profile (including treatment-emergent adverse events (TEAE))
timeFrame: at 1-Year post treatment
Secondary outcomes:
measure: Free serum Cu
timeFrame: at 1-Year post treatment
measure: Total serum Cu
timeFrame: at 1-Year post treatment
measure: 24-hour urinary Cu
timeFrame: at 1-Year post treatment
measure: Serum ceruloplasmin activity (enzymatic assay)
timeFrame: at 1-Year post treatment
measure: VTX-801 Responder status
timeFrame: At Week 12 and Week 36
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: New Haven
country: United States
facility: Yale University School of Medecine
state: Connecticut
city: Orlando
country: United States
facility: Advent Health
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Winston-Salem
country: United States
facility: Wake Forest School of Medicine
state: North Carolina
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Essen
country: Germany
facility: University Hospital Essen
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen (UKT)
city: Guildford
country: United Kingdom
facility: Royal Surrey County Hospital
state: Surrey
Study officials:
References: