NCT04537377Active, not recruitingPhase 1
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
Vivet Therapeutics SAS10 sites in 4 countries4 target enrollmentStarted: September 3, 2021Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Vivet Therapeutics SAS
- Enrollment
- 4
- Locations
- 10
- Primary Endpoint
- Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion 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
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
Arms & Interventions
VTX-801
Experimental
Intervention: VTX-801 (Genetic)
Outcomes
Primary Outcomes
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.
Secondary Outcomes
- 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)
Investigators
Study Sites (10)
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