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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04537377
Recruitment Status : Recruiting
First Posted : September 3, 2020
Last Update Posted : April 24, 2023
Sponsor:
Information provided by (Responsible Party):
Vivet Therapeutics SAS

Tracking Information
First Submitted Date  ICMJE August 19, 2020
First Posted Date  ICMJE September 3, 2020
Last Update Posted Date April 24, 2023
Actual Study Start Date  ICMJE September 3, 2021
Estimated Primary Completion Date March 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 22, 2020)		 Safety and tolerability profile (including treatment-emergent adverse events (TEAE)) [ Time Frame: 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.
Original Primary Outcome Measures  ICMJE
 (submitted: August 28, 2020)		 Safety and tolerability profile (including treatment-emergent adverse events (TEAE) [ Time Frame: 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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 22, 2020)
  • Free serum Cu [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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.
  • Serum ceruloplasmin activity (enzymatic assay) [ Time Frame: 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.
  • VTX-801 Responder status [ Time Frame: 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: August 28, 2020)
  • Free serum Cu [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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.
  • Serum ceruloplasmin activity (enzymatic assay) [ Time Frame: 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.
  • VTX-801 Responder status [ Time Frame: 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.
  • Radiocopper-related parameters (blood PK) [ Time Frame: Baseline, Week 12 and Week 36 ]
    Radiocopper concentrations in plasma will be listed and summarized by dose cohort, planned visit and timepoint, using descriptive statistics.
  • Radiocopper-related parameters (fecal excretion) [ Time Frame: Baseline, Week 12 and Week 36 ]
    Fecal radiocopper excretion rate will be summarized across each of the 24 hour collection periods (i.e. 24h, 48h and 72h), by dose cohort and planned visit, using descriptive statistics.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
Official Title  ICMJE 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
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Sequential Assignment
Intervention Model Description:
Dose escalation study
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Wilson's Disease
Intervention  ICMJE Genetic: VTX-801

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.
Study Arms  ICMJE Experimental: VTX-801
Intervention: Genetic: VTX-801
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 28, 2020)		 16
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2029
Estimated Primary Completion Date March 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  • Male or female aged between 18 and 65 years
  • 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 > ULN
  • Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Sonia Valero +33 1 83 81 17 10 info@vivet-therapeutics.com
Listed Location Countries  ICMJE Denmark,   Germany,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04537377
Other Study ID Numbers  ICMJE VTX-801_CLN_001
2020-000963-22 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Vivet Therapeutics SAS
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Vivet Therapeutics SAS
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vivet Therapeutics SAS
Verification Date April 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP