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

Primary Purpose

Wilson's Disease

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
VTX-801
Sponsored by
Vivet Therapeutics SAS
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Wilson's Disease

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • UC Davis Medical CenterRecruiting
  • Yale University School of MedecineRecruiting
  • Advent HealthRecruiting
  • University of Michigan Health SystemRecruiting
  • Wake Forest School of MedicineRecruiting
  • University of Texas Southwestern Medical CenterRecruiting
  • Aarhus University HospitalRecruiting
  • Universitätsklinikum Tübingen (UKT)Recruiting
  • Royal Surrey County HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VTX-801

Arm Description

Outcomes

Primary Outcome Measures

Safety and tolerability profile (including treatment-emergent adverse events (TEAE))
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

Free serum Cu
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
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
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)
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
The number of Responders and Insufficient-Responders will be summarized by dose cohort and planned visit, with response to treatment.

Full Information

First Posted
August 19, 2020
Last Updated
April 21, 2023
Sponsor
Vivet Therapeutics SAS
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1. Study Identification

Unique Protocol Identification Number
NCT04537377
Brief Title
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
Acronym
GATEWAY
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

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 3, 2021 (Actual)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
March 2029 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vivet Therapeutics SAS

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Dose escalation study
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VTX-801
Arm Type
Experimental
Intervention Type
Genetic
Intervention Name(s)
VTX-801
Intervention Description
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.
Primary Outcome Measure Information:
Title
Safety and tolerability profile (including treatment-emergent adverse events (TEAE))
Description
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.
Time Frame
at 1-Year post treatment
Secondary Outcome Measure Information:
Title
Free serum Cu
Description
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.
Time Frame
at 1-Year post treatment
Title
Total serum Cu
Description
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.
Time Frame
at 1-Year post treatment
Title
24-hour urinary Cu
Description
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.
Time Frame
at 1-Year post treatment
Title
Serum ceruloplasmin activity (enzymatic assay)
Description
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.
Time Frame
at 1-Year post treatment
Title
VTX-801 Responder status
Description
The number of Responders and Insufficient-Responders will be summarized by dose cohort and planned visit, with response to treatment.
Time Frame
At Week 12 and Week 36

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sonia Valero
Phone
+33 1 83 81 17 10
Email
info@vivet-therapeutics.com
Facility Information:
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sandeep Dhaliwal
Phone
916-734-8696
Email
sandhaliwal@UCDAVIS.EDU
First Name & Middle Initial & Last Name & Degree
Valentina Medici, Dr
Facility Name
Yale University School of Medecine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Daksshi Hettiarachchi
Phone
203-737-5037
Email
daksshi.hettiarachchi@yale.edu
First Name & Middle Initial & Last Name & Degree
Michael Schilsky, Dr
Facility Name
Advent Health
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Calla Boatright
Phone
407-303-5600
Ext
112-3143
Email
Calla.Boatright@AdventHealth.com
First Name & Middle Initial & Last Name & Degree
Regino Gonzalez-Peralta, Dr
Facility Name
University of Michigan Health System
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ashley Page
Phone
734-998-9966
Email
ashpage@med.umich.edu
First Name & Middle Initial & Last Name & Degree
Frederick Askari, Dr
Facility Name
Wake Forest School of Medicine
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dee Faust
Phone
336-713-1442
Email
delannin@wakehealth.edu
First Name & Middle Initial & Last Name & Degree
Sean Rudnick, Dr
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dolores Santoyo
Phone
214-648-4412
Email
dolores.santoyo@utsouthwestern.edu
First Name & Middle Initial & Last Name & Degree
William Lee, Dr
Facility Name
Aarhus University Hospital
City
Aarhus
ZIP/Postal Code
8200
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Thomas Sandhal, Dr
Phone
+45 51504813
Email
thomsand@rm.dk
First Name & Middle Initial & Last Name & Degree
Thomas Sandhal, Dr
Facility Name
Universitätsklinikum Tübingen (UKT)
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ulrich Lauer, Dr
Phone
+49 7071-2983190
Email
ulrich.lauer@med.uni-tuebingen.de
First Name & Middle Initial & Last Name & Degree
Ulrich Lauer, Pr
Facility Name
Royal Surrey County Hospital
City
Guildford
State/Province
Surrey
ZIP/Postal Code
GU2 7XX
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aftab Ala, Dr.
Phone
(+44) 1483 571 122
Email
aftabala@nhs.net
First Name & Middle Initial & Last Name & Degree
Aftab Ala, Dr.

12. IPD Sharing Statement

Learn more about this trial

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

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