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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 : Not yet recruiting
First Posted : September 3, 2020
Last Update Posted : December 3, 2020
Sponsor:
Information provided by (Responsible Party):
Vivet Therapeutics SAS

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.

Condition or disease Intervention/treatment Phase
Wilson's Disease Genetic: VTX-801 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Intervention Model: Sequential Assignment
Intervention Model Description: Dose escalation study
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : January 2021
Estimated Primary Completion Date : July 2023
Estimated Study Completion Date : July 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease

Arm Intervention/treatment
Experimental: VTX-801 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.





Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Male or female aged between 18 and 60 years
  • Patient diagnosed with WD, as historically established and documented by Leipzig score ≥ 4, as per the 2012 EASL Clinical Practice Guidelines (EASL, 2012) and genetically confirmed
  • Treated for WD according to international recommendations with no current evidence for inadequate treatment (EASL, 2012; Roberts et al, 2008)
  • 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 including 24-hour urinary copper, non-ceruloplasmin copper (NCC), as well as liver enzymes, hemoglobin, and white blood cell count

Main Exclusion Criteria:

  • ALT level (mean of 2 measurements ≥ 1-week apart) > ULN or either of the 2 measurements > 1.5 x ULN
  • 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 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, according to the Investigator
  • Pregnancy or breastfeeding
  • Body Mass Index ≥ 30 kg/m2

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04537377


Contacts
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Contact: Sonia Valero +33 1 83 81 17 10 info@vivet-therapeutics.com

Locations
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United States, California
UC Davis Medical Center
Sacramento, California, United States, 95817
United States, Connecticut
Yale University School of Medecine
New Haven, Connecticut, United States, 06510
United States, Michigan
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
Vivet Therapeutics SAS
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Responsible Party: Vivet Therapeutics SAS
ClinicalTrials.gov Identifier: NCT04537377    
Other Study ID Numbers: VTX-801_CLN_001
2020-000963-22 ( EudraCT Number )
First Posted: September 3, 2020    Key Record Dates
Last Update Posted: December 3, 2020
Last Verified: November 2020

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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
Additional relevant MeSH terms:
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Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases