- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04537377
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (GATEWAY)
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 Overview
Study Type
Enrollment (Estimated)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Contact
- Name: Sonia Valero
- Phone Number: +33 1 83 81 17 10
- Email: info@vivet-therapeutics.com
Study Locations
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Aarhus, Denmark, 8200
- Recruiting
- Aarhus University Hospital
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Principal Investigator:
- Thomas Sandhal, Dr
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Contact:
- Thomas Sandhal, Dr
- Phone Number: +45 51293791
- Email: thomsand@rm.dk
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Essen, Germany, 45147
- Recruiting
- University Hospital Essen
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Contact:
- Hartmut Schmidt
- Phone Number: +452015990015
- Email: Hartmut.Schmidt@uk-essen.de
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Principal Investigator:
- Hartmut Schmidt, Pr.
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Tübingen, Germany, 72076
- Recruiting
- Universitätsklinikum Tübingen (UKT)
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Contact:
- Ulrich Lauer, Dr
- Phone Number: +49 7071-2983190
- Email: ulrich.lauer@med.uni-tuebingen.de
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Principal Investigator:
- Ulrich Lauer, Pr
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Surrey
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Guildford, Surrey, United Kingdom, GU2 7XX
- Recruiting
- Royal Surrey County Hospital
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Contact:
- Aftab Ala, Dr.
- Phone Number: (+44) 1483 571 122
- Email: aftabala@nhs.net
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Principal Investigator:
- Aftab Ala, Dr.
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California
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Sacramento, California, United States, 95817
- Recruiting
- UC Davis Medical Center
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Contact:
- Sandeep Dhaliwal
- Phone Number: 916-734-8696
- Email: sandhaliwal@ucdavis.edu
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Principal Investigator:
- Valentina Medici, Dr
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Connecticut
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New Haven, Connecticut, United States, 06510
- Recruiting
- Yale University School of Medecine
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Contact:
- Daksshi Hettiarachchi
- Phone Number: 203-737-5037
- Email: daksshi.hettiarachchi@yale.edu
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Principal Investigator:
- Michael Schilsky, Dr
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Florida
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Orlando, Florida, United States, 32803
- Recruiting
- Advent Health
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Principal Investigator:
- Regino Gonzalez-Peralta, Dr
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Contact:
- Pamela Hedrick
- Phone Number: 112-3143 407-303-9826
- Email: Pamela.Hedrick@AdventHealth.com
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Michigan
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Ann Arbor, Michigan, United States, 48109
- Recruiting
- University of Michigan Health System
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Contact:
- Ashley Page
- Phone Number: 734-998-9966
- Email: ashpage@med.umich.edu
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Principal Investigator:
- Frederick Askari, Dr
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North Carolina
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Winston-Salem, North Carolina, United States, 27157
- Recruiting
- Wake Forest School of Medicine
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Contact:
- Dee Faust
- Phone Number: 336-713-1442
- Email: delannin@wakehealth.edu
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Principal Investigator:
- Sean Rudnick, Dr
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Texas
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Dallas, Texas, United States, 75235
- Recruiting
- University of Texas Southwestern Medical Center
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Principal Investigator:
- William Lee, Dr
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Contact:
- Amy Pugh
- Phone Number: 214-648-4412
- Email: amyb.pugh@utsouthwestern.edu
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
at 1-Year post treatment
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
at 1-Year post treatment
|
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.
|
at 1-Year post treatment
|
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.
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at 1-Year post treatment
|
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.
|
at 1-Year post treatment
|
VTX-801 Responder status
Time Frame: At Week 12 and Week 36
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The number of Responders and Insufficient-Responders will be summarized by dose cohort and planned visit, with response to treatment.
|
At Week 12 and Week 36
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Basal Ganglia Diseases
- Movement Disorders
- Neurodegenerative Diseases
- Metabolism, Inborn Errors
- Heredodegenerative Disorders, Nervous System
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Metal Metabolism, Inborn Errors
- Hepatolenticular Degeneration
Other Study ID Numbers
- VTX-801_CLN_001
- 2020-000963-22 (EudraCT Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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