A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (GATEWAY)
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| ClinicalTrials.gov Identifier: NCT04537377 |
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Recruitment Status :
Recruiting
First Posted : September 3, 2020
Last Update Posted : November 7, 2023
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Sponsor:
Vivet Therapeutics SAS
Information provided by (Responsible Party):
Vivet Therapeutics SAS
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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 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 16 participants |
| Allocation: | N/A |
| 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 |
| Actual Study Start Date : | September 3, 2021 |
| Estimated Primary Completion Date : | March 2025 |
| Estimated Study Completion Date : | March 2029 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Wilson disease
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 :
- 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 :
- 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.
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| Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
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
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
| Contact: Sonia Valero | +33 1 83 81 17 10 | info@vivet-therapeutics.com |
Locations
| United States, California | |
| UC Davis Medical Center | Recruiting |
| Sacramento, California, United States, 95817 | |
| Contact: Sandeep Dhaliwal 916-734-8696 sandhaliwal@UCDAVIS.EDU | |
| Principal Investigator: Valentina Medici, Dr | |
| United States, Connecticut | |
| Yale University School of Medecine | Recruiting |
| New Haven, Connecticut, United States, 06510 | |
| Contact: Daksshi Hettiarachchi 203-737-5037 daksshi.hettiarachchi@yale.edu | |
| Principal Investigator: Michael Schilsky, Dr | |
| United States, Florida | |
| Advent Health | Recruiting |
| Orlando, Florida, United States, 32803 | |
| Contact: Pamela Hedrick 407-303-9826 ext 112-3143 Pamela.Hedrick@AdventHealth.com | |
| Principal Investigator: Regino Gonzalez-Peralta, Dr | |
| United States, Michigan | |
| University of Michigan Health System | Recruiting |
| Ann Arbor, Michigan, United States, 48109 | |
| Contact: Ashley Page 734-998-9966 ashpage@med.umich.edu | |
| Principal Investigator: Frederick Askari, Dr | |
| United States, North Carolina | |
| Wake Forest School of Medicine | Recruiting |
| Winston-Salem, North Carolina, United States, 27157 | |
| Contact: Dee Faust 336-713-1442 delannin@wakehealth.edu | |
| Principal Investigator: Sean Rudnick, Dr | |
| United States, Texas | |
| University of Texas Southwestern Medical Center | Recruiting |
| Dallas, Texas, United States, 75235 | |
| Contact: Amy Pugh 214-648-4412 amyb.pugh@utsouthwestern.edu | |
| Principal Investigator: William Lee, Dr | |
| Denmark | |
| Aarhus University Hospital | Recruiting |
| Aarhus, Denmark, 8200 | |
| Contact: Thomas Sandhal, Dr +45 51293791 thomsand@rm.dk | |
| Principal Investigator: Thomas Sandhal, Dr | |
| Germany | |
| University Hospital Essen | Recruiting |
| Essen, Germany, 45147 | |
| Contact: Hartmut Schmidt +452015990015 Hartmut.Schmidt@uk-essen.de | |
| Principal Investigator: Hartmut Schmidt, Pr. | |
| Universitätsklinikum Tübingen (UKT) | Recruiting |
| Tübingen, Germany, 72076 | |
| Contact: Ulrich Lauer, Dr +49 7071-2983190 ulrich.lauer@med.uni-tuebingen.de | |
| Principal Investigator: Ulrich Lauer, Pr | |
| United Kingdom | |
| Royal Surrey County Hospital | Recruiting |
| Guildford, Surrey, United Kingdom, GU2 7XX | |
| Contact: Aftab Ala, Dr. (+44) 1483 571 122 aftabala@nhs.net | |
| Principal Investigator: Aftab Ala, Dr. | |
Sponsors and Collaborators
Vivet Therapeutics SAS
| 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: | November 7, 2023 |
| Last Verified: | November 2023 |
| 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 |