GATEWAY clinical trial
Research type
Research Study
Full 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
IRAS ID
290707
Contact name
Jean-Philippe Combal
Contact email
Sponsor organisation
Vivet Therapeutics SAS
Eudract number
2020-000963-22
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 8 months, 26 days
Research summary
This study is being conducted to investigate the effects of an experimental drug called VTX-801 intended to treat Wilson's Disease (WD).Approximately 16 patients will participate in 4 countries (UK, USA, Germany and Denmark).
WD is a genetic disorder which causes copper to accumulate to toxic levels in the liver, brain and other vital organs. This is due to a defect in a gene that carries the information for the body to produce ATP7B, a protein whose role in the liver is to get rid of excess copper. Medical treatments for this condition, are available and must be taken for life. Patients enrolled in this study will have Wilson's Disease and will currently be receiving treatment with either a zinc salt therapy, copper chelator (D-penicillamine or trientine) or the association thereof. They may also follow a low copper diet.VTX-801 is a type of gene therapy. Gene therapy is based upon the administration of a gene to patients in order to treat or prevent their disease. In gene therapy, an inactivated virus (called a vector) is used to deliver the gene to living cells. In the case of VTX-801, a virus called “adeno-associated virus (AAV)” has been modified to transport a version of the ATP7B gene into liver cells. Importantly, the vector cannot cause an infection. VTX-801 is administered as a single injection in the subject's veins.
It is hoped that patients receiving VTX-801 will benefit from long-term disease stabilization (possibly an improvement) together with complete cessation of standard of care WD therapy and low copper diet, if any.
REC name
North East - York Research Ethics Committee
REC reference
22/NE/0048
Date of REC Opinion
7 Jun 2022
REC opinion
Further Information Favourable Opinion