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

NCTID	NCT04537377 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Inactive
Indication	Wilson Disease
Disease Ontology Term	DOID:893
Compound Name	VTX-801
Compound Alias	nimatpagene pariparvovec
Compound Description	AAV8-AAT-miniATP7B
Sponsor	Vivet Therapeutics SAS
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	4
Results Posted	Not Available

Target Gene/Variant	ATP7B-minigene
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Liver
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type	none
Dose 1	Undisclosed dose escalation, 3 levels
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	10
Locations	United States,Denmark,United Kingdom,Germany

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Sponsor terminated the trial due to efficacy concerns at the two doses tested, insufficient funding for further dose escalation