Study of UX701 Gene Transfer for the Treatment of Wilson Disease

NCTID	NCT04884815 (View at clinicaltrials.gov)
Description	The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the effect of UX701 on copper regulation. (Show More)
Indication	Wilson's Disease
Compound Name	Rivunatpagene miziparvovec (UX701)
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	78

Target Gene/Variant	ATP7B
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
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	5.0 x 10^12 GC/kg
Dose 2	1.0 x 10^13 GC/kg
Dose 3	2.0 x 10^13 GC/kg
Dose 4	Undisclosed dose 4
Dose 5

No.of Trial Sites	17
Locations	Canada,United States,United Kingdom,Portugal,Spain

Has US IND	True
Recent Updates	All patients in Phase I have been dosed, data expected H2 2024, expansion dose selection to follow