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Gene Therapy Trial Report

Summary

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


NCTID NCT04537377 (View at clinicaltrials.gov)
Description
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 (ACTUAL)
Results Posted View Results

Therapy Information


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

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-08-19
Completion Date 2029-06-18
Last Update 2026-01-30

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


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

Regulatory Information


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

Resources/Links