Contact SCGE




Gene Therapy Trial Report

Summary

Gene Transfer Clinical Study in Crigler-Najjar Syndrome


NCTID NCT03223194 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Crigler-Najjar Syndrome
Disease Ontology Term DOID:3803
Compound Name AT342
Sponsor Audentes Therapeutics
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 1 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant UGT1A1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 1.5E12 vg/kg
Dose 2 Planned medium dose: 6.0E12 vg/kg (never administered)
Dose 3 Planned high dose: 1.5E12 vg/kg (never administered)

Study Record Dates


Current Stage Phase1
Submit Date 2017-07-17
Completion Date 2021-02-11
Last Update 2022-05-18

Participation Criteria


Eligible Age >=1 Year
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: * Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene as assessed by a Sponsor-approved testing facility. * Subject is aged ≥1 year. * Subject is prescribed daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time). Key Exclusion Criteria: * Subject is currently participating in an interventional study or has received gene or cell therapy. * Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the treatment period of this study. * Subject has significant cholestatic disease at screening. * Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening. * Subject tests positive for AAV8 neutralizing antibodies with titers above protocol specified threshold. * Other than as required per protocol, subject has received immune-modulating agents within 3 months before dosing (use of inhaled corticosteroids to manage chronic respiratory conditions is allowed); use of other concomitant medications to manage chronic conditions must have been stable for at least 4 weeks before dosing. * Subject has any clinically significant laboratory values, in the opinion of the investigator. * Subject has clinically significant underlying liver disease (other than CN) at screening. * Subject has a history of, or currently has, a clinically important condition other than CN, in the opinion of the investigator.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States,United Kingdom,Israel

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Sponsor terminated development, only 1 patient was enrolled in the study prior to termination and received the lowest dose which was well-tolerated but did not demonstrate efficacy beyond a few weeks

Resources/Links