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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
Drug Product 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
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV8
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 Phase 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