Gene Transfer Clinical Study in Crigler-Najjar Syndrome

NCTID	NCT03223194 (View at clinicaltrials.gov)
Description	This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year. Subjects will receive a single dose of AT342 and will be followed for safety and efficacy for 5 years. (Show More)
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
Results Posted	Not Available

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
Target Tissue/Cell
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)
Dose 4
Dose 5

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

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