Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects

NCTID	NCT00891306 (View at clinicaltrials.gov)
Description	This trial is designed to expand the currently available data on the safety and efficacy of alipogene tiparvovec treatment in lipoprotein lipase deficiency (LPLD) and to further the understanding of possible mechanisms of action of the therapy. (Show More)
Development Status	EMA approved - product is unavailable
Indication	Familial Lipoprotein Lipase Deficiency
Disease Ontology Term	DOID:14118
Compound Name	GLYBERA
Compound Alias	alipogene tiparvovec, AT-011
Compound Description	AAV1-LPL(S447X)
Sponsor	Amsterdam Molecular Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	5
Results Posted	Not Available

Target Gene/Variant	LPL
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intramuscular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV1
Editor Type	none
Dose 1	1E11 gc/kg
Dose 2	3E11 gc/kg
Dose 3	1E12 gc/kg (approved dose)
Dose 4
Dose 5

No.of Trial Sites	2
Locations	Canada

Has US IND
FDA Designations	Orphan Drug Designation
Recent Updates	Product was acquired by uniQure, uniQure did not renew their EMA marketing application when it expired 10/25/17, and Glybera is no longer commercially available