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

• Status: Completed
• Trial end date: 2011-04-01

Summary

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.

• Phase: Phase 2/Phase 3

Details

• Lead Sponsor: Amsterdam Molecular Therapeutics
• Collaborator: The Clinical Trial Company
• Treatments: Cyclosporine; Cyclosporins; Methylprednisolone; Methylprednisolone acetate; Methylprednisolone Hemisuccinate; Mycophenolate mofetil; Mycophenolic Acid; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate