An Open-label Study to Assess the Efficacy and Safety of Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Expressed by an Adeno-Associated Viral Vector After Intramuscular Administration in LPL-deficient Adult Subjects

NCT00891306 Phase 2/Phase 3 COMPLETED

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.

Details

Lead sponsor	Amsterdam Molecular Therapeutics
Phase	Phase 2/Phase 3
Status	COMPLETED
Enrolment	5
Start date	2009-02
Completion	2011-04

Conditions

• Familial Lipoprotein Lipase Deficiency

Interventions

• Alipogene Tiparvovec (AMT-011), Human LPL [S447X]
• mycophenolate mofetil
• cyclosporine
• methylprednisolone

Primary outcomes

• Reduction of triglyceride (TG) concentrations — 12 weeks

Countries

Canada