NCT00891306

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

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

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Amsterdam Molecular Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

LPLD is a rare autosomal recessive disorder, characterized by the presence of marked chylomicronemia and hence hypertriglyceridemia. Clinically the most severe manifestation of chylomicronemia, is acute pancreatitis, which can be lethal. There is no effective therapy available to modulate the course of the illness and prevent complications for these patients. The current clinical management consists of severe reduction of dietary fat that is hard if not almost impossible to comply with. LPLD subjects continue to experience pancreatitis attacks, and are admitted to intensive care units on several occasions. Alipogene tiparvovec corrects or restores lipoprotein lipase (LPL) function long term, and hence reverses some symptoms, halts the disease progression and prevents further complications. Alipogene tiparvovec gene therapy ensures that a catabolically beneficial variant of the human LPL gene, LPL\[S447X\] is expressed and active in the relevant tissues in humans. Delivery of the gene is realized via intramuscular injection of an adeno-associated viral vector, pseudotyped with AAV1 capsids.

Conditions

• Familial Lipoprotein Lipase Deficiency

Interventions

Type	Name	Description
GENETIC	Alipogene Tiparvovec (AMT-011), Human LPL [S447X]	intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections
DRUG	mycophenolate mofetil	oral, 2 g/day, day -3 till week 12
DRUG	cyclosporine	oral, 3 mg/kg/day, day -3 till week 12
DRUG	methylprednisolone	single intravenous bolus of methylprednisolone (1 mg/kg bodyweight)

Timeline

• Start date: 2009-02-01
• Primary completion: 2010-08-01
• Completion: 2011-04-01
• First posted: 2009-05-01
• Last updated: 2011-09-29

Locations

2 sites across 1 country: Canada