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

Exclusion Criteria

• •Having a chronic inflammatory muscle disease.
• •Any current or relevant previous history of serious, severe or unstable physical or psychiatric illness, any medical disorder that may make the subject unlikely to fully complete the study, or any condition that presents undue risk from the study medication or procedures based on the investigator's opinion(eg. malignant neoplasia)
• •Active infectious disease of any nature, including clinically active viral infections
• •Having one of the following outcomes from the blood screening tests after appropriate correction due to the presence of chylomicronemia:
• •* Platelet count \< 100 x 109 /L
• •* Hemoglobin \< 6.2 mmol/L
• •* Liver function disturbances (bilirubin ≥1.5 x normal, ALT \> 2 x ULN (upper limit of normal)
• •* CPK \> 2 x ULN
• •* Cockcroft-Gault estimated creatinine clearance \< 50cc/min
• •* PT and PTT outside normal range or not determinable unless judged as acceptable for the subjects by the investigator
• •* Having a positive test for HIV, Hepatitis B, Hepatitis C or being positive for tuberculosis
• •Obesity defined as body mass index (BMI) \> 30 kg/m2
• •Having a recent history of alcohol or drug abuse e.g. barbiturates, cannabinoids and amphetamines, and the subject is positive in a urine screen for drugs of abuse
• •Using anti-coagulants
• •Participation in another clinical trial or receipt of any other investigational drug within 30 days of screening or planning to participate in another clinical trial during the course of the study, except observational studies
• •Subjects which cannot be treated with immunosuppressive medication or steroids
• •Known to be allergic to any constituent of the therapy (including the immune suppressors) or a having a condition that prohibits the use of therapy
• •Received previous treatment with AMT-010, Alipogene tiparvovec or other gene therapy investigational product
• •Requiring a post heparin plasma LPL activity test for diagnostic confirmation and having a history of heparin induced thrombocytopenia or other heparin related complications