An update on gene therapy for the treatment of lipoprotein lipase deficiency

Figures & data

Table 1 Summary of clinical trial data for alipogene tiparvovec

Trial	Study size	Dose (gc/kg)	Study duration	Follow-up duration	Immunosuppressants	Primary endpoint	Secondary endpoint	Number of subjects meeting endpoint	References
CT-AMT010-01	8	1 × 10^11 (n=4) 3×10^11 (n=4)	12 weeks	5 years	No	TG reduction ≤ 10 mmol/L or TG reduction ≥40%	None	3 (38%)	9–13
CT-AMT011-01	14	3×10^11 (n=6) 1 × 10^12 (n=8)	12 weeks	5 years	Yes (n= 12) No (n=2)	TG reduction ≤ 10 mmol/L or TG reduction ≥40%	None	7 (50%)	9,1 1–13
CT-AMT011-02	5	1 × 10^12	14 weeks	1 year	Yes (n=5)	TG reduction ≤ 10 mmol/L or TG reduction ≥40%	Reduction in ppCM TG	5 (100%)	12,13,23
CT-AMT011-03	17 (from previous trials)	N/A	N/A	N/A	N/A	Reduction in pancreatitis occurrence	None	8 (47%)	13,25

Note: A total of 27 subjects participated in four clinical trials.

Abbreviations: ppCM, postprandial chylomicron; Ref, reference; TG, triglyceride; N/A, not available.