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Prospective, Non-interventional, Non-randomised, Open-label, Adult Study to Assess the Long Term Biological Therapeutic Response to Alipogene Tiparvovec in Lipoprotein Lipase Deficiency (LPLD) and Comparing Postprandial Chylomicron Metabolism Following a Radiolabeled Meal in LPLD Subjects Previously Treated With Alipogene Tiparvovec (Studies CT-AMT-011-01 or -02) to Untreated LPLD Subjects (Study PREPARATION-02) and to Healthy Volunteers
Sponsor study identifier
observational
19
1 country
1
Study Start
September 2011
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
September 2012
Actual (reported)Study Completion
September 2012
Actual (reported)LPL (Lipoprotein Lipase) is an enzyme which plays an important role in the elimination of triglycerides (fat) and the clearance of dietary fat particles known as chylomicrons (CM) in the blood. In patients who have an abnormal LPL gene, the enzyme does not work (total, hereditary LPL deficiency), which results in a large increase in the amount of triglycerides (fats) and chylomicrons in the blood. This increases the risk of inflammation in the pancreas and leads to long term negative effects for bloods vessels (atherosclerosis). Current medications and / or a strict and low fat diet do not sufficiently reduce the level of triglycerides in order to prevent these conditions. To solve this problem, the company, AMT is developing a gene therapy (AMT-011). In normal healthy individuals, fat particles are rapidly cleared from the circulation following a standard meal. Within approximately 3 hours the highest levels of fat is reached and clearance is achieved within the subsequent 9 hours. In LPLD subjects, the clearance of fat is greatly reduced as a direct consequence of the lack of LPL. During this study, a standard meal with a tracer (3H-palmitate) is given. Since palmitate is incorporated in the dietary fat, this study enabled monitoring of appearance of newly formed dietary fat into- and clearance of these newly formed dietary fats from the circulation, over time. The principal aim of the study is to verify if the gene therapy (AMT 011) is still effective in the treatment of this condition. Systemic appearance and clearance of new formed dietary fat particles after ingestion of the meal will be determined by measuring the level of tracer at different time points.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
September 2012
Actual (reported)Click on a node to explore related trials.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
September 2011
Date type not reportedThis date has not been verified as actual or estimated.
September 28, 2011
Actual (reported)October 6, 2011
Actual (reported)September 2012
Actual (reported)September 2012
Actual (reported)Composite of Pharmacodynamics
Peak level, time-to-peak, and area under the curve (AUC) for tracer in plasma and chylomicron (CM) fraction to assess metabolism of newly-formed CMs in LPLD subjects previously treated with alipogene tiparvovec and to compare with untreated LPLD subjects and healthy controls.
pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose
Triglyceride (TG)-rich lipoproteins
pre dose, 0, 1, 2, 3, 4, 4, 6, 7, 8, 9, 12, 24 hours post dose
Glucose
pre dose, 0, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose
Adverse Events (AE)
Up to 21 days. Serious AEs will be followed to their resolution.
Laboratory tests
Up to 21 days
Vital signs
Up to 21 days
Subjects in Cohort 1 (previously treated LPLD Cohort) must have received AMT-011 during Studies CT-AMT-011-01 or -02
Subjects in Cohort 2 (untreated LPLD control Cohort)) may have completed study PREPARATION-02 or known patients with genetically confirmed LPLD
Volunteers in Cohort 3 (normal healthy control Cohort) must not have LPLD
* Lipoprotein Lipase Deficiency (LPLD) Patients * Healthy volunteers (community sample)
ECOGENE-21 Clinical Trial Center
Chicoutimi, Quebec, G7H 7P2, Canada
Bickerton AS, Roberts R, Fielding BA, Hodson L, Blaak EE, Wagenmakers AJ, Gilbert M, Karpe F, Frayn KN. Preferential uptake of dietary Fatty acids in adipose tissue and muscle in the postprandial period. Diabetes. 2007 Jan;56(1):168-76. doi: 10.2337/db06-0822.
Black DM, Sprecher DL. Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat. Am J Dis Child. 1993 Jan;147(1):60-2. doi: 10.1001/archpedi.1993.02160250062018.
whole blood, serum, urine, faeces
Daniel Gaudet, MD PhD
ECOGENE-21 Clinical Trial Center
First Submitted
September 28, 2011
First Posted
October 6, 2011
Study Start
September 2011
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
September 2012
Actual (reported)Contact the study team to confirm eligibility.
Chait A, Brunzell JD. Chylomicronemia syndrome. Adv Intern Med. 1992;37:249-73.
Fortson MR, Freedman SN, Webster PD 3rd. Clinical assessment of hyperlipidemic pancreatitis. Am J Gastroenterol. 1995 Dec;90(12):2134-9.
Miles JM, Park YS, Walewicz D, Russell-Lopez C, Windsor S, Isley WL, Coppack SW, Harris WS. Systemic and forearm triglyceride metabolism: fate of lipoprotein lipase-generated glycerol and free fatty acids. Diabetes. 2004 Mar;53(3):521-7. doi: 10.2337/diabetes.53.3.521.
Normand-Lauziere F, Frisch F, Labbe SM, Bherer P, Gagnon R, Cunnane SC, Carpentier AC. Increased postprandial nonesterified fatty acid appearance and oxidation in type 2 diabetes is not fully established in offspring of diabetic subjects. PLoS One. 2010 Jun 4;5(6):e10956. doi: 10.1371/journal.pone.0010956.
Rip J, Nierman MC, Ross CJ, Jukema JW, Hayden MR, Kastelein JJ, Stroes ES, Kuivenhoven JA. Lipoprotein lipase S447X: a naturally occurring gain-of-function mutation. Arterioscler Thromb Vasc Biol. 2006 Jun;26(6):1236-45. doi: 10.1161/01.ATV.0000219283.10832.43. Epub 2006 Mar 30.
Rip J, Nierman MC, Sierts JA, Petersen W, Van den Oever K, Van Raalte D, Ross CJ, Hayden MR, Bakker AC, Dijkhuizen P, Hermens WT, Twisk J, Stroes E, Kastelein JJ, Kuivenhoven JA, Meulenberg JM. Gene therapy for lipoprotein lipase deficiency: working toward clinical application. Hum Gene Ther. 2005 Nov;16(11):1276-86. doi: 10.1089/hum.2005.16.1276.
Ross CJ, Twisk J, Bakker AC, Miao F, Verbart D, Rip J, Godbey T, Dijkhuizen P, Hermens WT, Kastelein JJ, Kuivenhoven JA, Meulenberg JM, Hayden MR. Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum Gene Ther. 2006 May;17(5):487-99. doi: 10.1089/hum.2006.17.487.
Santamarina-Fojo S. The familial chylomicronemia syndrome. Endocrinol Metab Clin North Am. 1998 Sep;27(3):551-67, viii. doi: 10.1016/s0889-8529(05)70025-6.
Wittrup HH, Tybjaerg-Hansen A, Nordestgaard BG. Lipoprotein lipase mutations, plasma lipids and lipoproteins, and risk of ischemic heart disease. A meta-analysis. Circulation. 1999 Jun 8;99(22):2901-7. doi: 10.1161/01.cir.99.22.2901.
Study Completion
September 2012
Actual (reported)Last Updated
March 30, 2015
Record last verified: 2015-03
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