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
Sponsor study identifier
interventional
5
1 country
2
Study Start
February 2009
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
August 2010
Actual (reported)Study Completion
April 2011
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
April 2011
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
February 2009
Date type not reportedThis date has not been verified as actual or estimated.
April 30, 2009
Actual (reported)May 1, 2009
Actual (reported)August 2010
Actual (reported)April 2011
Actual (reported)Reduction of triglyceride (TG) concentrations
12 weeks
Reduction of chylomicrons and/or chylomicron-TG ratio
12 weeks
To determine the biological activity and expression of the lipoprotein lipase [LPLS447X] transgene product
14 weeks
To assess the safety profile
14 weeks
To assess shedding of viral vector
14 weeks
Gene Therapy
intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections
oral, 2 g/day, day -3 till week 12
ECOGENE-21 Clinical Trial Center / Centre de santé et de services sociaux de Chicoutimi
Chicoutimi, Quebec, G7H 5H6, Canada
Centre des Maladies Lipidiques de Québec
Québec, Quebec, G1V 4M6, Canada
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, Meulenberg JM, Liu G, van den Oever K, Moraal E, Hermens WT, Rip J, Kastelein JJ, Kuivenhoven JA, Hayden MR. Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation. Hum Gene Ther. 2004 Sep;15(9):906-19. doi: 10.1089/hum.2004.15.906.
Daniel Gaudet, MD, Ph.D.
ECOGENE-21 Clinical Trial Center / Centre de santé et de services sociaux de Chicoutimi, Chicoutimi Hospital, Quebec, Canada
5 participants (actual enrollment reported by the registry).
First Submitted
April 30, 2009
First Posted
May 1, 2009
Study Start
February 2009
Date type not reportedThis date has not been verified as actual or estimated.
oral, 3 mg/kg/day, day -3 till week 12
single intravenous bolus of methylprednisolone (1 mg/kg bodyweight)
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Contact the study team to confirm eligibility.
Ross CJ, Liu G, Kuivenhoven JA, Twisk J, Rip J, van Dop W, Excoffon KJ, Lewis SM, Kastelein JJ, Hayden MR. Complete rescue of lipoprotein lipase-deficient mice by somatic gene transfer of the naturally occurring LPLS447X beneficial mutation. Arterioscler Thromb Vasc Biol. 2005 Oct;25(10):2143-50. doi: 10.1161/01.ATV.0000176971.27302.b0. Epub 2005 Jul 7.
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.
Stroes ES, Nierman MC, Meulenberg JJ, Franssen R, Twisk J, Henny CP, Maas MM, Zwinderman AH, Ross C, Aronica E, High KA, Levi MM, Hayden MR, Kastelein JJ, Kuivenhoven JA. Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol. 2008 Dec;28(12):2303-4. doi: 10.1161/ATVBAHA.108.175620. Epub 2008 Sep 18. No abstract available.
Carpentier AC, Frisch F, Labbe SM, Gagnon R, de Wal J, Greentree S, Petry H, Twisk J, Brisson D, Gaudet D. Effect of alipogene tiparvovec (AAV1-LPL(S447X)) on postprandial chylomicron metabolism in lipoprotein lipase-deficient patients. J Clin Endocrinol Metab. 2012 May;97(5):1635-44. doi: 10.1210/jc.2011-3002. Epub 2012 Mar 21.
Primary Completion
August 2010
Actual (reported)Study Completion
April 2011
Actual (reported)Last Updated
September 29, 2011
Record last verified: 2011-09
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Source: ClinicalTrials.gov.
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