Last updated on October 2016

Alipogene Tiparvovec for the Treatment of LPLD Patients


Brief description of study

The aim of the study is to provide further confirmatory evidence of clinical benefit in LPLD patients treated with alipogene tiparvovec by assessing both the "clinical response" (as defined by a range of parameters), and "the metabolic response" (postprandial CM metabolism) in LPLD patients with and without an immunosuppressant regimen.

Detailed Study Description

This is a prospective, interventional, randomised, open-label, parallel group study evaluating the clinical response as well as the dynamics of postprandial chylomicron metabolism in patients treated with alipogene tiparvovec with and without immunosuppressants. The study will be conducted in 12 LPLD patients who will be randomised into the Immuno+ (cyclosporin and mycophenolate mofetil) or the Immuno- group.

Clinical Study Identifier: NCT02904772

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Richard L Dunbar, MD

Perelman School of Medicine at The University of Pennsylvania Translational Medicine & Human Genetics
Philadelphia, PA United States
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André Carpentier, MD, FRCPC

Centre hospitalier universitaire de Sherbrooke
Sherbrooke, QC Canada
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