Clinical Trial: Alipogene Tiparvovec for the Treatment of LPLD Patients

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open Label, Multi-centre Trial of Alipogene Tiparvovec for the Treatment of LPLD Patients

Brief Summary: 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 Summary: 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.
Sponsor: UniQure Biopharma B.V.

Current Primary Outcome:

  • The Clinical Response of alipogene tiparvovec in LPLD patients [ Time Frame: 2 years ]
    The overall clinical response of alipogene tiparvovec in LPLD patients will be assessed compared to baseline, by a combination of measurements, of which each gives relevant information to obtain enough and solid evidence in a small trial. Each of these outcome measures will be evaluated in combination with the results of other measures (to get an overall conclusion relating the clinical response). Descriptive methods will be used (so no formal statistical analyses will be performed), due to the specific nature and the small sample size of a rare disease trial.
  • The long term effect of alipogene tiparvovec on post prandial metabolism of chylomicrons (ppCM) in LPLD patients. [ Time Frame: 2 years ]
    CM [3H]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • The effect of alipogene tiparvovec on postprandial metabolism of chylomicrons (ppCM) in LPLD patients with and without immunosuppression treatment, at 14 weeks post-administration. [ Time Frame: Baseline, 14 weeks ]
    CM [3H]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.
  • Immuno response of alipogene tiparvovec by analysis of antibody formation [ Time Frame: Baseline, 1 and 2 years post dose ]
    The immuno response of alipogene tiparvovec will be assessed by measuring the antibody formation compared to baseline.
  • Immuno response of alipogene tiparvovec by analysis of T-cell response [ Time Frame: Baseline, 1 and 2 years post dose ]
    T-cell responses against alipogene tiparvovec will be assessed by measuring the T-cell response compared to baseline.


Original Secondary Outcome: Same as current

Information By: UniQure Biopharma B.V.

Dates:
Date Received: May 10, 2016
Date Started: October 2016
Date Completion: September 2020
Last Updated: October 14, 2016
Last Verified: October 2016