Alipogene Tiparvovec for the Treatment of LPLD Patients
- Conditions
- LPL Deficiency
- Interventions
- Registration Number
- NCT02904772
- Lead Sponsor
- UniQure Biopharma B.V.
- 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 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.
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description alipogene tiparvovec with IS alipogene tiparvovec Patients in the Immuno+ group will receive an immunosuppressant regimen to be initiated three days prior to alipogene tiparvovec administration. The regimen is to be continued for 12 weeks: Cyclosporins (3 mg/kg/day) and mycophenolate mofetil (2 x 1 g/day). Patients will receive IV bolus of 1mg/kg of methyl Prednisolone half an hour prior to IMP administration. alipogene tiparvovec with IS Prednisolone Patients in the Immuno+ group will receive an immunosuppressant regimen to be initiated three days prior to alipogene tiparvovec administration. The regimen is to be continued for 12 weeks: Cyclosporins (3 mg/kg/day) and mycophenolate mofetil (2 x 1 g/day). Patients will receive IV bolus of 1mg/kg of methyl Prednisolone half an hour prior to IMP administration. alipogene tiparvovec with IS Cyclosporins Patients in the Immuno+ group will receive an immunosuppressant regimen to be initiated three days prior to alipogene tiparvovec administration. The regimen is to be continued for 12 weeks: Cyclosporins (3 mg/kg/day) and mycophenolate mofetil (2 x 1 g/day). Patients will receive IV bolus of 1mg/kg of methyl Prednisolone half an hour prior to IMP administration. alipogene tiparvovec without IS alipogene tiparvovec Patients in the Immuno- group will not receive an immunosuppressant regimen during 12 weeks. Patients will receive IV bolus of 1mg/kg of methyl Prednisolone half an hour prior to IMP administration. alipogene tiparvovec with IS Mycophenolate mofetil Patients in the Immuno+ group will receive an immunosuppressant regimen to be initiated three days prior to alipogene tiparvovec administration. The regimen is to be continued for 12 weeks: Cyclosporins (3 mg/kg/day) and mycophenolate mofetil (2 x 1 g/day). Patients will receive IV bolus of 1mg/kg of methyl Prednisolone half an hour prior to IMP administration. alipogene tiparvovec without IS Prednisolone Patients in the Immuno- group will not receive an immunosuppressant regimen during 12 weeks. Patients will receive IV bolus of 1mg/kg of methyl Prednisolone half an hour prior to IMP administration.
- Primary Outcome Measures
Name Time Method The Clinical Response of alipogene tiparvovec in LPLD patients 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. 2 years CM \[3H\]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.
- Secondary Outcome Measures
Name Time Method Immuno response of alipogene tiparvovec by analysis of antibody formation 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 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.
The effect of alipogene tiparvovec on postprandial metabolism of chylomicrons (ppCM) in LPLD patients with and without immunosuppression treatment, at 14 weeks post-administration. Baseline, 14 weeks CM \[3H\]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.
Trial Locations
- Locations (2)
Perelman School of Medicine at The University of Pennsylvania Translational Medicine & Human Genetics
🇺🇸Philadelphia, Pennsylvania, United States
Centre hospitalier universitaire de Sherbrooke
🇨🇦Sherbrooke, Quebec, Canada