A Phase 2 interventional study of alipogene tiparvovec and Prednisolone in LPL Deficiency, sponsored by UniQure Biopharma B.V.. Withdrawn at 2 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2017-08-17.
Sponsored by UniQure Biopharma B.V. · Phase 2, Interventional, and Treatment
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.
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.
23 studies on the registry are indexed under Hyperlipoproteinemia Type I; 3 are open to participants now.
Browse Hyperlipoproteinemia Type I studies →UniQure Biopharma B.V. is the lead sponsor of 7 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Main inclusion criteria are:
Main exclusion criteria are:
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.
Drug: alipogene tiparvovec · Drug: Prednisolone · Drug: Cyclosporins · Drug: Mycophenolate mofetil
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.
Drug: alipogene tiparvovec · Drug: Prednisolone
A dose of 1x10(\*12) gc/kg alipogene tiparvovec (Glybera) of body weight administered as a single set of intramuscular injections at multiple sites in multiple muscles of both upper legs and if necessary, the lower legs.
Also known as: Glybera
IV bolus methylprednisolone 1mg/kg half hour prior to administration
Immuno + group will receive cyclosporine (3 mg/kg/day) from three days prior to until 12 weeks following IMP administration
Immuno + group will receive Mycophenolate mofetil (2x 1 g/day) from three days prior to until 12 weeks following IMP administration
The Clinical Response of alipogene tiparvovec in LPLD patients
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.
Time frame: 2 years
The long term effect of alipogene tiparvovec on post prandial metabolism of chylomicrons (ppCM) in LPLD patients.
CM \[3H\]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.
Time frame: 2 years
The effect of alipogene tiparvovec on postprandial metabolism of chylomicrons (ppCM) in LPLD patients with and without immunosuppression treatment, at 14 weeks post-administration.
CM \[3H\]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.
Time frame: Baseline, 14 weeks
Immuno response of alipogene tiparvovec by analysis of antibody formation
The immuno response of alipogene tiparvovec will be assessed by measuring the antibody formation compared to baseline.
Time frame: Baseline, 1 and 2 years post dose
Immuno response of alipogene tiparvovec by analysis of T-cell response
T-cell responses against alipogene tiparvovec will be assessed by measuring the T-cell response compared to baseline.
Time frame: Baseline, 1 and 2 years post dose
Plan to share: No
No publications or documents are linked to this record.
This study is withdrawn, as verified in Jul 2017. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
UniQure Biopharma B.V.