NCT02904772

Alipogene Tiparvovec for the Treatment of LPLD Patients

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

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: UniQure Biopharma B.V. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• LPL Deficiency

Interventions

Type	Name	Description
DRUG	alipogene tiparvovec	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.
DRUG	Prednisolone	IV bolus methylprednisolone 1mg/kg half hour prior to administration
DRUG	Cyclosporins	Immuno + group will receive cyclosporine (3 mg/kg/day) from three days prior to until 12 weeks following IMP administration
DRUG	Mycophenolate mofetil	Immuno + group will receive Mycophenolate mofetil (2x 1 g/day) from three days prior to until 12 weeks following IMP administration

Timeline

• Start date: 2016-10-01
• Primary completion: 2020-06-01
• Completion: 2020-09-01
• First posted: 2016-09-19
• Last updated: 2017-08-17

Locations

2 sites across 2 countries: United States, Canada