Alipogene Tiparvovec for the Treatment of LPLD Patients

• Status: Withdrawn
• Trial end date: 2020-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: UniQure Biopharma B.V.
• Collaborator: Chiesi Farmaceutici S.p.A.
• Treatments: Cyclosporine; Cyclosporins; Methylprednisolone; Methylprednisolone acetate; Methylprednisolone Hemisuccinate; Mycophenolate mofetil; Mycophenolic Acid; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate

Criteria

Main inclusion criteria are:

• Patients with a history of severe or multiple pancreatitis attacks despite dietary fat restriction.

• Genetically confirmed diagnosis of LPLD

• Post-heparin plasma LPL protein mass > 5% of normal

• LPL activity ≤20% of normal (in post- heparin plasma)

• Fasting plasma TG concentration >10 mmol/L.

Main exclusion criteria are:

• Females with a positive pregnancy test or who are breastfeeding, or on contraceptive use.

• Patients with a positive HIV, Hepatitis B, Hepatitis C or being positive for tuberculosis.

• Patients under treatment with antiplatelet or other anti-coagulants.

• Patient allergic to or having a condition that prohibits the use of immunosuppressants.