Alipogene Tiparvovec for the Treatment of LPLD Patients

UniQure

Status and phase: Withdrawn; Phase 2

Conditions: LPL Deficiency

Treatments: Drug: alipogene tiparvovec; Drug: Mycophenolate mofetil; Drug: Prednisolone; Drug: Cyclosporins

Study type: Interventional

Funder types: Industry

Identifiers

NCT02904772

Gly-CD-001

Details and patient eligibility

About

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.

Full 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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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0 participants in 2 patient groups

alipogene tiparvovec with IS

Other group

Description:

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.

Treatment:

Drug: Cyclosporins

Drug: Prednisolone

Drug: Mycophenolate mofetil

Drug: alipogene tiparvovec

alipogene tiparvovec without IS

Other group

Description:

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.

Treatment:

Drug: Prednisolone

Drug: alipogene tiparvovec