Alipogene Tiparvovec for the Treatment of LPLD Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-31

Study Completion Date

2020-09-30

Brief Summary

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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.

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Detailed Description

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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

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LPL Deficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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alipogene tiparvovec with IS

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.

Group Type OTHER

alipogene tiparvovec

Intervention Type DRUG

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.

Prednisolone

Intervention Type DRUG

IV bolus methylprednisolone 1mg/kg half hour prior to administration

Cyclosporins

Intervention Type DRUG

Immuno + group will receive cyclosporine (3 mg/kg/day) from three days prior to until 12 weeks following IMP administration

Mycophenolate mofetil

Intervention Type DRUG

Immuno + group will receive Mycophenolate mofetil (2x 1 g/day) from three days prior to until 12 weeks following IMP administration

alipogene tiparvovec without IS

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.

Group Type OTHER

alipogene tiparvovec

Intervention Type DRUG

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.

Prednisolone

Intervention Type DRUG

IV bolus methylprednisolone 1mg/kg half hour prior to administration

Interventions

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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.

Intervention Type DRUG

Prednisolone

IV bolus methylprednisolone 1mg/kg half hour prior to administration

Intervention Type DRUG

Cyclosporins

Immuno + group will receive cyclosporine (3 mg/kg/day) from three days prior to until 12 weeks following IMP administration

Intervention Type DRUG

Mycophenolate mofetil

Immuno + group will receive Mycophenolate mofetil (2x 1 g/day) from three days prior to until 12 weeks following IMP administration

Intervention Type DRUG

Other Intervention Names

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Glybera

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No