Clinical trial NCT02904772: Alipogene Tiparvovec for the Treatment of LPLD Patients
Official title: An Open Label, Multi-centre Trial of Alipogene Tiparvovec for the Treatment of LPLD Patients
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: LPL Deficiency
Lead sponsor: UniQure Biopharma B.V.
Sponsor class: INDUSTRY
Collaborators: name: Chiesi Farmaceutici S.p.A.
First posted: 2016-09-19
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Eligibility 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.

Primary outcomes:
measure: The Clinical Response of alipogene tiparvovec in LPLD patients
timeFrame: 2 years
measure: The long term effect of alipogene tiparvovec on post prandial metabolism of chylomicrons (ppCM) in LPLD patients.
timeFrame: 2 years
Secondary outcomes:
measure: The effect of alipogene tiparvovec on postprandial metabolism of chylomicrons (ppCM) in LPLD patients with and without immunosuppression treatment, at 14 weeks post-administration.
timeFrame: Baseline, 14 weeks
measure: Immuno response of alipogene tiparvovec by analysis of antibody formation
timeFrame: Baseline, 1 and 2 years post dose
measure: Immuno response of alipogene tiparvovec by analysis of T-cell response
timeFrame: Baseline, 1 and 2 years post dose
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Perelman School of Medicine at The University of Pennsylvania Translational Medicine & Human Genetics
state: Pennsylvania
city: Sherbrooke
country: Canada
facility: Centre hospitalier universitaire de Sherbrooke
state: Quebec
Study officials:
affiliation: Centre de recherche du Centre hospitalier universitaire de Sherbrooke
name: André Carpentier, MD
role: PRINCIPAL_INVESTIGATOR
References: