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Alipogene Tiparvovec for the Treatment of LPLD Patients

NCT02904772 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2017-08-17

No results posted yet for this study

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.

Conditions

  • LPL Deficiency

Interventions

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

Sponsors & Collaborators

  • Chiesi Farmaceutici S.p.A.

    collaborator INDUSTRY

  • UniQure Biopharma B.V.

    lead INDUSTRY

Principal Investigators

  • André Carpentier, MD · Centre de recherche du Centre hospitalier universitaire de Sherbrooke

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-10-31
Primary Completion
2020-06-30
Completion
2020-09-30

Countries

  • United States
  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02904772 on ClinicalTrials.gov