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Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects

NCT00891306 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2011-09-29

No results posted yet for this study

Summary

This trial is designed to expand the currently available data on the safety and efficacy of alipogene tiparvovec treatment in lipoprotein lipase deficiency (LPLD) and to further the understanding of possible mechanisms of action of the therapy.

Conditions

  • Familial Lipoprotein Lipase Deficiency

Interventions

GENETIC

Alipogene Tiparvovec (AMT-011), Human LPL [S447X]

intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections

DRUG

mycophenolate mofetil

oral, 2 g/day, day -3 till week 12

DRUG

cyclosporine

oral, 3 mg/kg/day, day -3 till week 12

DRUG

methylprednisolone

single intravenous bolus of methylprednisolone (1 mg/kg bodyweight)

Sponsors & Collaborators

  • The Clinical Trial Company

    collaborator INDUSTRY

  • Amsterdam Molecular Therapeutics

    lead INDUSTRY

Principal Investigators

  • Daniel Gaudet, MD, Ph.D. · ECOGENE-21 Clinical Trial Center / Centre de santé et de services sociaux de Chicoutimi, Chicoutimi Hospital, Quebec, Canada

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-02-28
Primary Completion
2010-08-31
Completion
2011-04-30

Countries

  • 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 NCT00891306 on ClinicalTrials.gov