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Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X]

NCT01109498 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2011-09-30

No results posted yet for this study

Summary

LPLD is a rare autosomal recessive disorder, characterized by the presence of marked chylomicronemia and hence hypertriglyceridemia. Clinically the most severe manifestation of chylomicronemia, is acute pancreatitis, which can be lethal. There is no effective therapy available to modulate the course of the illness and prevent complications for these patients. The current clinical management consists of severe reduction of dietary fat that is hard if not almost impossible to comply with. LPLD subjects continue to experience pancreatitis attacks, and are admitted to intensive care units on several occasions.

Alipogene tiparvovec corrects or restores lipoprotein lipase (LPL) function long term, and hence reverses some symptoms, halts the disease progression and prevents further complications. Alipogene tiparvovec gene therapy ensures that a catabolically beneficial variant of the human LPL gene, LPL\[S447X\] is expressed and active in the relevant tissues in humans. Delivery of the gene is realized via intramuscular injection of an adeno-associated viral vector, pseudotyped with AAV1 capsids.

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

GENETIC

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

intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections

DRUG

cyclosporine

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

Sponsors & Collaborators

  • International Antiviral Therapy Evaluation Center

    collaborator OTHER

  • Amsterdam Molecular Therapeutics

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-08-31
Primary Completion
2013-06-30
Completion
2013-06-30

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 NCT01109498 on ClinicalTrials.gov