Status:
TERMINATED
Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
Lead Sponsor:
Amsterdam Molecular Therapeutics
Collaborating Sponsors:
ICON plc
Conditions:
Hyperlipoproteinemia Type I
Eligibility:
All Genders
18-70 years
Brief Summary
LPL (Lipoprotein Lipase) is an enzyme which plays an important role in the elimination of triglycerides (fat) and the clearance of dietary fat particles known as chylomicrons (CM) in the blood. In pat...
Detailed Description
Lipoprotein lipase deficiency (LPLD) is a rare autosomal recessive inherited disorder caused by loss-of-function mutations in the lipoprotein lipase (LPL) gene. It is the most common genetic cause of ...
Eligibility Criteria
Inclusion
- Has provided signed informed consent.
- Male or female aged 18 to 70 years, inclusive at the time of consent.
- Females of childbearing potential must have a negative serum pregnancy test and agree to use a medically acceptable effective form of birth control from screening through Day 21 visit.
- Subjects in Cohort 1 (previously treated LPLD Cohort) must have received AMT-011 during Studies CT-AMT-011-01 or -02 as verified by site personnel.
- Subjects in Cohort 2 (untreated LPLD control Cohort)) may have completed study PREPARATION-02 as verified by site personnel or known patients with genetically confirmed LPLD.
- Volunteers in Cohort 3 (normal healthy control Cohort) must not have LPLD.
- Subjects must be in good general physical health with, in the opinion of the investigator, no other clinically significant and relevant abnormalities of medical history, and no abnormalities at the physical examination and routine laboratory evaluation performed prior to the study.
- Must be able to communicate fully and effectively with the study personnel.
Exclusion
- Female subjects who have a positive serum pregnancy test or who are nursing.
- Known allergy to any of the constituents of the radiolabeled meal/radio labeled agent, or a history of severe allergic or anaphylactic reactions.
- Investigator-determined clinically significant disease (other than LPLD for those subjects with LPLD), that would affect the subject's participation in the study.
- Healthy Volunteers with a history or presence of neurological, haematological, psychiatric, gastrointestinal, pulmonary, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including plasma lipids out side normal range for age and gender and a body mass index (BMI) \>30.
- Any current or relevant previous history of serious, severe, or unstable physical or psychiatric illness, any medical disorder that may make the participant unlikely to fully complete the study, or any condition that presents undue risk from the study medication or procedures.
- A laboratory value at screening outside the normal range unless it is judged by the investigator as not clinically significant after appropriate evaluation.
- Clinically significant ECG at screening as determined by the investigator.
- Blood donations (≥1 unit) during the 2 months preceding and following the study or other significant blood loss.
- Other unspecified reasons that, in the opinion of the investigator or sponsor, make the subject unsuitable for enrolment.
- Any individual involved in the planning or conduct of this study. -
Key Trial Info
Start Date :
September 1 2011
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 1 2012
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT01447901
Start Date
September 1 2011
End Date
September 1 2012
Last Update
March 30 2015
Active Locations (1)
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1
ECOGENE-21 Clinical Trial Center
Chicoutimi, Quebec, Canada, G7H 7P2