A Phase 2/3 interventional study of Alipogene Tiparvovec (AMT-011), Human LPL [S447X] and Mycophenolate mofetil in Familial Lipoprotein Lipase Deficiency, sponsored by Amsterdam Molecular Therapeutics. Status unknown at 1 site in Canada. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2011-09-30.
Sponsored by Amsterdam Molecular Therapeutics · Phase 2/3, Interventional, and Treatment
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.
The CT-AMT-011-01 study is an open-label, dose-escalating study evaluating the safety and efficacy of a single intramuscular administration of AMT-011 (at multiple sites). The study will be performed in the Community Genomic medicineCenter (CGMC) Chicoutimi, Canada, under the supervision of their medical ethical committee and according to the local biosafety procedures. The study participants will be treated under the responsibility of a Principal Investigator specialised in the treatment of lipid disorders. A total number of 14 subjects will be administered. Participants will be screened 3 weeks prior to administration of AMT-011 and will be evaluated for 12 weeks post administration in this study. After the study, subjects will be followed up long term with particular emphasis on the safety and efficacy aspects of LPL gene therapy using AMT-011. Subjects will be evaluated at the clinical site at 19 weeks, 26 weeks, 39 weeks, 1 year, 1.5 years, 2 years, 3 years, 4 years and 5 years after administration of AMT-011. The TG values that are obtained at week 26 will be used for secondary efficacy analysis.
25 studies on the registry are indexed under Hyperlipoproteinemia Type I; 3 are open to participants now.
This study's enrollment of 14 is below the median of 16 across 13 interventional studies indexed under Hyperlipoproteinemia Type I.
Browse Hyperlipoproteinemia Type I studies →Amsterdam Molecular Therapeutics is the lead sponsor of 4 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
The following blood screening tests will result in exclusion from participation:
intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections
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 with immunosuppressants
Drug: Mycophenolate mofetil · Genetic: Alipogene Tiparvovec (AMT-011), Human LPL [S447X] · Drug: cyclosporine
intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections with immunosuppressants
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
Also known as: Glybera, AMT-011
oral, 2 g/day, day -3 till week 12
Also known as: CellCept
intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections
Also known as: Glybera, AMT 011
oral, 3 mg/kg/day, day -3 till week 12
Also known as: Neoral
Reduction of fasting triglyceride (TG) concentrations
To achieve a reduction in fasting plasma TG such that the difference in median plasma TG observed before administration and up to 12 weeks after administration represents approximately 40% reduction, on top of a low-fat diet.
Time frame: 12 weeks
safety profile of AMT-011
Between 6 and 15 years after administration of AMT-011, patients will be annually contacted by phone to monitor delayed adverse events related to administration of AMT-011.
Time frame: 15 years
Reduction of TG concentrations
To achieve sustained efficacy, defined as approximately 40% reduction in fasting plasma TG up to 26 weeks after administration, on top of a low-fat diet.
Time frame: 26 weeks
Reduction of TG concentrations
To achieve a reduction in fasting plasma TG to a level equal to or less than 10.00 mmol/L on top of a low-fat diet at 12 weeks after administration.
Time frame: 12 weeks
Reduction of TG concentrations
To achieve sustained efficacy, defined as a reduction in fasting plasma TG at 26 weeks after administration to a level equal to or less than 10.00 mmol/L on top of a low-fat diet.
Time frame: 26 weeks
Biological activity and expression of the transgene product.
To determine the biological activity and expression of the lipoprotein lipase (LPLS447X) transgene product.
Time frame: 1 year
Evaluation immune respons
To evaluate potential immune responses against the lipoprotein lipase (LPLS447X) transgene product and the adeno-associated viral (AAV) vector.
Time frame: 5 years
To assess the shedding of the viral vector
To assess shedding of AMT-011
Time frame: 1 year
The record gives a start of Aug 2007, a primary completion of Jun 2013 and a completion of Jun 2013. It does not say whether these dates are actual or estimated.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Apr 2010. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drugs tested here: Mycophenolic Acid · Cyclosporine
Amsterdam Molecular Therapeutics