Hyperlipoproteinemia Type INCT01447901
Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
An observational study of Hyperlipoproteinemia Type I, run by Amsterdam Molecular Therapeutics at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Hyperlipoproteinemia Type I
- How many people
- 19 participants
- Where
- 1 siteCanada
- Study dates
- Sep 2011to Sep 2012
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: * Lipoprotein Lipase Deficiency (LPLD) Patients * Healthy volunteers (community sample)
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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. -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prospective, Non-interventional, Non-randomised, Open-label, Adult Study to Assess the Long Term Biological Therapeutic Response to Alipogene Tiparvovec in Lipoprotein Lipase Deficiency (LPLD) and Comparing Postprandial Chylomicron Metabolism Following a Radiolabeled Meal in LPLD Subjects Previously Treated With Alipogene Tiparvovec (Studies CT-AMT-011-01 or -02) to Untreated LPLD Subjects (Study PREPARATION-02) and to Healthy Volunteers
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 patients who have an abnormal LPL gene, the enzyme does not work (total, hereditary LPL deficiency), which results in a large increase in the amount of triglycerides (fats) and chylomicrons in the blood. This increases the risk of inflammation in the pancreas and leads to long term negative effects for bloods vessels (atherosclerosis). Current medications and / or a strict and low fat diet do not sufficiently reduce the level of triglycerides in order to prevent these conditions. To solve this problem, the company, AMT is developing a gene therapy (AMT-011).
In normal healthy individuals, fat particles are rapidly cleared from the circulation following a standard meal. Within approximately 3 hours the highest levels of fat is reached and clearance is achieved within the subsequent 9 hours. In LPLD subjects, the clearance of fat is greatly reduced as a direct consequence of the lack of LPL. During this study, a standard meal with a tracer (3H-palmitate) is given. Since palmitate is incorporated in the dietary fat, this study enabled monitoring of appearance of newly formed dietary fat into- and clearance of these newly formed dietary fats from the circulation, over time.
The principal aim of the study is to verify if the gene therapy (AMT 011) is still effective in the treatment of this condition. Systemic appearance and clearance of new formed dietary fat particles after ingestion of the meal will be determined by measuring the level of tracer at different time points.
Read the 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 hyperchylomicronaemia, a condition which results in continuous and excessively high levels of plasma chylomicrons (CM) and severe hypertriglyceridaemia. Lipoprotein lipase normally mediates the hydrolysis of triglycerides (TG) in CMs and very low-density lipoproteins (VLDL) and thereby aids in the clearance of TG-rich lipoproteins and reduction of TGs in the circulation.
Alipogene tiparvovec (Glybera®) is in development for the therapy of LPLD. In summary, alipogene tiparvovec contains the human lipoprotein (LPL) gene variant LPLS447X in a non-replicating vector in solution administered in a one-time series of intramuscular injections in the arms/legs.The aim of alipogene tiparvovec (Glybera®) administration is to provide LPL activity and to stimulate CM metabolism in LPLD patients.
To test the activity of LPL in subjects previously treated with alipogene tiparvovec in this study LPLD subjects will be given a radiolabeled meal supplemented with a labeled tracer, 3H-palmitate. Since dietary palmitate is incorporated into CMs as they are formed in the enterocytes of the gut, this enables monitoring of the appearance and subsequent clearance of newly formed CMs from the circulation over time, the so-called "postprandial test". The radiolabeled meal will be provided in a liquid form similar to a milkshake. After ingestion of the radiolabeled meal, level of radiolabel in the CM fraction at different time points prior to and during the postprandial phase will be measured and thus determine the appearance and clearance of CMs within the circulation.
The principal aim of the study is to increase the understanding of how long alipogene tiparvovec may be effective in the treatment of LPLD. To understand this, 3 cohorts of subjects will be studied: 1) Subjects with LPLD who have previously been treated with alipogene tiparvovec; 2) Subjects with LPLD who have not been treated with alipogene tiparvovec; and 3) Subjects who do not have LPLD (healthy volunteers). The subject's general state of health will also be monitored during the clinical study, and the possible disadvantages associated with the postprandial test will be assessed.
- Conditions
- Hyperlipoproteinemia Type I
- Keywords
- Hyperlipoproteinemia Type I
- Medical subject headings
- Hyperlipoproteinemia Type I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| previously treated LPLD Cohort | Subjects in Cohort 1 (previously treated LPLD Cohort) must have received AMT-011 during Studies CT-AMT-011-01 or -02 | |
| untreated LPLD control Cohort | Subjects in Cohort 2 (untreated LPLD control Cohort)) may have completed study PREPARATION-02 or known patients with genetically confirmed LPLD | |
| normal healthy control Cohort | Volunteers in Cohort 3 (normal healthy control Cohort) must not have LPLD |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite of Pharmacodynamics
Peak level, time-to-peak, and area under the curve (AUC) for tracer in plasma and chylomicron (CM) fraction to assess metabolism of newly-formed CMs in LPLD subjects previously treated with alipogene tiparvovec and to compare with untreated LPLD subjects and healthy controls.
Measured: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose
Secondary outcomes (9)
Triglyceride (TG)-rich lipoproteins
Surface and core components of TG-rich lipoproteins (TG, TChol, apoB100, and apoB48)in plasma and in the CM fraction will be measured.
Measured: pre dose, 0, 1, 2, 3, 4, 4, 6, 7, 8, 9, 12, 24 hours post dose
Glucose
The levels of glucose in plasma.
Measured: pre dose, 0, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose
Adverse Events (AE)
Monitoring general state of health of subjects and to assess the safety of ingestion of a radiolabeled meal, containing the radiolabel 3H palmitate through review of the incidence and severity of adverse events (AEs).
Measured: Up to 21 days. Serious AEs will be followed to their resolution.
Laboratory tests
Monitoring general state of health in LPLD subjects previously treated with alipogene tiparvovec compared to untreated LPLD subjects and healthy volunteers through review of laboratory findings (haematology, clinical chemistry, and urinalysis).
Measured: Up to 21 days
Vital signs
To monitor the general state of health in LPLD subjects previously treated with alipogene tiparvovec compared to untreated LPLD subjects and healthy volunteers through review of the vital signs.
Measured: Up to 21 days
Physical examination
To monitor the general state of health in LPLD subjects previously treated with alipogene tiparvovec compared to untreated LPLD subjects and healthy volunteers through review of physical examination findings.
Measured: Up to 21 days
ECG
To monitor the general state of health in LPLD subjects previously treated with alipogene tiparvovec compared to untreated LPLD subjects and healthy volunteers through review of 12-lead electrocardiograms (ECGs).
Measured: Up to 21 days
C-peptide
Levels of C-peptide in plasma
Measured: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 post dose
Non Esterified Fatty Acids (NEFA)
Levels of NEFA in plasma will be measured
Measured: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2011 (estimated)
- Study startSep 2011
- Primary completionSep 2012
- Study completionSep 2012
Why it stopped: Business reason
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for Hyperlipoproteinemia Type IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 19 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Amsterdam Molecular Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
- Investigators
- Daniel Gaudet, MD PhD, Principal investigator, ECOGENE-21 Clinical Trial Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01447901
- Sponsor's protocol ID
- CT-AMT-011-04
- Status last verified
- Mar 2015
- First submitted
- 28 Sep 2011
- Last updated
- 30 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Bickerton AS, Roberts R, Fielding BA, Hodson L, Blaak EE, Wagenmakers AJ, Gilbert M, Karpe F, Frayn KN. Preferential uptake of dietary Fatty acids in adipose tissue and muscle in the postprandial period. Diabetes. 2007 Jan;56(1):168-76. doi: 10.2337/db06-0822.
PubMed 17192479Black DM, Sprecher DL. Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat. Am J Dis Child. 1993 Jan;147(1):60-2. doi: 10.1001/archpedi.1993.02160250062018.
PubMed 8418601Chait A, Brunzell JD. Chylomicronemia syndrome. Adv Intern Med. 1992;37:249-73.
PubMed 1557997Fortson MR, Freedman SN, Webster PD 3rd. Clinical assessment of hyperlipidemic pancreatitis. Am J Gastroenterol. 1995 Dec;90(12):2134-9.
PubMed 8540502Miles JM, Park YS, Walewicz D, Russell-Lopez C, Windsor S, Isley WL, Coppack SW, Harris WS. Systemic and forearm triglyceride metabolism: fate of lipoprotein lipase-generated glycerol and free fatty acids. Diabetes. 2004 Mar;53(3):521-7. doi: 10.2337/diabetes.53.3.521.
PubMed 14988233Normand-Lauziere F, Frisch F, Labbe SM, Bherer P, Gagnon R, Cunnane SC, Carpentier AC. Increased postprandial nonesterified fatty acid appearance and oxidation in type 2 diabetes is not fully established in offspring of diabetic subjects. PLoS One. 2010 Jun 4;5(6):e10956. doi: 10.1371/journal.pone.0010956.
PubMed 20532041Rip J, Nierman MC, Ross CJ, Jukema JW, Hayden MR, Kastelein JJ, Stroes ES, Kuivenhoven JA. Lipoprotein lipase S447X: a naturally occurring gain-of-function mutation. Arterioscler Thromb Vasc Biol. 2006 Jun;26(6):1236-45. doi: 10.1161/01.ATV.0000219283.10832.43. Epub 2006 Mar 30.
PubMed 16574898Rip J, Nierman MC, Sierts JA, Petersen W, Van den Oever K, Van Raalte D, Ross CJ, Hayden MR, Bakker AC, Dijkhuizen P, Hermens WT, Twisk J, Stroes E, Kastelein JJ, Kuivenhoven JA, Meulenberg JM. Gene therapy for lipoprotein lipase deficiency: working toward clinical application. Hum Gene Ther. 2005 Nov;16(11):1276-86. doi: 10.1089/hum.2005.16.1276.
PubMed 16259561Ross CJ, Twisk J, Bakker AC, Miao F, Verbart D, Rip J, Godbey T, Dijkhuizen P, Hermens WT, Kastelein JJ, Kuivenhoven JA, Meulenberg JM, Hayden MR. Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum Gene Ther. 2006 May;17(5):487-99. doi: 10.1089/hum.2006.17.487.
PubMed 16716106Santamarina-Fojo S. The familial chylomicronemia syndrome. Endocrinol Metab Clin North Am. 1998 Sep;27(3):551-67, viii. doi: 10.1016/s0889-8529(05)70025-6.
PubMed 9785052Wittrup HH, Tybjaerg-Hansen A, Nordestgaard BG. Lipoprotein lipase mutations, plasma lipids and lipoproteins, and risk of ischemic heart disease. A meta-analysis. Circulation. 1999 Jun 8;99(22):2901-7. doi: 10.1161/01.cir.99.22.2901.
PubMed 10359734
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Non-interventional, Non-randomised, Open-label, Adult Study to Assess the Long Term Biological Therapeutic Response to Alipogene Tiparvovec in Lipoprotein Lipase Deficiency (LPLD) and Comparing Postprandial Chylomicron Metabolism Following a Radiolabeled Meal in LPLD Subjects Previously Treated With Alipogene Tiparvovec (Studies CT-AMT-011-01 or -02) to Untreated LPLD Subjects (Study PREPARATION-02) and to Healthy Volunteers
- ClinicalTrials.gov ID
- NCT01447901
- Acronym / sponsor ID
- CT-AMT-011-04
- Conditions
- Hyperlipoproteinemia Type I
- Keywords
- Hyperlipoproteinemia Type I
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Business reason
- Enrolment
- 19 participants (estimated)
- Lead sponsor
- Amsterdam Molecular TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- Sep 2011
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 6 Oct 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 patients who have an abnormal LPL gene, the enzyme does not work (total, hereditary LPL deficiency), which results in a large increase in the amount of triglycerides (fats) and chylomicrons in the blood. This increases the risk of inflammation in the pancreas and leads to long term negative effects for bloods vessels (atherosclerosis). Current medications and / or a strict and low fat diet do not sufficiently reduce the level of triglycerides in order to prevent these conditions. To solve this problem, the company, AMT is developing a gene therapy (AMT-011).
In normal healthy individuals, fat particles are rapidly cleared from the circulation following a standard meal. Within approximately 3 hours the highest levels of fat is reached and clearance is achieved within the subsequent 9 hours. In LPLD subjects, the clearance of fat is greatly reduced as a direct consequence of the lack of LPL. During this study, a standard meal with a tracer (3H-palmitate) is given. Since palmitate is incorporated in the dietary fat, this study enabled monitoring of appearance of newly formed dietary fat into- and clearance of these newly formed dietary fats from the circulation, over time.
The principal aim of the study is to verify if the gene therapy (AMT 011) is still effective in the treatment of this condition. Systemic appearance and clearance of new formed dietary fat particles after ingestion of the meal will be determined by measuring the level of tracer at different time points.
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 hyperchylomicronaemia, a condition which results in continuous and excessively high levels of plasma chylomicrons (CM) and severe hypertriglyceridaemia. Lipoprotein lipase normally mediates the hydrolysis of triglycerides (TG) in CMs and very low-density lipoproteins (VLDL) and thereby aids in the clearance of TG-rich lipoproteins and reduction of TGs in the circulation.
Alipogene tiparvovec (Glybera®) is in development for the therapy of LPLD. In summary, alipogene tiparvovec contains the human lipoprotein (LPL) gene variant LPLS447X in a non-replicating vector in solution administered in a one-time series of intramuscular injections in the arms/legs.The aim of alipogene tiparvovec (Glybera®) administration is to provide LPL activity and to stimulate CM metabolism in LPLD patients.
To test the activity of LPL in subjects previously treated with alipogene tiparvovec in this study LPLD subjects will be given a radiolabeled meal supplemented with a labeled tracer, 3H-palmitate. Since dietary palmitate is incorporated into CMs as they are formed in the enterocytes of the gut, this enables monitoring of the appearance and subsequent clearance of newly formed CMs from the circulation over time, the so-called "postprandial test". The radiolabeled meal will be provided in a liquid form similar to a milkshake. After ingestion of the radiolabeled meal, level of radiolabel in the CM fraction at different time points prior to and during the postprandial phase will be measured and thus determine the appearance and clearance of CMs within the circulation.
The principal aim of the study is to increase the understanding of how long alipogene tiparvovec may be effective in the treatment of LPLD. To understand this, 3 cohorts of subjects will be studied: 1) Subjects with LPLD who have previously been treated with alipogene tiparvovec; 2) Subjects with LPLD who have not been treated with alipogene tiparvovec; and 3) Subjects who do not have LPLD (healthy volunteers). The subject's general state of health will also be monitored during the clinical study, and the possible disadvantages associated with the postprandial test will be assessed.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| previously treated LPLD Cohort | – | –Subjects in Cohort 1 (previously treated LPLD Cohort) must have received AMT-011 during Studies CT-AMT-011-01 or -02 |
| untreated LPLD control Cohort | – | –Subjects in Cohort 2 (untreated LPLD control Cohort)) may have completed study PREPARATION-02 or known patients with genetically confirmed LPLD |
| normal healthy control Cohort | – | –Volunteers in Cohort 3 (normal healthy control Cohort) must not have LPLD |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- * Lipoprotein Lipase Deficiency (LPLD) Patients * Healthy volunteers (community sample)
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Composite of Pharmacodynamics
Time frame: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose
Peak level, time-to-peak, and area under the curve (AUC) for tracer in plasma and chylomicron (CM) fraction to assess metabolism of newly-formed CMs in LPLD subjects previously treated with alipogene tiparvovec and to compare with untreated LPLD subjects and healthy controls.
9 secondary outcomes
Triglyceride (TG)-rich lipoproteins
Time frame: pre dose, 0, 1, 2, 3, 4, 4, 6, 7, 8, 9, 12, 24 hours post dose
Glucose
Time frame: pre dose, 0, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose
Adverse Events (AE)
Time frame: Up to 21 days. Serious AEs will be followed to their resolution.
Laboratory tests
Time frame: Up to 21 days
Vital signs
Time frame: Up to 21 days
Physical examination
Time frame: Up to 21 days
ECG
Time frame: Up to 21 days
C-peptide
Time frame: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 post dose
Non Esterified Fatty Acids (NEFA)
Time frame: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 post dose
Results and safety
The study was stopped early (reason given: Business reason). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2015.
- Background: PubMed 17192479Bickerton AS, Roberts R, Fielding BA, Hodson L, Blaak EE, Wagenmakers AJ, Gilbert M, Karpe F, Frayn KN. Preferential uptake of dietary Fatty acids in adipose tissue and muscle in the postprandial period. Diabetes. 2007 Jan;56(1):168-76. doi: 10.2337/db06-0822.
- Background: PubMed 8418601Black DM, Sprecher DL. Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat. Am J Dis Child. 1993 Jan;147(1):60-2. doi: 10.1001/archpedi.1993.02160250062018.
- Background: PubMed 1557997Chait A, Brunzell JD. Chylomicronemia syndrome. Adv Intern Med. 1992;37:249-73.
- Background: PubMed 8540502Fortson MR, Freedman SN, Webster PD 3rd. Clinical assessment of hyperlipidemic pancreatitis. Am J Gastroenterol. 1995 Dec;90(12):2134-9.
- Background: PubMed 14988233Miles JM, Park YS, Walewicz D, Russell-Lopez C, Windsor S, Isley WL, Coppack SW, Harris WS. Systemic and forearm triglyceride metabolism: fate of lipoprotein lipase-generated glycerol and free fatty acids. Diabetes. 2004 Mar;53(3):521-7. doi: 10.2337/diabetes.53.3.521.
- Background: PubMed 20532041Normand-Lauziere F, Frisch F, Labbe SM, Bherer P, Gagnon R, Cunnane SC, Carpentier AC. Increased postprandial nonesterified fatty acid appearance and oxidation in type 2 diabetes is not fully established in offspring of diabetic subjects. PLoS One. 2010 Jun 4;5(6):e10956. doi: 10.1371/journal.pone.0010956.
- Background: PubMed 16574898Rip J, Nierman MC, Ross CJ, Jukema JW, Hayden MR, Kastelein JJ, Stroes ES, Kuivenhoven JA. Lipoprotein lipase S447X: a naturally occurring gain-of-function mutation. Arterioscler Thromb Vasc Biol. 2006 Jun;26(6):1236-45. doi: 10.1161/01.ATV.0000219283.10832.43. Epub 2006 Mar 30.
- Background: PubMed 16259561Rip J, Nierman MC, Sierts JA, Petersen W, Van den Oever K, Van Raalte D, Ross CJ, Hayden MR, Bakker AC, Dijkhuizen P, Hermens WT, Twisk J, Stroes E, Kastelein JJ, Kuivenhoven JA, Meulenberg JM. Gene therapy for lipoprotein lipase deficiency: working toward clinical application. Hum Gene Ther. 2005 Nov;16(11):1276-86. doi: 10.1089/hum.2005.16.1276.
- Background: PubMed 16716106Ross CJ, Twisk J, Bakker AC, Miao F, Verbart D, Rip J, Godbey T, Dijkhuizen P, Hermens WT, Kastelein JJ, Kuivenhoven JA, Meulenberg JM, Hayden MR. Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum Gene Ther. 2006 May;17(5):487-99. doi: 10.1089/hum.2006.17.487.
- Background: PubMed 9785052Santamarina-Fojo S. The familial chylomicronemia syndrome. Endocrinol Metab Clin North Am. 1998 Sep;27(3):551-67, viii. doi: 10.1016/s0889-8529(05)70025-6.
- Background: PubMed 10359734Wittrup HH, Tybjaerg-Hansen A, Nordestgaard BG. Lipoprotein lipase mutations, plasma lipids and lipoproteins, and risk of ischemic heart disease. A meta-analysis. Circulation. 1999 Jun 8;99(22):2901-7. doi: 10.1161/01.cir.99.22.2901.
- Homepage SponsorLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel Gaudet, MD PhD principal investigator
ECOGENE-21 Clinical Trial Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hyperlipoproteinemia Type I, run by Amsterdam Molecular Therapeutics at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Business reason). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Composite of Pharmacodynamics (time frame: pre dose, 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 12, 24 hours post dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01447901, which also lists 11 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2015.