Familial Lipoprotein Lipase DeficiencyPhase 2/3NCT01109498

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

A Phase 2/3 study testing Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Mycophenolate mofetil and cyclosporine in people with Familial Lipoprotein Lipase Deficiency, run by Amsterdam Molecular Therapeutics at 1 site in Canada.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Familial Lipoprotein Lipase Deficiency
What is being tested?: Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Mycophenolate mofetil and cyclosporine
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 14 participants
Where: 1 siteCanada
Study dates: Aug 2007to Jun 2013
Phase: Phase 2/3
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Eligible Population Study participants must have participated in the preceding observation study (Prep-02: Appendix III) and be diagnosed with lipoprotein lipase deficiency, meeting the following criteria: (I) Their lipoprotein lipase activity levels in post-heparin plasma should be ≤20 % of normal; (II) Confirmed homozygocity or compound heterozygocity for mutations in the LPL gene; (III) Post-heparin plasma LPL mass should be >5% of normal; (IV) Median fasting plasma TG concentrations >10.00mmol/L, as determined on the basis of 5 consecutive time points in the preceding observation study with a history of pancreatitis.
General Health The participant 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 trial.
Age Age ≥18 years old.
Sex Male or female. Females must be of non-child bearing potential or with a negative pregnancy test and not breast feeding. Female subjects must use appropriate contraception (if relevant) and their spouse must use barrier contraception for the duration of the study (12 weeks). Males must practice barrier birth control and their spouse should use appropriate contraception until three consecutive semen samples, taken at least 75 days after administration, are negative for AMT-011 vector DNA.
Compliance The participant is willing to fully comply with all study procedures and requirements of the trial such as restrictions to a low-fat diet (see section 8.1).
Consent The participant has the mental ability to give voluntary written informed consent to participate in the study.

Who cannot join

Disease
Apolipoprotein CII deficiency.
Inflammatory muscle disease (e.g. myositis, myopathies or rhabdomolysis).
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 (e.g. malignant neoplasia).
Active infectious disease of any nature, including clinically active viral infections.
Laboratory Parameters

Show all 12Show fewer

The following blood screening tests will result in exclusion from participation:
Platelet count < 100 x 109 /L.
Hemoglobin < 7.0 mmol/L.
Liver function disturbances (bilirubin >2.50 x normal, transaminases >3 x ULN).
CPK > 3 x ULN.
Creatinine > 3 x ULN.

Trial locations and contacts

1 site in 1 country.

Canada1 site

Ecogene-21 Clinical Trial Center/ Centre de santé et de services sociaux de Chicoutimi
Chicoutimi, Quebec

Other trials in Canada

About this study

Official title: A Study to Determine the Safety and Efficacy in Lipoprotein Lipase-Deficient Subjects After Intramuscular Administration of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein LipaseS447X

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.

Read the detailed description

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.

Conditions: Familial Lipoprotein Lipase Deficiency
Being tested: Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Mycophenolate mofetil, cyclosporine
Keywords: LPLD, Lipoprotein Lipase Deficiency, Chylomicronaemia, Genetherapy, AAV, Alipogene Tiparvovec, AMT-011, Lipoprotein Lipase
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.

Alipogene Tiparvovec (AMT-011), Human LPL [S447X]Genetic
Also called Glybera, AMT-011
intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections
Mycophenolate mofetilDrug
Also called CellCept
oral, 2 g/day, day -3 till week 12
Alipogene Tiparvovec (AMT-011), Human LPL [S447X]Genetic
Also called Glybera, AMT 011
intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections
cyclosporineDrug
Also called Neoral
oral, 3 mg/kg/day, day -3 till week 12

Study groups in NCT01109498
Study group	Role	What they receive
Dose cohort 3 x 10^11gc/kg	Experimental	intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections Receives: Genetic: Alipogene Tiparvovec (AMT-011), Human LPL [S447X]
Alipogene Tiparvovec, Human LPL [S447X]	Experimental	intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections with immunosuppressants Receives: Drug: Mycophenolate mofetil; Genetic: Alipogene Tiparvovec (AMT-011), Human LPL [S447X]; Drug: cyclosporine
Alipogene Tiparvovec, Human LPL [S447X], 1xE12 gc/kg	Experimental	intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections with immunosuppressants Receives: Genetic: Alipogene Tiparvovec (AMT-011), Human LPL [S447X]

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 15 years

Secondary outcomes (6)

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.
Measured: 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.
Measured: 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.
Measured: 26 weeks
Biological activity and expression of the transgene product.
To determine the biological activity and expression of the lipoprotein lipase (LPLS447X) transgene product.
Measured: 1 year
Evaluation immune respons
To evaluate potential immune responses against the lipoprotein lipase (LPLS447X) transgene product and the adeno-associated viral (AAV) vector.
Measured: 5 years
To assess the shedding of the viral vector
To assess shedding of AMT-011
Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2007Jun 2013

First posted23 Apr 2010 (estimated)
Study startAug 2007
Primary completionJun 2013 (estimated)
Study completionJun 2013 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (7)Papers the sponsor has linked to this study.
Other trials for Familial Lipoprotein Lipase DeficiencyStudies 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: Interventional
Phase: Phase 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 14 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01109498
Sponsor's protocol ID: CT-AMT-011-01
Status last verified: Apr 2010
First submitted: 22 Apr 2010
Last updated: 30 Sep 2011
Results posted: No
Data monitoring committee: Yes

Publications

Rip 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 16259561
Ross CJ, Twisk J, Meulenberg JM, Liu G, van den Oever K, Moraal E, Hermens WT, Rip J, Kastelein JJ, Kuivenhoven JA, Hayden MR. Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation. Hum Gene Ther. 2004 Sep;15(9):906-19. doi: 10.1089/hum.2004.15.906.
PubMed 15353045
Ross CJ, Liu G, Kuivenhoven JA, Twisk J, Rip J, van Dop W, Excoffon KJ, Lewis SM, Kastelein JJ, Hayden MR. Complete rescue of lipoprotein lipase-deficient mice by somatic gene transfer of the naturally occurring LPLS447X beneficial mutation. Arterioscler Thromb Vasc Biol. 2005 Oct;25(10):2143-50. doi: 10.1161/01.ATV.0000176971.27302.b0. Epub 2005 Jul 7.
PubMed 16002740
Ross 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 16716106
Stroes ES, Nierman MC, Meulenberg JJ, Franssen R, Twisk J, Henny CP, Maas MM, Zwinderman AH, Ross C, Aronica E, High KA, Levi MM, Hayden MR, Kastelein JJ, Kuivenhoven JA. Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol. 2008 Dec;28(12):2303-4. doi: 10.1161/ATVBAHA.108.175620. Epub 2008 Sep 18. No abstract available.
PubMed 18802015
Burnett JR, Hooper AJ. Alipogene tiparvovec, an adeno-associated virus encoding the Ser(447)X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency. Curr Opin Mol Ther. 2009 Dec;11(6):681-91.
PubMed 20072945
Gaudet D, Methot J, Dery S, Brisson D, Essiembre C, Tremblay G, Tremblay K, de Wal J, Twisk J, van den Bulk N, Sier-Ferreira V, van Deventer S. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther. 2013 Apr;20(4):361-9. doi: 10.1038/gt.2012.43. Epub 2012 Jun 21.
PubMed 22717743

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Familial Lipoprotein Lipase Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Sep 2011. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Study to Determine the Safety and Efficacy in Lipoprotein Lipase-Deficient Subjects After Intramuscular Administration of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein LipaseS447X
ClinicalTrials.gov ID: NCT01109498
Acronym / sponsor ID: CT-AMT-011-01
Conditions: Familial Lipoprotein Lipase Deficiency
Keywords: LPLD, Lipoprotein Lipase Deficiency, Chylomicronaemia, Genetherapy, AAV, Alipogene Tiparvovec, AMT-011, Lipoprotein Lipase
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Unknown status
Enrolment: 14 participants (actual)
Lead sponsor: Amsterdam Molecular TherapeuticsIndustry (pharma, biotech, devices)
Collaborators: International Antiviral Therapy Evaluation Center
Study start: Aug 2007
Primary completion: Jun 2013 (estimated)
Study completion: Jun 2013 (estimated)
First posted: 23 Apr 2010 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Dose cohort 3 x 10^11gc/kg	Experimental	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
Alipogene Tiparvovec, Human LPL [S447X]	Experimental	Drug: Mycophenolate mofetil, Genetic: Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Drug: cyclosporineintra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections with immunosuppressants
Alipogene Tiparvovec, Human LPL [S447X], 1xE12 gc/kg	Experimental	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 with immunosuppressants

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Eligible Population Study participants must have participated in the preceding observation study (Prep-02: Appendix III) and be diagnosed with lipoprotein lipase deficiency, meeting the following criteria: (I) Their lipoprotein lipase activity levels in post-heparin plasma should be ≤20 % of normal; (II) Confirmed homozygocity or compound heterozygocity for mutations in the LPL gene; (III) Post-heparin plasma LPL mass should be >5% of normal; (IV) Median fasting plasma TG concentrations >10.00mmol/L, as determined on the basis of 5 consecutive time points in the preceding observation study with a history of pancreatitis.
General Health The participant 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 trial.
Age Age ≥18 years old.
Sex Male or female. Females must be of non-child bearing potential or with a negative pregnancy test and not breast feeding. Female subjects must use appropriate contraception (if relevant) and their spouse must use barrier contraception for the duration of the study (12 weeks). Males must practice barrier birth control and their spouse should use appropriate contraception until three consecutive semen samples, taken at least 75 days after administration, are negative for AMT-011 vector DNA.
Compliance The participant is willing to fully comply with all study procedures and requirements of the trial such as restrictions to a low-fat diet (see section 8.1).

Key exclusion criteria (12 listed)

Disease
Apolipoprotein CII deficiency.
Inflammatory muscle disease (e.g. myositis, myopathies or rhabdomolysis).
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 (e.g. malignant neoplasia).
Active infectious disease of any nature, including clinically active viral infections.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Alipogene Tiparvovec (AMT-011), Human LPL [S447X]Also: Glybera, AMT-011intra muscular, 1 x E12 gc per kg body weight, injected in a single series of intramuscular injections	Genetic	Alipogene Tiparvovec, Human LPL [S447X], 1xE12 gc/kg
Mycophenolate mofetilAlso: CellCeptoral, 2 g/day, day -3 till week 12	Drug	Alipogene Tiparvovec, Human LPL [S447X]
Alipogene Tiparvovec (AMT-011), Human LPL [S447X]Also: Glybera, AMT 011intra muscular, 3 x E11 gc per kg body weight, injected in a single series of intramuscular injections	Genetic	Alipogene Tiparvovec, Human LPL [S447X], Dose cohort 3 x 10^11gc/kg
cyclosporineAlso: Neoraloral, 3 mg/kg/day, day -3 till week 12	Drug	Alipogene Tiparvovec, Human LPL [S447X]

Primary outcome

Reduction of fasting triglyceride (TG) concentrations
Time frame: 12 weeks
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.
safety profile of AMT-011
Time frame: 15 years
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.

6 secondary outcomes

Reduction of TG concentrations
Time frame: 26 weeks
Reduction of TG concentrations
Time frame: 12 weeks
Reduction of TG concentrations
Time frame: 26 weeks
Biological activity and expression of the transgene product.
Time frame: 1 year
Evaluation immune respons
Time frame: 5 years
To assess the shedding of the viral vector
Time frame: 1 year

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2011.
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 15353045Ross CJ, Twisk J, Meulenberg JM, Liu G, van den Oever K, Moraal E, Hermens WT, Rip J, Kastelein JJ, Kuivenhoven JA, Hayden MR. Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation. Hum Gene Ther. 2004 Sep;15(9):906-19. doi: 10.1089/hum.2004.15.906.
Background: PubMed 16002740Ross CJ, Liu G, Kuivenhoven JA, Twisk J, Rip J, van Dop W, Excoffon KJ, Lewis SM, Kastelein JJ, Hayden MR. Complete rescue of lipoprotein lipase-deficient mice by somatic gene transfer of the naturally occurring LPLS447X beneficial mutation. Arterioscler Thromb Vasc Biol. 2005 Oct;25(10):2143-50. doi: 10.1161/01.ATV.0000176971.27302.b0. Epub 2005 Jul 7.
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 18802015Stroes ES, Nierman MC, Meulenberg JJ, Franssen R, Twisk J, Henny CP, Maas MM, Zwinderman AH, Ross C, Aronica E, High KA, Levi MM, Hayden MR, Kastelein JJ, Kuivenhoven JA. Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol. 2008 Dec;28(12):2303-4. doi: 10.1161/ATVBAHA.108.175620. Epub 2008 Sep 18. No abstract available.
Background: PubMed 20072945Burnett JR, Hooper AJ. Alipogene tiparvovec, an adeno-associated virus encoding the Ser(447)X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency. Curr Opin Mol Ther. 2009 Dec;11(6):681-91.
Derived from this study: PubMed 22717743Gaudet D, Methot J, Dery S, Brisson D, Essiembre C, Tremblay G, Tremblay K, de Wal J, Twisk J, van den Bulk N, Sier-Ferreira V, van Deventer S. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther. 2013 Apr;20(4):361-9. doi: 10.1038/gt.2012.43. Epub 2012 Jun 21.

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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.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 6 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 30 Sep 2011. 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.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

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?

Reduction of fasting triglyceride (TG) concentrations (time frame: 12 weeks). The study lists 2 primary outcomes in all. 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 NCT01109498, which also lists 7 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 Sep 2011.