A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability … (NCT01514461) | Clinical Trial Compass
CompletedPhase 3
A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
United States, Canada, France45 participantsStarted 2012-07
Plain-language summary
The purpose of this study is to determine whether LCQ908 is effective and safe in lowering triglycerides in subjects with Familial Chylomicronemia Syndrome (FCS) (Hyperlipoproteinemia \[HLP\] type I). Data from this study will be used to support a registration submission of LCQ908 20 mg and 40 mg as treatment of chylomicronemia in subjects with FCS (HLP Type 1).
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Written informed consent given before any assessment was performed for Period I.
. Male and female patients ages at least 18 years of age.
. Fasting triglyceride ≥ 8.4 mmol/L (750 mg/dL) at Screening.
. An established diagnosis of FCS (HLP Type I) confirmed through ultracentrifugation or by documented medical history of a fasting triglyceride ≥ 8.4 mmol/L (750 mg/dL) and by documentation of any of the following at Screening or during the Screening Period:
. History of pancreatitis.
Exclusion criteria
. Current pancreatitis, pancreatitis was required to be inactive for at least 1 week prior to the screening Visit.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial tested LCQ908 against a placebo for 12 weeks and measured how much fasting triglycerides changed — since this was a Phase 3 study that has now completed, has the results data been published, and what did they show about whether LCQ908 actually lowered triglycerides in people with FCS?
2Since the trial is completed, is LCQ908 approved or available to me now as a treatment, or is it still not accessible outside of research settings?
3FCS is a very rare condition with limited treatment options — based on this trial's findings, how does LCQ908 compare to whatever options I currently have for managing my triglyceride levels and reducing my risk of pancreatitis?
4The trial specifically focused on fasting triglycerides at 12 weeks as its main measure — does that timeframe and that particular outcome tell us enough about long-term safety and real-world benefit for someone in my situation, or are there still unknowns I should understand?
5Given how rare FCS is, is there a specialist or center with experience in this condition who you'd recommend I consult with before deciding on any treatment path, including any options that emerged from this trial?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percent Change in Fasting Triglycerides From Baseline to 12 Weeks
. Treatment with fish oil preparations within 4 weeks prior to randomization.
. Treatment with bile acid binding resins (i.e., colesevelam, etc.) within 4 weeks prior to randomization.
. Treatment with fibrates within 4 weeks prior to randomization.
. Glybera \[alipogene tiparvovec (AAV1-LPLS447X)\] gene therapy exposure within the two years prior to screening.
. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
. Any surgical or medical conditions, acute or unstable chronic disease which may, based on the investigator's opinion, jeopardize the patient in case of participation in the study or might significantly alter the absorption, distribution, metabolism or excretion of the study drug.
. History of drug or alcohol abuse within the 12 months prior to randomization or evidence of such abuse at screening.