Trial · NCT01514461
A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
NCT01514461, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2015-06-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome. Trials, record NCT01514461. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01514461
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
- NCT number
- NCT01514461
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 45 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Familial Chylomicronemia Syndrome (FCS)
- Interventions
- LCQ908, Placebo
- First posted
- 2012-01-23
- Last updated
- 2015-06-03
- Other registries' IDs
- eudract:2011-005535-68
- Results first posted
- 2015-06-03
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in Fasting Triglycerides From Baseline to 12 Weeks | Baseline to 12 weeks | Blood samples were collected for a fasting lipid panel, including triglycerides. If the 12-week value was missing, the measurement value at 12 weeks or the last available post-baseline measurement value during the double-blind treatment period was analyzed. Baseline is defined as the average of fasting triglyceride values taken at day -3 and day 1. Adjusted geometric means are calculated by back-transforming the adjusted means from the model and expressing as a percentage change from baseline. |
| Secondary | Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | Baseline, 12 weeks, 24 weeks, 52 weeks | Percentage calculated as (m/n)\*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride. |
| Secondary | Percentage of Patients Responding to Investigational Treatment by Achieving Final Fasting Triglycerides < 8.4 mmol/L (750 mg/dL) | 12 weeks, 24 weeks, 52 weeks | Percentage calculated as (m/n)\*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride. |
| Secondary | Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline | Baseline, 12 weeks, 24 weeks, 52 weeks | Percentage calculated as (m/n)\*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride. |
| Secondary | Percentage of Patients Achieving Fasting Triglycerides (TG) Target Thresholds | 12 weeks, 24 weeks, 52 weeks | Percentage of patients reaching target values of \<1000 mg/dL or target values of \< 2000 mg/dL for fasting triglycerides is reported. Pecentage calculated as (m/n)\*100; where 'm' The number of patients who reach target values for fasting triglyceride, 'n' the number of patients with non-missing fasting triglyceride. |
| Secondary | Percent Change From Baseline in Fasting Triglycerides | Baseline, 24 weeks, 52 weeks | |
| Secondary | Percent Change From Baseline for Postprandial Triglycerides Following the Standardized Meal Tolerance Test at Week 12 | 0-24 hours at Baseline, Week 12 | Post prandial peak triglycerides - maximum triglyceride value over 0-24 hours Post prandial triglycerides AUC0-24 - area under the time curve for triglycerides over 0-24 Adjusted geometric means are calculated by back-transforming the adjusted means from the model and expressed as a percentage change from baseline. hours |
| Secondary | Pharmacokinetics of LCQ908 - Trough Concentration (Cmin) and Observed Maximum Blood Concentration (Cmax) | 0, 1, 2, 3, 4, 6, and 24 hours at Week 12 | Lowest observed blood concentration (Cmin) and observed maximum blood concentration (Cmax) following drug administration derived from non-compartmental analysis using scheduled sampling time for the whole dataset. |
| Secondary | Pharmacokinetics of LCQ908- Area Under the Plasma Concentration Time Curve AUC (0-24hour) | 0, 1, 2, 3, 4, 6, and 24 hours at Week 12 | The area under the concentration-time curve from time zero to 24 hours after drug administration was calculated by using linear trapezoidal rule. |
| Secondary | Pharmacokinetics of LCQ908- Time to Reach Maximum Concentration Following Drug Administration Tmax (Hours) | 0, 1, 2, 3, 4, 6, and 24 hours at Week 12 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in Fasting Triglycerides From Baseline to 12 Weeks | Baseline to 12 weeks | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. The number of randomized patients with non-missing fasting triglycerides values at baseline and Week 12 are included in this analysis. | percent change | GEOMETRIC_MEAN |
| SECONDARY | Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | Baseline, 12 weeks, 24 weeks, 52 weeks | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Patients Responding to Investigational Treatment by Achieving Final Fasting Triglycerides < 8.4 mmol/L (750 mg/dL) | 12 weeks, 24 weeks, 52 weeks | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline | Baseline, 12 weeks, 24 weeks, 52 weeks | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Patients Achieving Fasting Triglycerides (TG) Target Thresholds | 12 weeks, 24 weeks, 52 weeks | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. | Percentage of patients | NUMBER |
| SECONDARY | Percent Change From Baseline in Fasting Triglycerides | Baseline, 24 weeks, 52 weeks | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. | Percent change | GEOMETRIC_MEAN |
| SECONDARY | Percent Change From Baseline for Postprandial Triglycerides Following the Standardized Meal Tolerance Test at Week 12 | 0-24 hours at Baseline, Week 12 | Full analysis set (FAS) consisted of all randomized patients, except for those who were mis-randomized. For each category, the number of randomized patients who have non-missing values are included in this analysis. | Percent change | GEOMETRIC_MEAN |
| SECONDARY | Pharmacokinetics of LCQ908 - Trough Concentration (Cmin) and Observed Maximum Blood Concentration (Cmax) | 0, 1, 2, 3, 4, 6, and 24 hours at Week 12 | Safety set (SAF) consisted of all patients who received at least one dose of study drug and had at least one post-baseline safety assessment | ng/mL | MEAN |
| SECONDARY | Pharmacokinetics of LCQ908- Area Under the Plasma Concentration Time Curve AUC (0-24hour) | 0, 1, 2, 3, 4, 6, and 24 hours at Week 12 | Safety set (SAF) consisted of all patients who received at least one dose of study drug and had at least one post-baseline safety assessment | ng/mL *hr | MEAN |
| SECONDARY | Pharmacokinetics of LCQ908- Time to Reach Maximum Concentration Following Drug Administration Tmax (Hours) | 0, 1, 2, 3, 4, 6, and 24 hours at Week 12 | Safety set (SAF) consisted of all patients who received at least one dose of study drug and had at least one post-baseline safety assessment. | hours | MEDIAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group79
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change in Fasting Triglycerides From Baseline to 12 Weeks | Placebo | 45.6 | 95% Confidence Interval 6.8 to 98.7 | percent change | |
| Percent Change in Fasting Triglycerides From Baseline to 12 Weeks | LCQ908 20mg | 3.7 | 95% Confidence Interval -24.3 to 42.1 | percent change | |
| Percent Change in Fasting Triglycerides From Baseline to 12 Weeks | LCQ908 40 mg | -13.9 | 95% Confidence Interval -38.9 to 21.3 | percent change | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | Placebo | Week 12 (n = 14, 14, 12) | 14.3 | Percentage of participants | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | LCQ908 20 mg | Week 12 (n = 14, 14, 12) | 21.4 | Percentage of participants | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | LCQ908 40 mg | Week 12 (n = 14, 14, 12) | 50.0 | Percentage of participants | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | Placebo | Week 24 (n = 13, 14, 12) | 30.8 | Percentage of participants | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | LCQ908 20 mg | Week 24 (n = 13, 14, 12) | 35.7 | Percentage of participants | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | LCQ908 40 mg | Week 24 (n = 13, 14, 12) | 33.3 | Percentage of participants | |
| Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL) | Placebo | Week 52 (n = 11, 14, 11) | 18.2 | Percentage of participants |
1 to 10 of 79Source ClinicalTrials.gov
See all 79 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 15 | |
| Overall Study | STARTED | LCQ908 20 mg | 15 | |
| Overall Study | STARTED | LCQ908 40 mg | 15 | |
| Overall Study | COMPLETED | Placebo | 10 | |
| Overall Study | COMPLETED | LCQ908 20 mg | 12 | |
| Overall Study | COMPLETED | LCQ908 40 mg | 11 | |
| Overall Study | NOT COMPLETED | Placebo | 5 | |
| Overall Study | NOT COMPLETED | LCQ908 20 mg | 3 | |
| Overall Study | NOT COMPLETED | LCQ908 40 mg | 4 |
Source ClinicalTrials.gov
Adverse events453
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | ANAEMIA | Blood and lymphatic system disorders | Placebo | 2 | 15 | |
| other | ANAEMIA | Blood and lymphatic system disorders | LCQ908 20 mg | 1 | 15 | |
| other | ANAEMIA | Blood and lymphatic system disorders | LCQ908 40 mg | 0 | 14 | |
| other | LEUKOPENIA | Blood and lymphatic system disorders | Placebo | 0 | 15 | |
| other | LEUKOPENIA | Blood and lymphatic system disorders | LCQ908 20 mg | 0 | 15 | |
| other | LEUKOPENIA | Blood and lymphatic system disorders | LCQ908 40 mg | 1 | 14 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | Placebo | 0 | 15 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | LCQ908 20 mg | 0 | 15 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | LCQ908 40 mg | 1 | 14 | |
| other | CARDIAC ARREST | Cardiac disorders | Placebo | 0 | 15 |
1 to 10 of 453Source ClinicalTrials.gov
See all 453 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | ||
| Total, all-cause mortality | LCQ908 20 mg | ||
| Total, all-cause mortality | LCQ908 40 mg | ||
| Total, other adverse events | Placebo | 15 | 15 |
| Total, other adverse events | LCQ908 20 mg | 15 | 15 |
| Total, other adverse events | LCQ908 40 mg | 14 | 14 |
| Total, serious adverse events | Placebo | 6 | 15 |
| Total, serious adverse events | LCQ908 20 mg | 6 | 15 |
| Total, serious adverse events | LCQ908 40 mg | 3 | 14 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01514461
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