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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-000802-32 | EudraCT Number |
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Interim analysis suggested that size of benefit anticipated from continued participation of patients in Part B no longer supported trial extension beyond Part A
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This study was to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I).
This study was an 52 weeks open label extension starting at the lowest treatment dose used in CLCQ908B2302/NCT01514461 (i.e., 10 mg) with optional up-titrations, to evaluate the overall long-term safety and tolerability of LCQ908 in patients with Familial Chylomicronemia Syndrome, who either discontinued from the CLCQ908B2302/NCT01514461 study (due to tolerability issues) or completed the CLCQ908B2302/NCT01514461 study after 52 weeks. In addition, patients who had previously completed study CLCQ908A2212/NCT01146522 were eligible to participate.
Following Protocol amendment 2, the original 52 week duration of this study (CLCQ908B2305) became Part A of LCQ908B2305 and a 78 week extension became Part B. However, following Protocol amendment 3, Part B was ended at the same time as the last patient of Part A completed 52 weeks. The reason for termination of Part B was the findings from the December 2014 interim analysis which suggested that the size of benefit that was anticipated from continued participation of patients in the 18 month extension trial (Part B) no longer supported trial extension beyond Part A.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LCQ908 | Experimental | Patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose will be allowed. One down titration allowed from the highest dose attained. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LCQ908 | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients With Any Adverse Events, Serious Adverse Events and Death | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Changes From Baseline in Triglyceride Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including total triglycerides. Lipid measurements were collected after a 12 hour (overnight) fast. The maintenance of effect was assessed on triglyceride levels during continued therapy with LCQ908 for up to 52 weeks. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol defined inclusion/exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Seatlle | Washington | 98104 | United States | ||
| Novartis Investigative Site |
100% patients who completed the screening phase were enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo of Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to placebo in study CLCQ908B2302/NCT01514461. In current study, patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (52 Weeks) |
|
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| Baseline, Week 12, 24 and 52 |
| Changes From Baseline in Cholesterol Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including cholesterol level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including HDL and non HDL cholesterol level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Changes From Baseline in Glycerol Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including glycerol level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including free fatty acid level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks | Fasting blood samples were collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipoprotein biomarkers such as Apolipoprotein A1. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks | Fasting blood samples were collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipoprotein biomarkers such as Apolipoprotein B-48. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks | Fasting blood samples were collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipoprotein biomarkers such as Apolipoprotein B-100. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Baseline, Week 12, 24 and 52 |
| Chicoutimi |
| Quebec |
| G7H 7P2 |
| Canada |
| Novartis Investigative Site | Ste-Foy | Quebec | G1V4M6 | Canada |
| Novartis Investigative Site | Ouest-Montreal | H2W1R7 | Canada |
| Novartis Investigative Site | Nantes | 44093 | France |
| Novartis Investigative Site | Paris | 75651 | France |
| Novartis Investigative Site | Hamburg | 20246 | Germany |
| Novartis Investigative Site | Meibergdreef 9 | 1105 AZ | Netherlands |
| Novartis Investigative Site | Cape Town | 7925 | South Africa |
| Novartis Investigative Site | Manchester | M13 9NT | United Kingdom |
| FG001 | 20 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| FG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| FG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Part B (Planned for 78 Week-terminated) |
|
|
Full analysis set (FAS) - All subjects in the extension study who were in full analysis set in either study LCQ908A2212/NCT01146522 or LCQ908B2302/NCT01514461.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo of Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to placebo in study CLCQ908B2302/NCT01514461. In current study, patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| BG001 | 20 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| BG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| BG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Patients With Any Adverse Events, Serious Adverse Events and Death | Safety set (SAF) - All subjects who received at least one dose of study drug and had at least one post-baseline safety assessment in this extension study. | Posted | Number | Participants | 52 weeks |
|
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| Secondary | Changes From Baseline in Triglyceride Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including total triglycerides. Lipid measurements were collected after a 12 hour (overnight) fast. The maintenance of effect was assessed on triglyceride levels during continued therapy with LCQ908 for up to 52 weeks. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
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| Secondary | Changes From Baseline in Cholesterol Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including cholesterol level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
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| Secondary | Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including HDL and non HDL cholesterol level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
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| Secondary | Changes From Baseline in Glycerol Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including glycerol level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
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| Secondary | Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks | Blood samples were collected for a fasting lipid panel, including free fatty acid level. Lipid measurements were collected after a 12 hour (overnight) fast. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
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| Secondary | Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks | Fasting blood samples were collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipoprotein biomarkers such as Apolipoprotein A1. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks | Fasting blood samples were collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipoprotein biomarkers such as Apolipoprotein B-48. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
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| Secondary | Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks | Fasting blood samples were collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipoprotein biomarkers such as Apolipoprotein B-100. For patients from LCQ908 arm of study CLCQ908A2212, baseline was the assessment obtained at Week 0 of current study. For patients from study CLCQ908B2302, baseline is defined as the average of values taken Day -3 and Week 0 (randomization) in study CLCQ908B2302. The geometric mean for the percentage (%) change is calculated from back-transforming the mean of the log transformed ratio to baseline values: (exp(mean of the log-transformed ratio to baseline values) -1)*100. | Full analysis set (FAS) - All subjects in the extension study who were in FAS in either study LCQ908A2212 or LCQ908B2302. At each time point post-baseline, only patients with a value at both baseline and the post-dose time point are included. | Posted | Geometric Mean | Geometric Coefficient of Variation | percentage change | Baseline, Week 12, 24 and 52 |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A-placebo of Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to placebo in study CLCQ908B2302/NCT01514461. In current study, patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. | 1 | 11 | 11 | 11 | ||
| EG001 | Part A-20mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. | 6 | 12 | 12 | 12 | ||
| EG002 | Part A-40mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. | 2 | 10 | 10 | 10 | ||
| EG003 | Part A: Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. | 2 | 5 | 5 | 5 | ||
| EG004 | Part B-placebo of Pradigastat (LCQ908) Regimen | Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. | 0 | 5 | 4 | 5 | ||
| EG005 | Part B-20mg Pradigastat (LCQ908) Regimen | Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. | 0 | 6 | 3 | 6 | ||
| EG006 | Part B- Pradigastat (LCQ908) Regimen- From Study A2212 | Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. | 0 | 4 | 3 | 4 | ||
| EG007 | Part B-40 mg Pradigastat (LCQ908) | Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients' tolerance and safety profile. | 0 | 4 | 0 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| CORONARY ARTERY STENOSIS | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| PANCREATITIS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| PANCREATITIS ACUTE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| POSTOPERATIVE WOUND INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| ASPARTATE AMINOTRANSFERASE INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| DEHYDRATION | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| DIABETES MELLITUS INADEQUATE CONTROL | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOGLYCAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| MALNUTRITION | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| CARDIAC FAILURE | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| ABNORMAL PALMAR/PLANTAR CREASES | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
| |
| EAR PAIN | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| VERTIGO POSITIONAL | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL DISCOMFORT | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL DISTENSION | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL PAIN UPPER | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| DYSPEPSIA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| FAECAL INCONTINENCE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| FOOD POISONING | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| GASTRITIS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| PANCREATITIS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| PANCREATITIS ACUTE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| STEATORRHOEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| TONGUE CYST | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA | Systematic Assessment |
| |
| NON-CARDIAC CHEST PAIN | General disorders | MedDRA | Systematic Assessment |
| |
| THIRST | General disorders | MedDRA | Systematic Assessment |
| |
| VESSEL PUNCTURE SITE INDURATION | General disorders | MedDRA | Systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| CYSTITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| INFLUENZA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| LUNG INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| NASOPHARYNGITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| POST PROCEDURAL INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| SINUSITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| VAGINAL INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| VULVOVAGINAL MYCOTIC INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| CONTUSION | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| FIBULA FRACTURE | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| LIGAMENT RUPTURE | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| MUSCLE STRAIN | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| POST PROCEDURAL INFLAMMATION | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| WOUND | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| CARDIAC MURMUR | Investigations | MedDRA | Systematic Assessment |
| |
| CAROTID BRUIT | Investigations | MedDRA | Systematic Assessment |
| |
| WEIGHT DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| DECREASED APPETITE | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| GOUT | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOGLYCAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOKALAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| VITAMIN B COMPLEX DEFICIENCY | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| VITAMIN D DEFICIENCY | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCULOSKELETAL PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MYALGIA | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| NECK PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| OSTEOARTHRITIS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| ROTATOR CUFF SYNDROME | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| SYNOVIAL CYST | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| TENDONITIS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| APHONIA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| DIZZINESS | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| MIGRAINE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| PARAESTHESIA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| RESTLESS LEGS SYNDROME | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| SCIATICA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| SYNCOPE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| AGITATION | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| DEPRESSED MOOD | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| INSOMNIA | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| STRESS | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| DYSMENORRHOEA | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| ERECTILE DYSFUNCTION | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| PREMATURE MENOPAUSE | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| PHARYNGEAL INFLAMMATION | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| PNEUMOTHORAX | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| RHINORRHOEA | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| ALOPECIA | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| PSORIASIS | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| RASH | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| XANTHOMA | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| AORTIC ANEURYSM | Vascular disorders | MedDRA | Systematic Assessment |
| |
| HOT FLUSH | Vascular disorders | MedDRA | Systematic Assessment |
| |
| HYPERTENSION | Vascular disorders | MedDRA | Systematic Assessment |
|
As the anticipated benefit from the continued participation of patients in 18 month extension (Part B) was not supported by results of the December 2014 interim analysis, Novartis decided to terminate the Part B to be effective as of May 31, 2015.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | trialandresults.registries@novartis.com |
| ID | Term |
|---|---|
| C538489 | Familial hyperchylomicronemia syndrome |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C000594809 | pradigastat |
Not provided
Not provided
Not provided
| Study terminated by sponsor |
|
| Physician Decision |
|
| Male |
|
| At least one serious AE |
|
| Death |
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|
Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.
| OG002 | 40 mg Pradigastat (LCQ908) Regimen | Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
| OG003 | Pradigastat (LCQ908) Regimen- From Study A2212 | Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained. |
|
|