Trial Outcomes & Findings for Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome. (NCT NCT01589237)
NCT ID: NCT01589237
Last Updated: 2016-11-15
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
38 participants
Primary outcome timeframe
52 weeks
Results posted on
2016-11-15
Participant Flow
100% patients who completed the screening phase were enrolled in the study.
Participant milestones
| Measure |
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.
|
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.
|
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.
|
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.
|
|---|---|---|---|---|
|
Part A (52 Weeks)
STARTED
|
11
|
12
|
10
|
5
|
|
Part A (52 Weeks)
COMPLETED
|
9
|
9
|
8
|
5
|
|
Part A (52 Weeks)
NOT COMPLETED
|
2
|
3
|
2
|
0
|
|
Part B (Planned for 78 Week-terminated)
STARTED
|
5
|
6
|
4
|
4
|
|
Part B (Planned for 78 Week-terminated)
COMPLETED
|
0
|
0
|
0
|
0
|
|
Part B (Planned for 78 Week-terminated)
NOT COMPLETED
|
5
|
6
|
4
|
4
|
Reasons for withdrawal
| Measure |
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.
|
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.
|
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.
|
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.
|
|---|---|---|---|---|
|
Part A (52 Weeks)
Subject/guardian decision
|
2
|
3
|
2
|
0
|
|
Part B (Planned for 78 Week-terminated)
Subject/guardian decision
|
0
|
1
|
0
|
0
|
|
Part B (Planned for 78 Week-terminated)
Study terminated by sponsor
|
5
|
5
|
4
|
3
|
|
Part B (Planned for 78 Week-terminated)
Physician Decision
|
0
|
0
|
0
|
1
|
Baseline Characteristics
Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.
Baseline characteristics by cohort
| Measure |
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Total
n=38 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
52.9 Years
STANDARD_DEVIATION 10.22 • n=5 Participants
|
44.1 Years
STANDARD_DEVIATION 14.26 • n=7 Participants
|
43.6 Years
STANDARD_DEVIATION 84.53 • n=5 Participants
|
52.2 Years
STANDARD_DEVIATION 12.72 • n=4 Participants
|
47.6 Years
STANDARD_DEVIATION 11.43 • n=21 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
16 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
22 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: 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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Number of Patients With Any Adverse Events, Serious Adverse Events and Death
Death
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Patients With Any Adverse Events, Serious Adverse Events and Death
At least one Adverse Event (any)
|
5 Participants
|
11 Participants
|
12 Participants
|
10 Participants
|
|
Number of Patients With Any Adverse Events, Serious Adverse Events and Death
At least one serious AE
|
2 Participants
|
1 Participants
|
6 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Triglyceride Levels up to 52 Weeks
Change in week 12 (n=10,11,10,5)
|
-19.36 percentage change
Geometric Coefficient of Variation 42.82
|
1.63 percentage change
Geometric Coefficient of Variation 45.19
|
-5.80 percentage change
Geometric Coefficient of Variation 66.10
|
43.94 percentage change
Geometric Coefficient of Variation 52.66
|
|
Changes From Baseline in Triglyceride Levels up to 52 Weeks
change in week 24 (n=10,10,9,5)
|
-26.05 percentage change
Geometric Coefficient of Variation 31.50
|
-14.59 percentage change
Geometric Coefficient of Variation 52.33
|
-36.19 percentage change
Geometric Coefficient of Variation 64.80
|
32.54 percentage change
Geometric Coefficient of Variation 87.83
|
|
Changes From Baseline in Triglyceride Levels up to 52 Weeks
change in week 52 (n=9,8,9,5)
|
-9.20 percentage change
Geometric Coefficient of Variation 43.34
|
16.46 percentage change
Geometric Coefficient of Variation 29.27
|
-30.03 percentage change
Geometric Coefficient of Variation 78.52
|
92.15 percentage change
Geometric Coefficient of Variation 60.21
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Cholesterol Levels up to 52 Weeks
Change in week 12 (n=10,11,10,5)
|
-10.42 percentage change
Geometric Coefficient of Variation 24.68
|
-5.58 percentage change
Geometric Coefficient of Variation 36.38
|
-4.76 percentage change
Geometric Coefficient of Variation 36.26
|
18.76 percentage change
Geometric Coefficient of Variation 31.54
|
|
Changes From Baseline in Cholesterol Levels up to 52 Weeks
change in week 24 (n=10,10,9,5)
|
-13.87 percentage change
Geometric Coefficient of Variation 25.64
|
-10.54 percentage change
Geometric Coefficient of Variation 27.87
|
-21.54 percentage change
Geometric Coefficient of Variation 24.98
|
7.45 percentage change
Geometric Coefficient of Variation 37.28
|
|
Changes From Baseline in Cholesterol Levels up to 52 Weeks
change in week 52 (n=9,8,9,5)
|
-6.84 percentage change
Geometric Coefficient of Variation 22.55
|
5.75 percentage change
Geometric Coefficient of Variation 17.51
|
-13.76 percentage change
Geometric Coefficient of Variation 36.10
|
40.95 percentage change
Geometric Coefficient of Variation 34.10
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
HDL: change in week 24 (n=10,10,9,5)
|
-15.60 percentage change
Geometric Coefficient of Variation 37.69
|
-7.11 percentage change
Geometric Coefficient of Variation 19.41
|
-1.83 percentage change
Geometric Coefficient of Variation 31.36
|
6.67 percentage change
Geometric Coefficient of Variation 22.19
|
|
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
Non HDL: change in week 24 (n=10,10,9,5)
|
-14.01 percentage change
Geometric Coefficient of Variation 28.04
|
-11.29 percentage change
Geometric Coefficient of Variation 29.53
|
-25.22 percentage change
Geometric Coefficient of Variation 31.49
|
7.17 percentage change
Geometric Coefficient of Variation 40.50
|
|
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
HDL: Change in week 12 (n=10,11,10,5)
|
-7.09 percentage change
Geometric Coefficient of Variation 27.11
|
-14.13 percentage change
Geometric Coefficient of Variation 30.08
|
3.37 percentage change
Geometric Coefficient of Variation 24.75
|
-5.99 percentage change
Geometric Coefficient of Variation 22.19
|
|
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
Non HDL: change in week 12 (n=10,11,10,5)
|
-10.57 percentage change
Geometric Coefficient of Variation 26.16
|
-5.37 percentage change
Geometric Coefficient of Variation 39.61
|
-7.72 percentage change
Geometric Coefficient of Variation 42.40
|
20.70 percentage change
Geometric Coefficient of Variation 34.18
|
|
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
HDL: change in week 52 (n=9,8,9,5)
|
-21.41 percentage change
Geometric Coefficient of Variation 22.91
|
-10.85 percentage change
Geometric Coefficient of Variation 19.78
|
8.11 percentage change
Geometric Coefficient of Variation 29.35
|
-7.33 percentage change
Geometric Coefficient of Variation 27.63
|
|
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
Non HDL: change in week 52 (n=9,8,9,5)
|
-6.09 percentage change
Geometric Coefficient of Variation 24.59
|
8.06 percentage change
Geometric Coefficient of Variation 21.42
|
-17.68 percentage change
Geometric Coefficient of Variation 42.65
|
45.25 percentage change
Geometric Coefficient of Variation 37.45
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Glycerol Levels up to 52 Weeks
change in week 24 (n=10,10,9,5)
|
-56.99 percentage change
Geometric Coefficient of Variation 96.06
|
-40.00 percentage change
Geometric Coefficient of Variation 67.28
|
-46.97 percentage change
Geometric Coefficient of Variation 62.46
|
-36.26 percentage change
Geometric Coefficient of Variation 133.58
|
|
Changes From Baseline in Glycerol Levels up to 52 Weeks
Change in week 12 (n=10,11,10,5)
|
-46.83 percentage change
Geometric Coefficient of Variation 105.58
|
-26.56 percentage change
Geometric Coefficient of Variation 72.88
|
-15.50 percentage change
Geometric Coefficient of Variation 73.33
|
0.68 percentage change
Geometric Coefficient of Variation 75.40
|
|
Changes From Baseline in Glycerol Levels up to 52 Weeks
change in week 52 (n=9,8,9,5)
|
-37.02 percentage change
Geometric Coefficient of Variation 81.67
|
-38.15 percentage change
Geometric Coefficient of Variation 70.10
|
-31.96 percentage change
Geometric Coefficient of Variation 64.76
|
-28.52 percentage change
Geometric Coefficient of Variation 101.85
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks
change in week 52 (n=9,8,9,5)
|
-18.29 percentage change
Geometric Coefficient of Variation 104.77
|
-16.99 percentage change
Geometric Coefficient of Variation 43.34
|
-7.69 percentage change
Geometric Coefficient of Variation 54.21
|
79.15 percentage change
Geometric Coefficient of Variation 49.37
|
|
Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks
Change in week 12 (n=10,11,10,5)
|
-46.58 percentage change
Geometric Coefficient of Variation 128.38
|
-23.11 percentage change
Geometric Coefficient of Variation 53.05
|
-17.85 percentage change
Geometric Coefficient of Variation 62.48
|
53.09 percentage change
Geometric Coefficient of Variation 44.83
|
|
Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks
change in week 24 (n=10,10,9,5)
|
-42.74 percentage change
Geometric Coefficient of Variation 108.73
|
-20.35 percentage change
Geometric Coefficient of Variation 80.16
|
-30.44 percentage change
Geometric Coefficient of Variation 62.01
|
36.18 percentage change
Geometric Coefficient of Variation 64.97
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks
change in week 24 (n=10,10,10,5)
|
5.11 percentage change
Geometric Coefficient of Variation 28.49
|
4.80 percentage change
Geometric Coefficient of Variation 14.16
|
1.41 percentage change
Geometric Coefficient of Variation 17.45
|
5.57 percentage change
Geometric Coefficient of Variation 25.69
|
|
Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks
Change in week 12 (n=11,11,10,5)
|
2.95 percentage change
Geometric Coefficient of Variation 33.54
|
-3.24 percentage change
Geometric Coefficient of Variation 23.82
|
2.55 percentage change
Geometric Coefficient of Variation 15.09
|
6.58 percentage change
Geometric Coefficient of Variation 17.04
|
|
Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks
change in week 52 (n=10,8,9,5)
|
-0.82 percentage change
Geometric Coefficient of Variation 16.79
|
4.51 percentage change
Geometric Coefficient of Variation 19.21
|
10.01 percentage change
Geometric Coefficient of Variation 16.28
|
4.43 percentage change
Geometric Coefficient of Variation 19.34
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks
change in week 24 (n=10,10,10,5)
|
-35.04 percentage change
Geometric Coefficient of Variation 31.11
|
9.13 percentage change
Geometric Coefficient of Variation 30.79
|
-10.23 percentage change
Geometric Coefficient of Variation 58.91
|
105.52 percentage change
Geometric Coefficient of Variation 71.45
|
|
Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks
change in week 52 (n=10,8,9,5)
|
27.75 percentage change
Geometric Coefficient of Variation 57.77
|
56.25 percentage change
Geometric Coefficient of Variation 57.65
|
-22.63 percentage change
Geometric Coefficient of Variation 107.21
|
135.33 percentage change
Geometric Coefficient of Variation 71.11
|
|
Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks
Change in week 12 (n=11,11,10,5)
|
-30.03 percentage change
Geometric Coefficient of Variation 61.78
|
-4.03 percentage change
Geometric Coefficient of Variation 79.87
|
33.24 percentage change
Geometric Coefficient of Variation 79.20
|
109.67 percentage change
Geometric Coefficient of Variation 52.50
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24 and 52Population: 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.
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.
Outcome measures
| Measure |
Pradigastat (LCQ908) Regimen- From Study A2212
n=5 Participants
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.
|
Placebo of Pradigastat (LCQ908) Regimen
n=11 Participants
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.
|
20 mg Pradigastat (LCQ908) Regimen
n=12 Participants
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.
|
40 mg Pradigastat (LCQ908) Regimen
n=10 Participants
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.
|
|---|---|---|---|---|
|
Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks
Change in week 12 (n=9,11,9,5)
|
3.33 percentage change
Geometric Coefficient of Variation 32.46
|
-15.75 percentage change
Geometric Coefficient of Variation 41.50
|
15.10 percentage change
Geometric Coefficient of Variation 58.53
|
-8.34 percentage change
Geometric Coefficient of Variation 32.73
|
|
Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks
change in week 24 (n=10,9,10,5)
|
11.68 percentage change
Geometric Coefficient of Variation 43.73
|
-2.75 percentage change
Geometric Coefficient of Variation 63.10
|
21.33 percentage change
Geometric Coefficient of Variation 38.76
|
-18.28 percentage change
Geometric Coefficient of Variation 52.94
|
|
Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks
change in week 52 (n=10,7,9,5)
|
10.02 percentage change
Geometric Coefficient of Variation 39.08
|
-12.52 percentage change
Geometric Coefficient of Variation 44.16
|
25.39 percentage change
Geometric Coefficient of Variation 36.77
|
-10.73 percentage change
Geometric Coefficient of Variation 37.20
|
Adverse Events
Part A-placebo of Pradigastat (LCQ908) Regimen
Serious events: 1 serious events
Other events: 11 other events
Deaths: 0 deaths
Part A-20mg Pradigastat (LCQ908) Regimen
Serious events: 6 serious events
Other events: 12 other events
Deaths: 0 deaths
Part A-40mg Pradigastat (LCQ908) Regimen
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Part A: Pradigastat (LCQ908) Regimen- From Study A2212
Serious events: 2 serious events
Other events: 5 other events
Deaths: 0 deaths
Part B-placebo of Pradigastat (LCQ908) Regimen
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Part B-20mg Pradigastat (LCQ908) Regimen
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Part B- Pradigastat (LCQ908) Regimen- From Study A2212
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Part B-40 mg Pradigastat (LCQ908)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A-placebo of Pradigastat (LCQ908) Regimen
n=11 participants at risk
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 A-20mg Pradigastat (LCQ908) Regimen
n=12 participants at risk
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 A-40mg Pradigastat (LCQ908) Regimen
n=10 participants at risk
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 A: Pradigastat (LCQ908) Regimen- From Study A2212
n=5 participants at risk
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-placebo of Pradigastat (LCQ908) Regimen
n=5 participants at risk
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.
|
Part B-20mg Pradigastat (LCQ908) Regimen
n=6 participants at risk
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.
|
Part B- Pradigastat (LCQ908) Regimen- From Study A2212
n=4 participants at risk
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.
|
Part B-40 mg Pradigastat (LCQ908)
n=4 participants at risk
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.
|
|---|---|---|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
ANAEMIA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Cardiac disorders
CORONARY ARTERY STENOSIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
PANCREATITIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
40.0%
2/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
PANCREATITIS ACUTE
|
9.1%
1/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
GASTROENTERITIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
POSTOPERATIVE WOUND INFECTION
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
DIABETES MELLITUS INADEQUATE CONTROL
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
HYPOGLYCAEMIA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
MALNUTRITION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
Other adverse events
| Measure |
Part A-placebo of Pradigastat (LCQ908) Regimen
n=11 participants at risk
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 A-20mg Pradigastat (LCQ908) Regimen
n=12 participants at risk
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 A-40mg Pradigastat (LCQ908) Regimen
n=10 participants at risk
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 A: Pradigastat (LCQ908) Regimen- From Study A2212
n=5 participants at risk
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-placebo of Pradigastat (LCQ908) Regimen
n=5 participants at risk
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.
|
Part B-20mg Pradigastat (LCQ908) Regimen
n=6 participants at risk
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.
|
Part B- Pradigastat (LCQ908) Regimen- From Study A2212
n=4 participants at risk
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.
|
Part B-40 mg Pradigastat (LCQ908)
n=4 participants at risk
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.
|
|---|---|---|---|---|---|---|---|---|
|
Skin and subcutaneous tissue disorders
XANTHOMA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
ANAEMIA
|
9.1%
1/11
|
8.3%
1/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Cardiac disorders
CARDIAC FAILURE
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Congenital, familial and genetic disorders
ABNORMAL PALMAR/PLANTAR CREASES
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
25.0%
1/4
|
0.00%
0/4
|
|
Ear and labyrinth disorders
EAR PAIN
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Ear and labyrinth disorders
VERTIGO POSITIONAL
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
ABDOMINAL DISCOMFORT
|
9.1%
1/11
|
16.7%
2/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
ABDOMINAL DISTENSION
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
18.2%
2/11
|
8.3%
1/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
16.7%
1/6
|
25.0%
1/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
0.00%
0/11
|
0.00%
0/12
|
20.0%
2/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
CONSTIPATION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
DIARRHOEA
|
72.7%
8/11
|
91.7%
11/12
|
50.0%
5/10
|
80.0%
4/5
|
40.0%
2/5
|
16.7%
1/6
|
75.0%
3/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
DYSPEPSIA
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
FAECAL INCONTINENCE
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
FOOD POISONING
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
GASTRITIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
NAUSEA
|
18.2%
2/11
|
33.3%
4/12
|
30.0%
3/10
|
40.0%
2/5
|
40.0%
2/5
|
33.3%
2/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
PANCREATITIS
|
0.00%
0/11
|
16.7%
2/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
PANCREATITIS ACUTE
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
STEATORRHOEA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
TONGUE CYST
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Gastrointestinal disorders
VOMITING
|
9.1%
1/11
|
16.7%
2/12
|
10.0%
1/10
|
20.0%
1/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
General disorders
FATIGUE
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
General disorders
NON-CARDIAC CHEST PAIN
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
General disorders
THIRST
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
General disorders
VESSEL PUNCTURE SITE INDURATION
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
BRONCHITIS
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
CYSTITIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
GASTROENTERITIS
|
9.1%
1/11
|
16.7%
2/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
INFLUENZA
|
54.5%
6/11
|
16.7%
2/12
|
20.0%
2/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
LUNG INFECTION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
NASOPHARYNGITIS
|
36.4%
4/11
|
33.3%
4/12
|
0.00%
0/10
|
20.0%
1/5
|
40.0%
2/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
PNEUMONIA
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
POST PROCEDURAL INFECTION
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
RESPIRATORY TRACT INFECTION
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
SINUSITIS
|
9.1%
1/11
|
16.7%
2/12
|
10.0%
1/10
|
20.0%
1/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
9.1%
1/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
URINARY TRACT INFECTION
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
VAGINAL INFECTION
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Infections and infestations
VULVOVAGINAL MYCOTIC INFECTION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
CONTUSION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
FALL
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
FIBULA FRACTURE
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
LIGAMENT RUPTURE
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
MUSCLE STRAIN
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
POST PROCEDURAL INFLAMMATION
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Injury, poisoning and procedural complications
WOUND
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Investigations
CARDIAC MURMUR
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Investigations
CAROTID BRUIT
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Investigations
WEIGHT DECREASED
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
GOUT
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
HYPOGLYCAEMIA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
HYPOKALAEMIA
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
VITAMIN B COMPLEX DEFICIENCY
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
VITAMIN D DEFICIENCY
|
0.00%
0/11
|
16.7%
2/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
9.1%
1/11
|
8.3%
1/12
|
20.0%
2/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL PAIN
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
MYALGIA
|
9.1%
1/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
NECK PAIN
|
0.00%
0/11
|
8.3%
1/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
OSTEOARTHRITIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
9.1%
1/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
ROTATOR CUFF SYNDROME
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
SYNOVIAL CYST
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
TENDONITIS
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
APHONIA
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
DIZZINESS
|
9.1%
1/11
|
8.3%
1/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
HEADACHE
|
9.1%
1/11
|
16.7%
2/12
|
20.0%
2/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
MIGRAINE
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
20.0%
1/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
PARAESTHESIA
|
0.00%
0/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
RESTLESS LEGS SYNDROME
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
SCIATICA
|
0.00%
0/11
|
0.00%
0/12
|
20.0%
2/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Nervous system disorders
SYNCOPE
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Psychiatric disorders
AGITATION
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Psychiatric disorders
DEPRESSED MOOD
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Psychiatric disorders
INSOMNIA
|
0.00%
0/11
|
8.3%
1/12
|
10.0%
1/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Psychiatric disorders
STRESS
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Reproductive system and breast disorders
DYSMENORRHOEA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Reproductive system and breast disorders
ERECTILE DYSFUNCTION
|
0.00%
0/11
|
8.3%
1/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Reproductive system and breast disorders
PREMATURE MENOPAUSE
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
25.0%
1/4
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
9.1%
1/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
PHARYNGEAL INFLAMMATION
|
9.1%
1/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
PNEUMOTHORAX
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
RHINORRHOEA
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
ALOPECIA
|
9.1%
1/11
|
0.00%
0/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
PSORIASIS
|
0.00%
0/11
|
8.3%
1/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
RASH
|
0.00%
0/11
|
16.7%
2/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Vascular disorders
AORTIC ANEURYSM
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
20.0%
1/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Vascular disorders
HOT FLUSH
|
0.00%
0/11
|
0.00%
0/12
|
0.00%
0/10
|
0.00%
0/5
|
0.00%
0/5
|
16.7%
1/6
|
0.00%
0/4
|
0.00%
0/4
|
|
Vascular disorders
HYPERTENSION
|
0.00%
0/11
|
16.7%
2/12
|
10.0%
1/10
|
0.00%
0/5
|
0.00%
0/5
|
0.00%
0/6
|
0.00%
0/4
|
0.00%
0/4
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER