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TerminatedNCT01589237Updated Nov 15, 2016Results posted

Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

A Phase 3 interventional study of LCQ908 in Familial Chylomicronemia Syndrome (FCS) (HLP Type I), sponsored by Novartis Pharmaceuticals. Terminated at 10 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-11-15.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment

Why this study was terminated
Interim analysis suggested that size of benefit anticipated from continued participation of patients in Part B no longer supported trial extension beyond Part A
Phase
Phase 3
Study type
Interventional
Enrollment
38
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

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).

Read the detailed description

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.

02

Conditions studied

  • Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

Keywords

  • Familial Chylomicronemia Syndrome (FCS) (HLP type I)
  • LCQ908
03

In context

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,711 studies on the registry; 218 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 435 (75%) have results posted.

Browse Novartis Pharmaceuticals studies →All Novartis studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Written informed consent must be obtained before any assessment is performed.
  2. Subjects that either discontinue prematurely or complete the CLCQ908B2302 study after 52 weeks or FCS subjects who have previously completed study CLCQ908A2212.

Exclusion criteria

Exclusion Criteria:

  1. Subjects discontinued from the CLCQ908B2302 study for serious, potentially study drug related adverse events.
  2. Subjects from the CLCQ908B2302 study who have developed any other contraindication to participation (for example, renal failure)
  3. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
  4. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
  5. Subjects with type 1 diabetes mellitus or type 2 diabetes mellitus if HbA1C is ≥ 8.5%.
  6. Treatment with fish oil preparations within 4 weeks prior to randomization.
  7. Treatment with bile acid binding resins (i.e., colesevelam, etc) within 4 weeks prior to randomization.
  8. Treatment with fibrates within 8 weeks prior to randomization. Washout may occur following screening if required.
  9. Glybera [alipogene tiparvovec (AAV1-LPLS447X )] gene therapy exposure within the two years prior to screening.
  10. eGFR \<45 ml/min/1.73m2 or history of chronic renal disease.

Other protocol defined inclusion/exclusion criteria may apply.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
38 participants (actual)

Study arms

  • Experimental
    LCQ908

    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.

    Drug: LCQ908

Interventions

06

What researchers measure

Primary outcomes

  1. Number of Patients With Any Adverse Events, Serious Adverse Events and Death

    Time frame: 52 weeks

Secondary outcomes

  1. 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.

    Time frame: Baseline, Week 12, 24 and 52

  2. 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.

    Time frame: Baseline, Week 12, 24 and 52

  3. 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.

    Time frame: Baseline, Week 12, 24 and 52

  4. 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.

    Time frame: Baseline, Week 12, 24 and 52

  5. 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.

    Time frame: Baseline, Week 12, 24 and 52

  6. 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.

    Time frame: Baseline, Week 12, 24 and 52

  7. 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.

    Time frame: Baseline, Week 12, 24 and 52

  8. 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.

    Time frame: Baseline, Week 12, 24 and 52

07

Results

Posted Nov 15, 2016
Limitations and caveats
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.

Participant flow

Part A (52 Weeks)
Participant flow — Part A (52 Weeks)
MilestonePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Started1112105
Completed9985
Not completed2320
Withdrew: Subject/guardian decision2320
Part B (Planned for 78 Week-terminated)
Participant flow — Part B (Planned for 78 Week-terminated)
MilestonePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Started5644
Completed0000
Not completed5644
Withdrew: Subject/guardian decision0100
Withdrew: Study terminated by sponsor5543
Withdrew: Physician decision0001

Outcome measures

PrimaryNumber of Patients With Any Adverse Events, Serious Adverse Events and Death
Time frame:
52 weeks
Reported as:
Number · Participants
Number of Patients With Any Adverse Events, Serious Adverse Events and Death
ParticipantsPlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
At least one Adverse Event (any)1112105
At least one serious AE1622
Death0000
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Triglyceride Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=10,11,10,5)1.63 ± 45.19-5.80 ± 66.1043.94 ± 52.66-19.36 ± 42.82
change in week 24 (n=10,10,9,5)-14.59 ± 52.33-36.19 ± 64.8032.54 ± 87.83-26.05 ± 31.50
change in week 52 (n=9,8,9,5)16.46 ± 29.27-30.03 ± 78.5292.15 ± 60.21-9.20 ± 43.34
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Cholesterol Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=10,11,10,5)-5.58 ± 36.38-4.76 ± 36.2618.76 ± 31.54-10.42 ± 24.68
change in week 24 (n=10,10,9,5)-10.54 ± 27.87-21.54 ± 24.987.45 ± 37.28-13.87 ± 25.64
change in week 52 (n=9,8,9,5)5.75 ± 17.51-13.76 ± 36.1040.95 ± 34.10-6.84 ± 22.55
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
HDL: Change in week 12 (n=10,11,10,5)-14.13 ± 30.083.37 ± 24.75-5.99 ± 22.19-7.09 ± 27.11
Non HDL: change in week 12 (n=10,11,10,5)-5.37 ± 39.61-7.72 ± 42.4020.70 ± 34.18-10.57 ± 26.16
HDL: change in week 24 (n=10,10,9,5)-7.11 ± 19.41-1.83 ± 31.366.67 ± 22.19-15.60 ± 37.69
Non HDL: change in week 24 (n=10,10,9,5)-11.29 ± 29.53-25.22 ± 31.497.17 ± 40.50-14.01 ± 28.04
HDL: change in week 52 (n=9,8,9,5)-10.85 ± 19.788.11 ± 29.35-7.33 ± 27.63-21.41 ± 22.91
Non HDL: change in week 52 (n=9,8,9,5)8.06 ± 21.42-17.68 ± 42.6545.25 ± 37.45-6.09 ± 24.59
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Glycerol Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=10,11,10,5)-26.56 ± 72.88-15.50 ± 73.330.68 ± 75.40-46.83 ± 105.58
change in week 24 (n=10,10,9,5)-40.00 ± 67.28-46.97 ± 62.46-36.26 ± 133.58-56.99 ± 96.06
change in week 52 (n=9,8,9,5)-38.15 ± 70.10-31.96 ± 64.76-28.52 ± 101.85-37.02 ± 81.67
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=10,11,10,5)-23.11 ± 53.05-17.85 ± 62.4853.09 ± 44.83-46.58 ± 128.38
change in week 24 (n=10,10,9,5)-20.35 ± 80.16-30.44 ± 62.0136.18 ± 64.97-42.74 ± 108.73
change in week 52 (n=9,8,9,5)-16.99 ± 43.34-7.69 ± 54.2179.15 ± 49.37-18.29 ± 104.77
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=11,11,10,5)-3.24 ± 23.822.55 ± 15.096.58 ± 17.042.95 ± 33.54
change in week 24 (n=10,10,10,5)4.80 ± 14.161.41 ± 17.455.57 ± 25.695.11 ± 28.49
change in week 52 (n=10,8,9,5)4.51 ± 19.2110.01 ± 16.284.43 ± 19.34-0.82 ± 16.79
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=11,11,10,5)-4.03 ± 79.8733.24 ± 79.20109.67 ± 52.50-30.03 ± 61.78
change in week 24 (n=10,10,10,5)9.13 ± 30.79-10.23 ± 58.91105.52 ± 71.45-35.04 ± 31.11
change in week 52 (n=10,8,9,5)56.25 ± 57.65-22.63 ± 107.21135.33 ± 71.1127.75 ± 57.77
SecondaryChanges 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.

Time frame:
Baseline, Week 12, 24 and 52
Reported as:
Geometric mean · percentage change
Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks
percentage changePlacebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212
Change in week 12 (n=9,11,9,5)-15.75 ± 41.5015.10 ± 58.53-8.34 ± 32.733.33 ± 32.46
change in week 24 (n=10,9,10,5)-2.75 ± 63.1021.33 ± 38.76-18.28 ± 52.9411.68 ± 43.73
change in week 52 (n=10,7,9,5)-12.52 ± 44.1625.39 ± 36.77-10.73 ± 37.2010.02 ± 39.08

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Part A-placebo of Pradigastat (LCQ908) Regimen—1/11 (9.1%)11/11 (100%)
Part A-20mg Pradigastat (LCQ908) Regimen—6/12 (50%)12/12 (100%)
Part A-40mg Pradigastat (LCQ908) Regimen—2/10 (20%)10/10 (100%)
Part A: Pradigastat (LCQ908) Regimen- From Study A2212—2/5 (40%)5/5 (100%)
Part B-placebo of Pradigastat (LCQ908) Regimen—0/5 (0%)4/5 (80%)
Part B-20mg Pradigastat (LCQ908) Regimen—0/6 (0%)3/6 (50%)
Part B- Pradigastat (LCQ908) Regimen- From Study A2212—0/4 (0%)3/4 (75%)
Part B-40 mg Pradigastat (LCQ908)—0/4 (0%)0/4 (0%)
Most frequent serious events
Showing 10 of 11
Most frequent serious events
EventPart A-placebo of Pradigastat (LCQ908) RegimenPart A-20mg Pradigastat (LCQ908) RegimenPart A-40mg Pradigastat (LCQ908) RegimenPart A: Pradigastat (LCQ908) Regimen- From Study A2212Part B-placebo of Pradigastat (LCQ908) RegimenPart B-20mg Pradigastat (LCQ908) RegimenPart B- Pradigastat (LCQ908) Regimen- From Study A2212Part B-40 mg Pradigastat (LCQ908)
PANCREATITISGastrointestinal disorders0/111/120/102/50/50/60/40/4
PANCREATITIS ACUTEGastrointestinal disorders1/110/121/100/50/50/60/40/4
POSTOPERATIVE WOUND INFECTIONInfections and infestations0/110/121/100/50/50/60/40/4
ANAEMIABlood and lymphatic system disorders0/111/120/100/50/50/60/40/4
CORONARY ARTERY STENOSISCardiac disorders0/111/120/100/50/50/60/40/4
GASTROENTERITISInfections and infestations0/111/120/100/50/50/60/40/4
ASPARTATE AMINOTRANSFERASE INCREASEDInvestigations0/111/120/100/50/50/60/40/4
DEHYDRATIONMetabolism and nutrition disorders0/111/120/100/50/50/60/40/4
DIABETES MELLITUS INADEQUATE CONTROLMetabolism and nutrition disorders0/111/120/100/50/50/60/40/4
HYPOGLYCAEMIAMetabolism and nutrition disorders0/111/120/100/50/50/60/40/4
Most frequent other events
Showing 10 of 91
Most frequent other events
EventPart A-placebo of Pradigastat (LCQ908) RegimenPart A-20mg Pradigastat (LCQ908) RegimenPart A-40mg Pradigastat (LCQ908) RegimenPart A: Pradigastat (LCQ908) Regimen- From Study A2212Part B-placebo of Pradigastat (LCQ908) RegimenPart B-20mg Pradigastat (LCQ908) RegimenPart B- Pradigastat (LCQ908) Regimen- From Study A2212Part B-40 mg Pradigastat (LCQ908)
DIARRHOEAGastrointestinal disorders8/1111/125/104/52/51/63/40/4
INFLUENZAInfections and infestations6/112/122/100/50/50/60/40/4
NAUSEAGastrointestinal disorders2/114/123/102/52/52/60/40/4
NASOPHARYNGITISInfections and infestations4/114/120/101/52/51/60/40/4
ABNORMAL PALMAR/PLANTAR CREASESCongenital, familial and genetic disorders0/110/120/100/50/50/61/40/4
ABDOMINAL PAINGastrointestinal disorders2/111/121/100/50/51/61/40/4
PREMATURE MENOPAUSEReproductive system and breast disorders0/110/120/100/50/50/61/40/4
ANAEMIABlood and lymphatic system disorders1/111/120/101/50/50/60/40/4
ABDOMINAL DISTENSIONGastrointestinal disorders0/110/120/101/50/50/60/40/4
ABDOMINAL PAIN UPPERGastrointestinal disorders0/110/122/101/50/50/60/40/4

Baseline characteristics

Full analysis set (FAS) - All subjects in the extension study who were in full analysis set in either study LCQ908A2212/NCT01146522 or LCQ908B2302/NCT01514461.

Age, Continuous
Age, Continuous(Years)Placebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212Total
Mean52.9 ± 10.2244.1 ± 14.2643.6 ± 84.5352.2 ± 12.7247.6 ± 11.43
Sex: Female, Male
Sex: Female, Male(Participants)Placebo of Pradigastat (LCQ908) Regimen20 mg Pradigastat (LCQ908) Regimen40 mg Pradigastat (LCQ908) RegimenPradigastat (LCQ908) Regimen- From Study A2212Total
Female562316
Male668222
08

Study locations

10 sites
  • Novartis Investigative Site
    Seatlle, Washington 98104, United States
  • Novartis Investigative Site
    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
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 15, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT01589237
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
May 1, 2012
Start date
Feb 2013
Primary completion
Jul 2015
Completion
Jul 2015
Results posted
Nov 15, 2016
Last update
Nov 15, 2016

Study contacts

Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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