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NCT02678312

Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LCZ696 Followed by a 52-week, Double-blind Study of LCZ696 Compared With Enalapril in Pediatric Patients With Heart Failure

Completed Phase 2, PHASE3 Results posted Last updated 10 February 2023
What this trial tests

Phase 2, PHASE3 trial testing LCZ696 in Pediatric Heart Failure in 393 participants. Completed in 3 January 2022.

Timeline
3 November 2016
Primary endpoint
3 January 2022
3 January 2022

Quick facts

Lead sponsorNovartis Pharmaceuticals
PhasePhase 2, PHASE3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingdouble
Primary purposetreatment
Enrollment393
Start date3 November 2016
Primary completion3 January 2022
Estimated completion3 January 2022
Sites98 locations across Italy, Jordan, Finland, Japan, Taiwan, Poland, South Korea, Lebanon

Drugs / interventions tested

Conditions studied

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

Adults 1 Month to 17, any sex, with Pediatric Heart Failure. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Part 1: Pharmacokinetics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Maximum Drug Concentration in Plasma (Cmax) Primary · Age group 1: Pre-dose and 0.5, 1, 2, 4, 8, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2; Age groups 2 and 3: Pre-dose and 1, 2, 4, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2

The analyses of Cmax was based on plasma concentrations of three sacubitril/valsartan analytes (AHU377 (sacubitril), LBQ657 (sacubitrilat), and valsartan). The plasma levels of sacubitril/valsartan analytes were determined using a validated LCMS/MS method with a lower limit of quantitation (LLOQ) of 1 ng/mL for sacubitril, 20 ng/mL for LBQ657, and 10 ng/mL for valsartan. The PK parameters were determined using the non-compartmental method(s).

Sacubitril
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)523± 390
LCZ696: 0.8 mg/kg (Age Group 2)179± 97
LCZ696: 3.1 mg/kg (Age Group 1)1970± 1666
LCZ696: 3.1 mg/kg (Age Group 2)549± 298
LCZ696: 0.4 mg/kg (Age Group 3)124± 80
LCZ696: 1.6 mg/kg (Age Group 3)433± 181
LBQ657
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1951± 839
LCZ696: 0.8 mg/kg (Age Group 2)1359± 711
LCZ696: 3.1 mg/kg (Age Group 1)6707± 1887
LCZ696: 3.1 mg/kg (Age Group 2)5453± 1032
LCZ696: 0.4 mg/kg (Age Group 3)632± 89
LCZ696: 1.6 mg/kg (Age Group 3)2326± 629
Valsartan
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1271± 1011
LCZ696: 0.8 mg/kg (Age Group 2)1112± 583
LCZ696: 3.1 mg/kg (Age Group 1)4035± 1678
LCZ696: 3.1 mg/kg (Age Group 2)4935± 1268
LCZ696: 0.4 mg/kg (Age Group 3)440± 275
LCZ696: 1.6 mg/kg (Age Group 3)2487± 1564
Part 1: Pharmacokinetics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Time to Maximum Plasma Concentration (Tmax) Primary · Age group 1: Pre-dose and 0.5, 1, 2, 4, 8, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2; Age groups 2 and 3: Pre-dose and 1, 2, 4, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2

The analyses of Tmax was based on plasma concentrations of three sacubitril/valsartan analytes (AHU377 (sacubitril), LBQ657 (sacubitrilat), and valsartan). The plasma levels of sacubitril/valsartan analytes were determined using a validated LCMS/MS method with a lower limit of quantitation (LLOQ) of 1 ng/mL for sacubitril, 20 ng/mL for LBQ657, and 10 ng/mL for valsartan. The PK parameters were determined using the non-compartmental method(s).

Sacubitril
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.1± 1.3
LCZ696: 0.8 mg/kg (Age Group 2)1.2± 0.5
LCZ696: 3.1 mg/kg (Age Group 1)0.8± 0.3
LCZ696: 3.1 mg/kg (Age Group 2)1.2± 0.4
LCZ696: 0.4 mg/kg (Age Group 3)1.1± 0.1
LCZ696: 1.6 mg/kg (Age Group 3)1.0± 0.0
LBQ657
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)4.0± 2.0
LCZ696: 0.8 mg/kg (Age Group 2)2.9± 1.1
LCZ696: 3.1 mg/kg (Age Group 1)2.9± 1.1
LCZ696: 3.1 mg/kg (Age Group 2)3.6± 3.2
LCZ696: 0.4 mg/kg (Age Group 3)2.8± 1.6
LCZ696: 1.6 mg/kg (Age Group 3)3.6± 0.9
Valsartan
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.7± 1.1
LCZ696: 0.8 mg/kg (Age Group 2)2.1± 1.4
LCZ696: 3.1 mg/kg (Age Group 1)2.6± 1.0
LCZ696: 3.1 mg/kg (Age Group 2)1.9± 0.4
LCZ696: 0.4 mg/kg (Age Group 3)1.8± 1.5
LCZ696: 1.6 mg/kg (Age Group 3)1.8± 1.3
Part 1: Pharmacokinetics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUCinf) Primary · Age group 1: Pre-dose and 0.5, 1, 2, 4, 8, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2; Age groups 2 and 3: Pre-dose and 1, 2, 4, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2

The analyses of AUCinf was based on plasma concentrations of three sacubitril/valsartan analytes (AHU377 (sacubitril), LBQ657 (sacubitrilat), and valsartan). The plasma levels of sacubitril/valsartan analytes were determined using a validated LCMS/MS method with a lower limit of quantitation (LLOQ) of 1 ng/mL for sacubitril, 20 ng/mL for LBQ657, and 10 ng/mL for valsartan. The PK parameters were determined using the non-compartmental method(s).

Sacubitril
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)690± 410
LCZ696: 0.8 mg/kg (Age Group 2)494± 286
LCZ696: 3.1 mg/kg (Age Group 1)3021± 1814
LCZ696: 3.1 mg/kg (Age Group 2)1214± 684
LCZ696: 0.4 mg/kg (Age Group 3)270± 182
LCZ696: 1.6 mg/kg (Age Group 3)1063± 266
LBQ657
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)48264± 22939
LCZ696: 0.8 mg/kg (Age Group 2)31042± 17259
LCZ696: 3.1 mg/kg (Age Group 1)150440± 49515
LCZ696: 3.1 mg/kg (Age Group 2)127625± 35634
LCZ696: 0.4 mg/kg (Age Group 3)15835± 2912
LCZ696: 1.6 mg/kg (Age Group 3)62377± 16035
Valsartan
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)13540± 12962
LCZ696: 0.8 mg/kg (Age Group 2)11036± 7031
LCZ696: 3.1 mg/kg (Age Group 1)40733± 21003
LCZ696: 3.1 mg/kg (Age Group 2)48561± 21163
LCZ696: 0.4 mg/kg (Age Group 3)3923± 1424
LCZ696: 1.6 mg/kg (Age Group 3)26170± 16826
Part 1: Pharmacokinetics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Number of Participants With Area Under the Plasma Concentration-time Curve From Time Zero to Last (AUClast) Primary · Age group 1: Pre-dose and 0.5, 1, 2, 4, 8, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2; Age groups 2 and 3: Pre-dose and 1, 2, 4, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2

As prespecified in protocol and SAP the analysis of this outcome measure was done based on dose of LCZ696 administered within the different age groups.

GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)7
LCZ696: 0.8 mg/kg (Age Group 2)8
LCZ696: 3.1 mg/kg (Age Group 1)7
LCZ696: 3.1 mg/kg (Age Group 2)6
LCZ696: 0.4 mg/kg (Age Group 3)4
LCZ696: 1.6 mg/kg (Age Group 3)5
Part 1: Pharmacokinetics of LCZ696 Analytes (Sacubitril, and Valsartan): Clearance From Plasma (CL/F) Primary · Age group 1: Pre-dose and 0.5, 1, 2, 4, 8, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2; Age groups 2 and 3: Pre-dose and 1, 2, 4, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2

The analyses was based on plasma concentrations of two sacubitril/valsartan analytes (AHU377 (sacubitril), and valsartan). The plasma levels of sacubitril/valsartan analytes were determined using a validated LCMS/MS method with a lower limit of quantitation (LLOQ) of 1 ng/mL for sacubitril, 20 ng/mL for LBQ657, and 10 ng/mL for valsartan. The PK parameters were determined using the non-compartmental method(s). CL/F was not estimated for LBQ657 as it is a metabolite.

Sacubitril
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)0.73± 0.35
LCZ696: 0.8 mg/kg (Age Group 2)1.19± 0.96
LCZ696: 3.1 mg/kg (Age Group 1)0.63± 0.28
LCZ696: 3.1 mg/kg (Age Group 2)1.67± 1.01
LCZ696: 0.4 mg/kg (Age Group 3)1.19± 1.11
LCZ696: 1.6 mg/kg (Age Group 3)1.67± 1.01
Valsartan
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)0.06± 0.05
LCZ696: 0.8 mg/kg (Age Group 2)0.07± 0.09
LCZ696: 3.1 mg/kg (Age Group 1)0.06± 0.06
LCZ696: 3.1 mg/kg (Age Group 2)0.04± 0.01
LCZ696: 0.4 mg/kg (Age Group 3)0.06± 0.02
LCZ696: 1.6 mg/kg (Age Group 3)0.05± 0.03
Part 1: Pharmacokinetics of LCZ696 Analytes (Sacubitril): Time Required to Drug Concentration to Decrease by Half (T 1/2) Primary · Age group 1: Pre-dose and 0.5, 1, 2, 4, 8, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2; Age groups 2 and 3: Pre-dose and 1, 2, 4, 10, optional 24 hours post-dose on Day 1 of Period 1 and 2

The analyses of T1/2 was based on plasma concentrations of sacubitril. The plasma levels of sacubitril/valsartan analytes were determined using a validated LCMS/MS method with a lower limit of quantitation (LLOQ) of 1 ng/mL for sacubitril, 20 ng/mL for LBQ657, and 10 ng/mL for valsartan. The PK parameters were determined using the non-compartmental method(s). T1/2 for other analytes of LCZ696 (LBQ657 and Valsartan) was not estimable due to the short sample collection timeframe.

GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.260.95 – 2.36
LCZ696: 0.8 mg/kg (Age Group 2)1.531.40 – 1.65
LCZ696: 3.1 mg/kg (Age Group 1)1.341.16 – 1.60
LCZ696: 3.1 mg/kg (Age Group 2)1.511.34 – 1.70
LCZ696: 1.6 mg/kg (Age Group 3)1.331.16 – 1.64
Part 1: Pharmacodynamics (PD) of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Change From Baseline in Plasma B-type Natriuretic Peptide (BNP) Primary · Baseline (0 hrs pre dose), 4 and 8 hrs post dose on Day 1 of Period 1 and Period 2

Biomarkers were used to assess the PD effects of LCZ696. Blood biomarkers of potential interest included plasma BNP. Biomarkers related to heart failure or the mechanism of action of the study drug were measured. Summary statistics for change from baseline at each time point is presented. The baseline assessment is defined as the last non-missing assessment (scheduled or unscheduled) prior to (the first dose time of the study drug within the dose associated period). For each post-dose time point, participants are included if and only if the participant has both pre-dose assessment and current

Baseline (0 hrs pre dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)100.8749.84 – 204.13
LCZ696: 0.8 mg/kg (Age Group 2)63.808.65 – 470.81
LCZ696: 3.1 mg/kg (Age Group 1)97.5251.59 – 184.32
LCZ696: 3.1 mg/kg (Age Group 2)120.515.21 – 2787.44
LCZ696: 0.4 mg/kg (Age Group 3)21.202.14 – 210.41
LCZ696: 1.6 mg/kg (Age Group 3)129.299.45 – 1768.43
Change From Baseline (4 hrs post dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.310.93 – 1.85
LCZ696: 0.8 mg/kg (Age Group 2)1.600.21 – 11.95
LCZ696: 3.1 mg/kg (Age Group 1)1.220.94 – 1.58
LCZ696: 3.1 mg/kg (Age Group 2)0.62NA – NA
LCZ696: 0.4 mg/kg (Age Group 3)0.770.56 – 1.05
LCZ696: 1.6 mg/kg (Age Group 3)1.090.61 – 1.93
Change From Baseline (8 hrs post dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.320.92 – 1.88
LCZ696: 0.8 mg/kg (Age Group 2)1.210.21 – 7.04
LCZ696: 3.1 mg/kg (Age Group 1)0.970.70 – 1.34
LCZ696: 3.1 mg/kg (Age Group 2)0.80NA – NA
LCZ696: 0.4 mg/kg (Age Group 3)0.590.27 – 1.30
LCZ696: 1.6 mg/kg (Age Group 3)0.550.15 – 2.02
Part 1: Pharmacodynamics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Change From Baseline in Plasma N-terminal Pro-brain Natriuretic Peptide (NTproBNP) Primary · Baseline (0 hrs pre dose) and optional 24 hrs post dosing on Day 1 of Period 1 and Period 2

Biomarkers were used to assess the PD effects of LCZ696. Blood biomarkers of potential interest included plasma NTproBNP. Biomarkers related to heart failure or the mechanism of action of the study drug were measured. Summary statistics for change from baseline at each time point is presented. The baseline assessment is defined as the last non-missing assessment (scheduled or unscheduled) prior to (the first dose time of the study drug within the dose associated period). For each post-dose time point, participants are included if and only if the participant has both pre-dose assessment and cur

Baseline (0 hrs pre dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)2385.341186.13 – 4796.98
LCZ696: 3.1 mg/kg (Age Group 1)2179.94932.77 – 5094.69
LCZ696: 0.4 mg/kg (Age Group 3)961.76125.65 – 7361.70
LCZ696: 1.6 mg/kg (Age Group 3)5086.37683.53 – 37849.47
Change From Baseline (24 hrs post dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)0.740.31 – 1.78
LCZ696: 0.4 mg/kg (Age Group 3)0.590.00 – 134.25
LCZ696: 1.6 mg/kg (Age Group 3)0.410.34 – 0.48
Part 1: Pharmacodynamics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Change From Baseline in Plasma Cyclic Guanosine Monophosphate (cGMP) Primary · Baseline (0 hrs pre dose), 4 and 8 hrs post dose on Day 1 of Period 1 and Period 2

Biomarkers were used to assess the PD effects of LCZ696. Blood biomarkers of potential interest included plasma cGMP. Biomarkers related to heart failure or the mechanism of action of the study drug were measured. Summary statistics for change from baseline at each time point is presented. The baseline assessment is defined as the last non-missing assessment (scheduled or unscheduled) prior to (the first dose time of the study drug within the dose associated period). For each post-dose time point, participants are included if and only if the participant has both pre-dose assessment and current

Baseline (0 hrs pre dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)18.1812.01 – 27.51
LCZ696: 0.8 mg/kg (Age Group 2)21.4112.83 – 35.71
LCZ696: 3.1 mg/kg (Age Group 1)12.208.13 – 18.32
LCZ696: 3.1 mg/kg (Age Group 2)24.5518.37 – 32.82
LCZ696: 0.4 mg/kg (Age Group 3)13.380.05 – 3883.59
LCZ696: 1.6 mg/kg (Age Group 3)22.8412.51 – 41.68
Change From Baseline (4 hrs post dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.300.89 – 1.90
LCZ696: 0.8 mg/kg (Age Group 2)0.900.50 – 1.62
LCZ696: 3.1 mg/kg (Age Group 1)1.541.12 – 2.10
LCZ696: 3.1 mg/kg (Age Group 2)1.020.52 – 2.01
LCZ696: 0.4 mg/kg (Age Group 3)0.800.00 – 618.02
LCZ696: 1.6 mg/kg (Age Group 3)0.780.27 – 2.22
Change From Baseline (8 hrs post dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.170.91 – 1.50
LCZ696: 0.8 mg/kg (Age Group 2)0.920.66 – 1.29
LCZ696: 3.1 mg/kg (Age Group 1)1.601.10 – 2.31
LCZ696: 3.1 mg/kg (Age Group 2)0.400.02 – 8.86
LCZ696: 0.4 mg/kg (Age Group 3)0.790.00 – 230.10
LCZ696: 1.6 mg/kg (Age Group 3)0.790.29 – 2.18
Part 1: Pharmacodynamics of LCZ696 Analytes (Sacubitril, LBQ657, and Valsartan): Change From Baseline in Urine cGMP Primary · Baseline (0 hrs pre dose), 4 to 8 hrs post dose on Day 1 of Period 1 and Period 2

Biomarkers were used to assess the PD effects of LCZ696. Blood biomarkers of potential interest included urine cGMP. Biomarkers related to heart failure or the mechanism of action of the study drug were measured. Summary statistics for change from baseline at each time point is presented. The baseline assessment is defined as the last non-missing assessment (scheduled or unscheduled) prior to (the first dose time of the study drug within the dose associated period). For each post-dose time point, participants are included if and only if the participant has both pre-dose assessment and current

Baseline (0 hrs pre dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1055.56352.98 – 3156.55
LCZ696: 0.8 mg/kg (Age Group 2)1349.91118.48 – 15380.33
LCZ696: 3.1 mg/kg (Age Group 1)914.57311.65 – 2683.88
LCZ696: 3.1 mg/kg (Age Group 2)1123.6975.21 – 16789.03
LCZ696: 0.4 mg/kg (Age Group 3)485.00NA – NA
LCZ696: 1.6 mg/kg (Age Group 3)386.32134.04 – 1113.42
Change From Baseline (4 to 8 hrs post dose)
GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)1.420.38 – 5.32
LCZ696: 0.8 mg/kg (Age Group 2)0.800.02 – 27.03
LCZ696: 3.1 mg/kg (Age Group 1)1.790.65 – 4.96
LCZ696: 3.1 mg/kg (Age Group 2)2.170.10 – 45.16
LCZ696: 0.4 mg/kg (Age Group 3)0.920.02 – 51.75
LCZ696: 1.6 mg/kg (Age Group 3)1.980.51 – 7.63
Part 2: Percentage of Participants With Worst Event in Each Category Based on Global Ranking Primary · Up to 52 weeks

Global ranking is based on 5 categories ranking worst to best outcome:Category 1:Death; United Network for Organ Sharing(UNOS)status 1A listing for heart transplant or equivalent; ventricular assist device(VAD)/extracorporeal membrane oxygenation(ECMO)/mechanical ventilation/intra-aortic balloon pump requirement for life support at end of study. Category 2:Worsening HF(WHF);defined by signs and symptoms of WHF that requires an intensification of HF therapy. Category 3:Worsened; worse New York Heart Association(NYHA)/Ross or worse Patient Global Impression of Severity(PGIS); and further ranking

Category 1
GroupValue95% CI
Part 2: LCZ69610.16
Part 2: Enalapril15.96
Category 2
GroupValue95% CI
Part 2: LCZ6969.63
Part 2: Enalapril4.79
Category 3
GroupValue95% CI
Part 2: LCZ6966.95
Part 2: Enalapril5.85
Category 4
GroupValue95% CI
Part 2: LCZ69620.86
Part 2: Enalapril26.60
Category 5
GroupValue95% CI
Part 2: LCZ69639.57
Part 2: Enalapril35.64
Missing
GroupValue95% CI
Part 2: LCZ69612.83
Part 2: Enalapril11.17
Part 1: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) Secondary · From first dose to 30 days after last dose of study drug in Part 1

An adverse event (AE) is any untoward medical occurrence associated with use of a drug in humans, whether considered drug related or not, that occurs after a participant provides informed consent. TEAEs during part 1 are defined as any recorded AE with its start date (recorded or imputed) later than or equal to the date of the first dose of the study drug within part 1 and its start date prior to or equal to the end date of the part 1.

GroupValue95% CI
LCZ696: 0.8 mg/kg (Age Group 1)28.57
LCZ696: 0.8 mg/kg (Age Group 2)50.00
LCZ696: 3.1 mg/kg (Age Group 1)28.57
LCZ696: 3.1 mg/kg (Age Group 2)50.00
LCZ696: 0.4 mg/kg (Age Group 3)50.00
LCZ696: 1.6 mg/kg (Age Group 3)80.00
Part 1: Dose Cohort S50.00

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were collected from first dose of study treatment plus 30 days post treatment up to approximately one year.. Reporting threshold: 1%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Part 1: Dose Cohort 1
Serious: 2/17 (12%)
Deaths: 0/17
Part 1: Dose Cohort 2
Serious: 1/18 (6%)
Deaths: 0/18
Part 1: Dose Cohort S
Serious: 0/2 (0%)
Deaths: 0/2
Part 2: LCZ696
Serious: 69/187 (37%)
Deaths: 8/187
Part 2: Enalapril
Serious: 62/188 (33%)
Deaths: 12/188

Serious adverse events (128 terms)

ReactionSystemPart 1: Dose Cohort 1Part 1: Dose Cohort 2Part 1: Dose Cohort SPart 2: LCZ696Part 2: Enalapril
Cardiac failureCardiac disorders
VomitingGastrointestinal disorders
PneumoniaInfections and infestations
Cardiac arrestCardiac disorders
Cardiac failure acuteCardiac disorders
Cardiac failure congestiveCardiac disorders
PyrexiaGeneral disorders
Upper respiratory tract infectionInfections and infestations
SeizureNervous system disorders
HypotensionVascular disorders
ArrhythmiaCardiac disorders
Ventricular tachycardiaCardiac disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
Atrial thrombosisCardiac disorders
Chest painGeneral disorders
BronchiolitisInfections and infestations
InfluenzaInfections and infestations
Viral upper respiratory tract infectionInfections and infestations
DehydrationMetabolism and nutrition disorders
HyperkalaemiaMetabolism and nutrition disorders
HypoglycaemiaMetabolism and nutrition disorders
SyncopeNervous system disorders
Acute kidney injuryRenal and urinary disorders
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
Other adverse events (147 terms — click to expand)

ReactionSystemPart 1: Dose Cohort 1Part 1: Dose Cohort 2Part 1: Dose Cohort SPart 2: LCZ696Part 2: Enalapril
CoughRespiratory, thoracic and mediastinal disorders
VomitingGastrointestinal disorders
Upper respiratory tract infectionInfections and infestations
PyrexiaGeneral disorders
NasopharyngitisInfections and infestations
DiarrhoeaGastrointestinal disorders
DizzinessNervous system disorders
HeadacheNervous system disorders
HypotensionVascular disorders
FatigueGeneral disorders
Abdominal painGastrointestinal disorders
BronchitisInfections and infestations
InfluenzaInfections and infestations
Glomerular filtration rate decreasedInvestigations
GastroenteritisInfections and infestations
RhinitisInfections and infestations
Abdominal pain upperGastrointestinal disorders
NauseaGastrointestinal disorders
Decreased appetiteMetabolism and nutrition disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
Oropharyngeal painRespiratory, thoracic and mediastinal disorders
AnaemiaBlood and lymphatic system disorders
ConstipationGastrointestinal disorders
Chest painGeneral disorders
Oedema peripheralGeneral disorders
RhinorrhoeaRespiratory, thoracic and mediastinal disorders
Cardiac failureCardiac disorders
COVID-19Infections and infestations
PharyngitisInfections and infestations
Blood creatinine increasedInvestigations
SARS-CoV-2 test negativeInvestigations
HyperkalaemiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
Pain in extremityMusculoskeletal and connective tissue disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
RashSkin and subcutaneous tissue disorders
Gastrooesophageal reflux diseaseGastrointestinal disorders
ToothacheGastrointestinal disorders
Otitis mediaInfections and infestations
Respiratory tract infectionInfections and infestations

Most-reported serious reactions: Cardiac failure, Vomiting, Pneumonia, Cardiac arrest, Cardiac failure acute, Cardiac failure congestive, Pyrexia, Upper respiratory tract infection.

Data from ClinicalTrials.gov NCT02678312 adverse events section.

Sponsor's own description

This study consists of two parts (Part 1 and Part 2). The purpose of Part 1 is to evaluate the way the body absorbs, distributes, metabolizes and removes the drug LCZ696. This will help determine the proper dose of LCZ696 for Part 2 of the study. The purpose for Part 2 is to compare the effectiveness and safety of LCZ696 with enalapril in a double-blind manner, in pediatric heart failure patients over 52 weeks of treatment.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Sacubitril/Valsartan in Pediatric Heart Failure (PANORAMA-HF): A Randomized, Multicenter, Double-Blind Trial.
    Shaddy R, Burch M, Kantor PF, Solar-Yohay S, et al · · 2024 · cited 30× · PMID 39319469 · DOI 10.1161/circulationaha.123.066605
  2. Sacubitril/valsartan in heart failure: latest evidence and place in therapy.
    Kaplinsky E. · · 2016 · cited 30× · PMID 27803793 · DOI 10.1177/2040622316665350
  3. Potential Expanded Indications for Neprilysin Inhibitors.
    Riddell E, Vader JM. · · 2017 · cited 22× · PMID 28281174 · DOI 10.1007/s11897-017-0327-y
  4. Baseline Characteristics of Pediatric Patients With Heart Failure Due to Systemic Left Ventricular Systolic Dysfunction in the PANORAMA-HF Trial.
    Shaddy R, Burch M, Kantor PF, Solar-Yohay S, et al · · 2023 · cited 16× · PMID 36601956 · DOI 10.1161/circheartfailure.122.009816
  5. Exploring the Food and Drug Administration's review and approval of Entresto (sacubitril/valsartan).
    Eadie AL, Brunt KR, Herder M. · · 2021 · cited 12× · PMID 34087050 · DOI 10.1002/prp2.794
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