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A Study to Assess the Hemodynamic Effects, Safety, Tolerability, and Pharmacokinetics of Intravenous APD418 in Adult Participants With Heart Failure With Reduced Ejection Fraction

Phase 2
Terminated
Conditions
Acute Heart Failure With Reduced Ejection Fraction
Interventions
Drug: APD418
Drug: Placebo
Registration Number
NCT05139615
Lead Sponsor
Pfizer
Brief Summary

The purpose of this study is to evaluate the safety, pharmacokinetics, and effect on cardiac function of intravenous APD418 in adult participants with heart failure with reduced ejection fraction (HFrEF).

Detailed Description

This study has an adaptive design, in which dose escalation in Part A will inform dose expansion in Part B. Part A is a single-ascending dose, placebo-controlled study planned to consist of 5 cohorts evaluating 5 doses of APD418. Part B is a parallel-treatment group study planned to evaluate 2 doses of APD418 and placebo. Participants in Part A cannot participate in Part B.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
22
Inclusion Criteria
  • Advanced chronic Heart Failure with Reduced Ejection Fraction (HFrEF), defined as left ventricular ejection fraction (LVEF) less than or equal to (≤) 35% at Screening, including documented history of HFrEF (LVEF ≤ 35%) for at least 4 months prior to Screening
  • New York Heart Association Class II-IV
  • Cardiac index ≤ 2.5 liters per minute per square meter (L/min/m^2) and pulmonary capillary wedge pressure ≥ 15 millimeters of mercury (mm Hg) at Day 1
  • Body mass index 18.0 to 37.0 kilograms per square meter (kg/m^2), inclusive, and body weight < 150 kg at Screening and Day 1
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Exclusion Criteria
  • Hemodynamically unstable at Day 1 or in the opinion of the Investigator likely to progress to becoming hemodynamically unstable during the course of the study
  • Treated with carvedilol or at a dose higher than a total of 25 milligrams per day any time within 72 hours of Day 1 through the end of the in-clinic observation Post-dose Period.
  • Receiving any mechanical (respiratory or circulatory) or renal support therapy at Screening or Day 1
  • Systolic Blood Pressure ≤ 90 millimeter of mercury (mm Hg) or ≥ 160 mm Hg, or Heart Rate < 50 beats per minute (bpm) or > 110 bpm, at Screening or Day 1
  • Recently treated with inotropic, intravenous (IV) vasoactive or IV diuretic therapy, or expected to require such therapy with these drugs any time from Day 1 through the end of study conduct.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
APD418 (Part A: Dose Cohort 1-5)APD418-
APD418 (Part B: Dose Group 1 and 2)APD418-
Placebo (Part A: Cohort 1-5 and Part B)Placebo-
Primary Outcome Measures
NameTimeMethod
Part A: Change in Cardiac Index (CI) Measured by Right Heart Catheterization (RHC) From Baseline to End of Intravenous (IV) Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

Cardiac index (CI) is a hemodynamic parameter that relates the cardiac output (CO) from left ventricle in one minute to body surface area (BSA), thus relating heart performance to the body size of the participant. It was measured by RHC.

Secondary Outcome Measures
NameTimeMethod
Part A: Change in Left Ventricular Ejection Fraction (LVEF) Measured by ECHO From Baseline to End of IV Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

LVEF is the central measure of left ventricular systolic function. LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). This was measured by ECHO.

Part A: Change in Fractional Shortening (FS) Measured by ECHO From Baseline to End of IV Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

FS was calculated by measuring the percentage reduction in left ventricular diameter during systole. This was measured by ECHO.

Part A: Change in Left Ventricular End-Systolic and Left Ventricular End-Diastolic Diameter Measured by ECHO From Baseline to End of IV Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

Left ventricular end-systolic diameter and left ventricular end-diastolic diameter were measured using ECHO.

Part A: Change in Left Ventricular Global Longitudinal Strain (LVGLS) and Left Ventricular Global Circumferential Strain (LVGCS) Measured by ECHO From Baseline to End of IV Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

Left ventricular global strain is the average strain of the cardiac chamber wall, where LVGLS presents longitudinal shortening as a percentage (change in length as a proportion to baseline length). LVGCS measures the chamber deformation along the circumference of the cardiac wall in a tangential xy-direction and similarly presents the circumferential shortening as a percentage. Both the parameters were measured by ECHO.

Part A: Change in CI Measured by RHC at 0.5, 1, 2, 3, 4 and 5 Hours During 6 Hour IV InfusionBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4 and 5 hours of IV infusion

CI is a hemodynamic parameter that relates the CO from left ventricle in one minute to BSA, thus relating heart performance to the body size of the participant. It was measured by RHC.

Part A: Change in Cardiac Output (CO) Measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

Change in CO measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 hours during the 6-hour IV infusion was reported in this outcome measure.

Part A: Change in Stroke Volume (SV), Left Ventricular End-Systolic Volume (LVESV) and Left Ventricular End-Diastolic Volume (LVEDV) Measured by Echocardiogram (ECHO) From Baseline to End of IV Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

SV is the volume of blood pumped from the left ventricle per beat. LVESV is the volume of blood in the left ventricle at the end of contraction and at diastole. LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. All these parameters were measured by ECHO.

Part A: Change in Stroke Volume Index (SVI) Measured by ECHO From Baseline to End of IV Infusion at 6 HoursBaseline (within 2 hours prior to start of study treatment administration) up to 6 Hours (end of IV infusion)

SVI was calculated as stroke volume divided by BSA. This was measured by ECHO.

Part A: Change in Pulmonary Capillary Wedge Pressure (PCWP), Right Atrial Pressure (RAP), Systolic Pulmonary Arterial Pressure/Diastolic Pulmonary Arterial Pressure (PAS/PAD) Measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

PCWP estimated the left atrial pressure and was the pressure measured by wedging a pulmonary artery catheter with an inflated balloon into a small pulmonary arterial branch. PCWP was assessed by 2 successive measurements at least 10 minutes apart. RAP is the blood pressure in the right atrium of the heart. Change in PCWP, RAP, PAS/PAD at 0.5, 1, 2, 3, 4, 5 and 6 hours during the 6-hour IV infusion was reported in this outcome measure. All the parameters were measured by RHC.

Part A: Change in Pulmonary Artery Pulsatility Index (PAPi) Measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

PAPi is a hemodynamic parameter that is derived from right atrial and pulmonary artery pulse pressures. PAPi = (PAS - PAD)/right atrial pressure. Change in PAPi measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 hours during the 6-hour IV infusion was reported in this outcome measure.

Part A: Change in Systemic Vascular Resistance Measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

Systemic vascular resistance (SVR) is the amount of force exerted on circulating blood by the vasculature of the body. SVR was calculated as 80\*(mean arterial pressure - mean venous pressure) divided by cardiac output. Change in SVR measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 hours during the 6-hour IV infusion was reported in this outcome measure.

Part A: Change in Systemic Vascular Resistance Index (SVRI) Measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

SVRI was calculated by dividing the difference between mean arterial pressure and central venous pressure by cardiac index and multiplying by 80. Change in SVRI measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 hours during the 6 hour IV infusion was reported in this outcome measure.

Part A: Change in Pulmonary Vascular Resistance (PVR) Measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

PVR is the resistance against blood flow from the pulmonary artery to the left atrium. Change in PVR measured by RHC at 0.5, 1, 2, 3, 4, 5 and 6 hours during the 6 hour IV infusion was reported in this outcome measure.

Part A: Change in Systolic Blood Pressure (SBP), Diastolic Blood Pressure (DBP) and Mean Arterial Pressure (MAP) at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

SBP, DBP and MAP were measured in a supine or seated position after participant had at least 5 minutes of rest. MAP is the average pressure of the blood circulating through a participant's arteries during the cardiac cycle. MAP was derived by using the following formula: DBP + 1/3(SBP-DBP).

Part A: Change in Heart Rate at 0.5, 1, 2, 3, 4, 5 and 6 HoursBaseline (within 2 hours prior to start of study treatment administration), 0.5, 1, 2, 3, 4, 5 and 6 hours of IV infusion

Heart rate was measured in a supine or seated position after participant had at least 5 minutes of rest.

Part A: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)From start of study treatment on Day 1 up to Day 9

An AE was any untoward medical occurrence that did not necessarily have a causal relationship with study treatment. TEAEs were defined as those AEs with onset after start date/timepoint of study drug administration.

Part A: Area Under the Plasma Concentration Time Curve From Time Zero to 6 Hours (AUC[0-6]) of APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6 hours post infusion start

AUC \[0-6\] was reported in this outcome measure.

Part A: Area Under the Plasma Concentration Time Curve From Time Zero to Last Quantifiable Plasma Concentration (AUCLast) of APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

AUClast was reported in this outcome measure.

Part A: Area Under the Plasma Concentration Time Curve From Time Zero up to Infinity (AUC[0-Infinity]) of APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

AUC \[0-infinity\] was reported in this outcome measure.

Part A: Maximum Observed Plasma Concentration (Cmax) of APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

Cmax of APD418 was reported in this outcome measure.

Part A: Terminal Elimination Half-Life (t1/2) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

Terminal t1/2 of APD418 was reported in this outcome measure.

Part A: Distributional Half-Life (t1/2a) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

Distributional t1/2 of APD418 was reported in this outcome measure.

Part A: Time to Maximum Observed Plasma Concentration (Tmax) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

Tmax of APD418 was reported in this outcome measure.

Part A: Total Clearance (CL) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

IV CL of APD418 was reported in this outcome measure.

Part A: Total Volume of Distribution Based on the Terminal Phase (Vdz) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

Vdz of APD418 was reported in this outcome measure.

Part A: Volume of Distribution at Steady State (Vdss) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

Vdss of APD418 was reported in this outcome measure.

Part A: Mean Residence Time From Time Zero to Time of Last Quantifiable Plasma Concentration (MRTlast) for APD418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6, 6.083, 6.167, 6.25, 6.5, 7, 8, 10, 18 and 24 hours post infusion start

MRTlast of APD418 was reported in this outcome measure.

Part A: Average Plasma Concentration During Dosing Interval (Cave) for ADP418Pre-dose, 0.5, 1, 2, 3, 4, 5, 6 hours post infusion start

Average plasma concentration calculated over the 6-hour infusion time was reported in this outcome measure.

Part A: Renal Clearance (CLr) for ADP418Pre-dose (0) to 24 hours post infusion start, collected over 0 to 6 hour, 6 to 10 hour and 10 to 24-hour intervals

CLr for APD418 was reported in this outcome measure.

Trial Locations

Locations (21)

Institute for Cardiovascular Diseases of Vojvodina

🇷🇸

Sremska Kamenica, Serbia

Clinical Center of Kragujevac

🇷🇸

Kragujevac, Serbia

Healthcare Center Uzice

🇷🇸

Užice, Serbia

James A. Haley Veterans' Hospital

🇺🇸

Tampa, Florida, United States

Immanuel Hospital Bernau Brandenburg Heart Center

🇩🇪

Bernau bei Berlin, Brandenburg, Germany

Kerckhoff-Klinik Forschungsgesellschaft GmbH

🇩🇪

Bad Nauheim, Germany

Health Science Center Utah

🇺🇸

Salt Lake City, Utah, United States

Interbalkan European Medical Center

🇬🇷

Pylaia, Thessaloniki, Greece

Clinical Hospital Center Bezanijska Kosa

🇷🇸

Belgrade, Serbia

Institute for Cardiovascular Diseases Dedinje

🇷🇸

Belgrade, Serbia

Universitatsmedizin Greifwald

🇩🇪

Greifswald, Germany

UnityPoint Health - Methodist Hospital

🇺🇸

Peoria, Illinois, United States

UTHealth

🇺🇸

Houston, Texas, United States

American Heart of Poland S.A.

🇵🇱

Gniezno, Poland

University of Wisconsin

🇺🇸

Madison, Wisconsin, United States

Konstantinopouleio General Hospital of Nea Ionia - Patision ''Agia Olga''

🇬🇷

Nea Ionia, Athens, Greece

Uniwersytecki Szpital Kliniczny im Jana Mikulicza-Radeckiego

🇵🇱

Wroclaw, Poland

University Clinical Centre of Serbia

🇷🇸

Belgrade, Serbia

General University Hospital of Larissa

🇬🇷

Larissa, Greece

Clinical Hospital Centre Zemun

🇷🇸

Belgrade, Serbia

Krakowski Szpital Specjalistyczny im. Jana Pawla II

🇵🇱

Krakow, Poland

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