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The Clinical Study of the Safety and Efficacy of Istaroxime in Treatment of Acute Decompensated Heart Failure

Phase 2
Completed
Conditions
Acute Decompensated Heart Failure
Interventions
Drug: Placebo
Registration Number
NCT02617446
Lead Sponsor
Windtree Therapeutics
Brief Summary

To assess the safety, tolerability and efficacy of two different doses of istaroxime, a new agent with lusitropic and inotropic activities that improves the cardiac contraction-relaxation cycle. The 2 doses of istaroxime (0.5 and 1.0 µg/kg/min) will be infused via i. v. for 24 hours in comparison with placebo, in treatment of Chinese and Italian patients with Acute Decompensated Heart Failure.

Detailed Description

To assess the safety, tolerability and efficacy of two different doses of istaroxime (0.5 and 1.0 µg/kg/min) in comparison with placebo, including cardiovascular and renal tolerability, as well as changes in biological markers such as N-terminal prohormone brain natriuretic peptide (NT-proBNP) and troponin T (cTnT). The study will be conducted in 96 Chinese and Italian patients with Acute Decompensated Heart Failure. This is a phase II, multicenter, randomized, double-blind, placebo-controlled, parallel group study. Patients were randomly assigned to one of two doses of istaroxime or placebo in a 2:1 ratio within two sequential cohorts of 60 patients each. This 31-day study includes a screening period (Days -1), a treatment period (Day 1), a post-treatment period (Days 2-4), and a follow-up period (which includes one patient visit on Day 30).

In all the Italian patients and in a subset of Chinese patients pharmacokinetics and metabolism of istaroxime shall also be studied.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
120
Inclusion Criteria

Patients who fulfill the following inclusion criteria at screening will be considered for the study:

  1. Signed informed consent;
  2. Male or female patients 18-85 years (inclusive);
  3. Admission for a recurrent acute decompensated heart failure (ADHF) episode with dyspnea at rest or minimal exertion and need of intravenous diuretic therapy (≥40 mg iv. furosemide);
  4. Systolic blood pressure between 90 and 125 mmHg (limits included) without signs or symptoms of hypoperfusion including cardiogenic shock, cold extremities and peripheral vasoconstriction, oliguria/anuria, signs of cerebral hypo perfusion such as confusion;
  5. Left ventricular (LV) Ejection fraction (EF) ≤ 40 % measured by 2D-Echocardiography
  6. E/Ea ratio >10
  7. BNP ≥ 350pg/mL or NT-pro-BNP ≥1400 pg/mL
  8. Adequate echocardiography window (defined as visualization of at least 13/16 segment of the left ventricle);
Exclusion Criteria

Any of the following criteria established at screening would render a patient ineligible for the study:

  1. Pregnant or breast-feeding women (women of child bearing potential must have the results of a negative pregnancy test recorded prior to study drug administration)

  2. Current (within 12 hours prior to screening) or planned (through the completion of study drug infusion) treatment with any iv. therapies, including vasodilators (including nitrates or nesiritide), positive inotropic agents and vasopressors

  3. Current or need of mechanical support (intra-aortic balloon pump, endotracheal intubation, mechanical ventilation, or any ventricular assist device),

  4. Ongoing treatment with oral digoxin. Patient treated with digoxin within the last week, can be randomised if the plasma concentration of digoxin is tested before randomization and its value will be less than 0.5 ng/ml.

  5. History of hypersensitivity to the study medication or any related medication

  6. Diagnosis of cardiogenic shock within the past month;

  7. Acute coronary syndrome or stroke within the past 3 months;

  8. Coronary artery bypass graft or percutaneous coronary intervention within the past month or planned in the next month;

  9. Primary hypertrophic or restrictive cardiomyopathy or systemic illness known to be associated with infiltrative heart disease;

  10. Cor pulmonale or other causes of right-sided heart failure (HF) not related to left ventricular dysfunction;

  11. Pericardial constriction or active pericarditis;

  12. Atrial fibrillation with marked irregularities of heart rhythm;

  13. Life threatening ventricular arrhythmia or implantable cardioverter-defibrillator (ICD) shock within the past month;

  14. Cardiac resynchronization therapy (CRT), ICD, or pacemaker implantation within the past month;

  15. Valvular disease as primary cause of HF;

  16. Heart rate >120 bpm or < 50 bpm

  17. Acute respiratory distress syndrome or ongoing sepsis;

  18. Fever >38°

  19. History of bronchial asthma or porphyria;

  20. Donation or loss of blood equal to or exceeding 500 mL, during the 8 weeks before administration of study medication;

  21. Positive testing for HIV, Hepatitis B and/or Hepatitis C;

  22. Participation in another interventional study within the past 30 days;

  23. The following laboratory exclusion criteria, verified based on results obtained within the last 24 hours of hospitalization:

    1. Serum creatinine > 3.0 mg/dl (> 265 µmol/L);
    2. Aspartate aminotransferase (ASAT) or alanine aminotransferase (ALAT) > 3 x upper limit of normal,
    3. Hemoglobin (Hb) < 10 g/dL,
    4. Platelet count < 100,000/µL,
    5. Serum potassium > 5.3 mmol/L or < 3.8 mmol/L,

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboIV infusion of placebo for 24 hours
Istaroxime 0.5 µg/kg/minIstaroximeThe istaroxime treatment dosed at 0.5 µg/kg/min via IV infusion for 24 hours
Istaroxime 1.0 µg/kg/minIstaroximeThe istaroxime treatment dosed at 1.0 µg/kg/min via IV infusion for 24 hours
Primary Outcome Measures
NameTimeMethod
Change in E/Ea Ratio24 hours

Change from baseline at 24 hours in the unitless ratio of E (cm/sec) to Ea (or e') (cm/sec) as measured by echocardiogram.

The endpoint is the Tissue Doppler echocardiography showing measurement of mitral E/Ea ratio for assessment of diastolic dysfunction. Initially mitral E wave is measured. After that, color Tissue Doppler (tissue velocity imaging or TVI) mode is switched on to assess tissue Doppler. The cursor is placed over the medial mitral annulus and tissue Doppler tracing obtained. This allows Ea velocity to be measured. Higher values are suggestive of a worse outcome; less than 8 is normal.

Secondary Outcome Measures
NameTimeMethod
Change in LVEF24 hours

Change from baseline at 24 hours in LV ejection fraction (LVEF) by tissue Doppler

Change in SVI24 hours

Change from baseline at 24 hours in stroke volume index (SVI) by tissue Doppler

Change in LV End Systolic Volume24 hours

Change from baseline in left ventricular end systolic volume (LVESV) by tissue Doppler

Change in Dyspnea24 hours

Measured using a visual analog scale (0 to 100). Higher scores indicate less dyspnea.

Change in LV End Diastolic Volume24 hours

Change from baseline in left ventricular end diastolic volume (LVEDV) by tissue Doppler

Change in E/A Ratio24 hours

Change from baseline at 24 hours in E/A ratio by tissue Doppler

Trial Locations

Locations (11)

University of Milano-Bicocca

🇮🇹

Milan, Italy

University and Civil Hospital of Brescia

🇮🇹

Brescia, Italy

Lanzhou University No.2 Hospital

🇨🇳

Lanzhou, Gansu, China

The First Hospital of Lanzhou University

🇨🇳

Lanzhou, Gansu, China

The General Hospital Of Shenyang Military Region

🇨🇳

Shenyang, Liaoning, China

Beijing Chao Yang Hospital

🇨🇳

Beijing, China

Renmin Hospital of Wuhan University

🇨🇳

Wuhan, Hubei, China

Jiangsu Province People's Hospital

🇨🇳

Nanjing, Jiangsu, China

The First Affiliated Hospital Of Xi'an Jiaotong University

🇨🇳

Xi'an, Shaanxi, China

Fuwai Hospital Chinese Academy of Medical Sciences

🇨🇳

Beijing, China

The 307th Hospital of Chinese People's Liberation Army

🇨🇳

Beijing, China

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