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Efficacy and Safety of Allogeneic Mesenchymal Precursor Cells (Rexlemestrocel-L) for the Treatment of Heart Failure.

Phase 3
Completed
Conditions
Chronic Heart Failure
Registration Number
NCT02032004
Lead Sponsor
Mesoblast, Inc.
Brief Summary

The primary objective of this study is to determine whether transendocardial delivery of allogeneic human bone marrow-derived mesenchymal precursor cells (MPCs \[rexlemestrocel-L\]) is effective in the treatment of chronic heart failure (HF) due to left ventricular (LV) systolic dysfunction.

Detailed Description

The purpose of this study is to evaluate the efficacy and safety of a single transendocardial delivery in the cardiac catheterization laboratory of human bone marrow-derived allogeneic MPCs (rexlemestrocel-L) for improvement in clinical outcomes (heart failure major adverse cardiac events \[HF-MACE\]), preventing further adverse cardiac remodeling (left ventricular end systolic volume \[LVESV\] and left ventricular end-diastolic volume \[LVEDV\]), and increasing exercise capacity (six-minute walking test \[6MWT\]) in patients with chronic HF due to LV systolic dysfunction of either ischemic or nonischemic etiology who have received optimal medical/revascularization therapy.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
566
Inclusion Criteria
  • The patient is 18 to 80 years of age, inclusive; both men and women will be enrolled.
  • The patient has a diagnosis of chronic HF of ischemic or nonischemic etiology for at least 6 months
  • The patient is on stable, optimally tolerated dosages of HF therapies including beta-blockers (approved for country-specific usage), angiotensin-converting enzyme (ACE) inhibitors or angiotensin-receptor blockers (ARBs), and/or aldosterone antagonists, without change in dose for at least 1 month before study intervention
  • The patient is on a stable, outpatient, oral diuretic dosing regimen in which the patient remains clinically stable during screening.
  • Other Criteria apply, please contact the investigator
Exclusion Criteria
  • The patient has NYHA Functional Class I or Functional Class IV symptoms.
  • Other Criteria apply, please contact the investigator

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
Time to recurrent non-fatal decompensated heart failure major adverse cardiac events (HF-MACE) that occur prior to the first terminal cardiac event (TCE).6 Month minimum
Secondary Outcome Measures
NameTimeMethod
Time-to-first terminal cardiac event (TCE)6 Month minimum
Total length of in-hospital stay in intensive care unit for non-fatal decompensated HF events6 Month minimum
Time-to-first HF-MACE (composite of hospital admissions for decompensated HF, urgent care outpatient HF visit, and successfully RCD events)6 Month minimum
Time-to-hospital admissions for non-fatal decompensated HF events6 Month minimum
Time-to-first HF-MACE (composite of hospital admissions for decompensated HF, urgent care outpatient HF visit, successfully RCD events or TCE)6 Month minimum
Time-to-cardiac death6 Month minimum
Time-to-all-cause death6 Month minimum
Time-to-urgent care outpatient HF visits6 Month minimum
Time-to-successfully resuscitated cardiac death (RCD) events6 Month minimum
Left Ventricular (LV) remodeling in LVESV determined by 2-D echocardiographyScreening, day 0 (post-procedure), months 3, 6, and 12, and every 12 months thereafter during study conduct (6 Month minimum)
Correlations between baseline LVESV <=100 mL and LVESV >100 mL and clinical outcomes6 Month minimum
Safety as assessed by occurrence of treatment-emergent adverse eventsScreening through 6 Month minimum
Safety as assessed by urinalysis (blood, glucose, ketones, total protein)Screening, day 10, months 1, 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)
Safety as assessed by vital signs (pulse, systolic blood pressure [BP], diastolic BP)Screening, day 0 (pre and post-procedure), day 1, day 10, months 1, 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)
Time-to-first non-fatal MI (myocardial infarction), non-fatal CVA (cerebrovascular attack) or coronary artery revascularization6 Month minimum
LV remodeling in LVEDV determined by 2-D echocardiographyScreening, day 0 (post-procedure), months 3, 6, and 12, and every 12 months thereafter during study conduct (6 Month minimum)
Overall Left Ventricular systolic performance as assessed by left ventricular ejection fraction (LVEF [radionuclide ventriculography {RVG} or echocardiogram])Screening, day 0 (post-procedure), months 3, 6, and 12, and every 12 months thereafter during study conduct (6 Month minimum)
Correlations between baseline LVESV <=100 mL and LVESV >100 mL and change in Month 6 to baseline LVESV and clinical outcomes6 Month minimum
Quality of Life Measure - European Quality of Life (EuroQoL) 5-dimensional (EQ-5D) questionnaireScreening, months 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)
Safety as assessed by rhythm analysis (specifically, ventricular arrhythmias) by interrogation of any implanted device capable of defibrillationDay 10, months 1, 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)
Safety as assessed by important cardiovascular events from adjudicated data6 Month minimum
Functional exercise capacity as assessed by 6 Minute Walk TestScreening, months 3, 6, and 12, and every 12 months thereafter during study conduct (6 Month minimum)
Functional status by New York Heart Association (NYHA) classScreening, months 3, 6, and 12, and every 12 months thereafter during study conduct (6 Month minimum)
Quality of Life Measure - Minnesota Living With Heart Failure (MLHF) questionnaireScreening, months 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)
Safety as assessed by occurrence of adverse events related to the index cardiac catheterization on Day 0Day 0 through discharge from Day 0 hospitalization
Safety as assessed by clinical laboratory tests (serum chemistry - ALT, AST, alkaline phosphate, GGT, LDH, BUN, creatinine, uric acid, total bilirubin - and hematology - hematocrit, hemoglobin, WBCs, eosinophils, ANC, platelet count)Screening, day 0 (post-procedure), day 10, months 1, 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)

ALT (alanine transaminase), AST (aspartate aminotransferase), GGT (gamma-glutamyl transferase), LDH (lactate dehydrogenase), BUN (blood urea nitrogen), WBCs (white blood cells), ANC (absolute neutrophil count)

Safety as assessed by 24-hr Holter monitoring (HR, rate & duration of arrhythmias, a-fib average rate, supra- & ventricular ectopy singles/couplets/runs/totals, sustained & non-sustained ventricular tachycardia, longest pauses RR duration, total pauses)Screening, day 0 (post-procedure), day 10, months 1 and 3
Safety as assessed by 12-lead electrocardiogram (ECG) findings - QT interval with Fridericia's correction (QTcF), heart rate-corrected QT interval (QTcB), QT, Q wave, R wave and S wave (QRS) complex, HR and T waves.Screening, day 0 (pre and post-procedure), day 1, day 10, months 1, 3, 6, and 12, and every 6 months thereafter during study conduct (6 Month minimum)
Safety as assessed by physical examination findings judged as clinically significant changes from baseline by the investigator or newly occurring abnormalities (including weight)Screening, month 12 and every 12 months thereafter until study conclusion (weight measured at screening, day 0 - pre and post-procedure, day 1, day 10, months 1, 3, 6 and 12 and every 6 months thereafter)
Safety as assessed by telemetry monitoring findings (clinically significant arrhythmias)Day 0 through Day 0 overnight post-procedure

Trial Locations

Locations (59)

Mesoblast Investigational Site 10757 - Cardiology, P.C.

🇺🇸

Birmingham, Alabama, United States

Mesoblast Investigational Site 13262 - University of Alabama at Birmingham Hospital

🇺🇸

Birmingham, Alabama, United States

Mesoblast Investigational Site 10779 - Mercy Gilbert Medical Center

🇺🇸

Gilbert, Arizona, United States

Mesoblast Investigational Site 10786 - Cardiovascular Associates of Mesa

🇺🇸

Mesa, Arizona, United States

Mesoblast Investigational Site 10756 - Mayo Clinic

🇺🇸

Phoenix, Arizona, United States

Mesoblast Investigational Site 13023 - University of Arizona Medical Center

🇺🇸

Tucson, Arizona, United States

Mesoblast Investigational Site 10754 - University of California, San Diego

🇺🇸

La Jolla, California, United States

Mesoblast Investigational Site 10759 - Scripps Clinic

🇺🇸

La Jolla, California, United States

Mesoblast Investigational Site 13265 - University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

Mesoblast Investigational Site 10775 - Cedars-Sinai Medical Care Foundation

🇺🇸

Los Angeles, California, United States

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Mesoblast Investigational Site 10757 - Cardiology, P.C.
🇺🇸Birmingham, Alabama, United States
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