MedPath

Cell Therapy in HFpEF

Phase 2
Completed
Conditions
Heart Failure With Normal Ejection Fraction
Interventions
Biological: Cell Therapy
Registration Number
NCT02923609
Lead Sponsor
University Medical Centre Ljubljana
Brief Summary

The primary objective of the study is to investigate safety and efficacy of transendocardial CD34+ cell therapy in patients with HFpEF by evaluating changes in myocardial structure and function, patient exercise capacity and clinical outcome.

The safety end-points include serious adverse events (SAEs), defined as any serious event that may result in persistent or significant disability or incapacity and included death, heart transplantation, sustained ventricular arrhythmia (ventricular tachycardia or ventricular fibrillation), and heart failure exacerbation requiring hospitalization.

Detailed Description

In all patients, peripheral blood stem cells will be mobilized by daily subcutaneous injections of G-CSF (10 mcg/kg, divided b.i.d) for 5 days. Peripheral blood stem cells will then be collected with Miltenyi cell separator (Miltenyi Biotech, Germany) and the magnetic cell separator Isolex 300i (Nexell Therapeutics Inc., California, USA) will be used for the immunomagnetic positive selection of the CD34+ cells.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
30
Inclusion Criteria
  • Preserved left ventricular systolic function on echocardiography (LVEF>50%)
  • Evidence of diastolic dysfunction by echocardiography (E/e'>15)
  • Symptoms of heart failure
  • NT-proBNP levels >300 pg/ml
  • absence of permanent atrial fibrillation
Exclusion Criteria
  • acute multi-organ failure
  • history of any malignant disease within 5 years
  • diminished functional capacity due to non-cardiac co-morbidities (COPD, PAOD, morbid obesity)
  • pregnancy

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
SC GroupCell TherapyIn Phase 1 of the study, all patients will be treated with optimization of medical therapy for 6 months. Thereafter, all patients will cros over to Phase 2 of the study, where they will receive transendocardial CD34+ cell therapy. Follow-up of Phase 2 will last for 6 months. At the time of enrollment (6 months before cell therapy), at time of cell therapy, and 6 months thereafter we will perform detailed clinical evaluation, laboratory assays, echocardiography, 6-minute walk test, and measure plasma levels of N-terminal pro B-type natriuretic peptide (NT-proBNP).
Primary Outcome Measures
NameTimeMethod
Change in left ventricular filling pressures (E/e') assessed by echocardiographyBaseline, 6 months and 1 year

The E/e' ratio will be calculated from early transmitral velocity divided by peak left ventricular relaxation velocity for estimation of the left ventricular filling pressure.

Secondary Outcome Measures
NameTimeMethod
Change in diastolic dysfunction gradeBaseline, 6 months and 1 year

Diastolic dysfunction will be graded according based on E/A ratio and left atrial pressure estimation.

Change in exercise capacityBaseline, 6 months and 1 year

6-minute walk test will be performed by a blinded observer according to the standard protocol.

Change in NT-proBNP levelsBaseline, 6 months and 1 year

NT-proBNP assays will be performed at a central independent laboratory, blinded to the patient's clinical data using a commercially available kit (Roche Diagnostics, Mannheim, Germany).

Change in systolic strainBaseline, 6 months and 1 year

Left ventricular longitudinal strains will be analyzed by speckle tracking echocardiography from apical four-chamber, two-chamber, and long-axis views.

Trial Locations

Locations (1)

University Medical Center Ljubljana

🇸🇮

Ljubljana, Slovenia

© Copyright 2025. All Rights Reserved by MedPath