Use of Bone Marrow Derived Stem Cell and G-CSF With Circulatory Assistance in the Treatment of DCM

Phase 2Active Not RecruitingNCT03572660

Barts & The London NHS Trust20 enrolled

Overview

DCM Support is recruiting patients with dilated cardiomyopathy and heart failure symptoms. The goal of this clinical trial is to examine whether treatment with a patient's own stem cells can improve their heart function and alleviate heart failure symptoms. * Stem cells will be collected from bone marrow in the patient's hip under local anaesthetic. * The stem cells will be infused into the arteries that supply blood to the heart under local anaesthetic. * A mini heart pump will be used to take the strain off the heart during the procedure. * The follow-up involves a phone call at 1 month and clinic visits at 3 and 12 months

DCM SUPPORT is a single centre, single arm clinical trial taking place at St Bartholomew's Hospital in London, UK. * It is recruiting patients with dilated cardiomyopathy and ongoing heart failure symptoms * All patients undergo a bone marrow aspiration after 5 days of subcutaneous G-CSF injections * After cell processing, bone marrow-derived mononuclear cells are infused into the coronary arteries using the stop-flow technique. An intra-procedural Impella CP device is used to support the circulation. * The primary endpoint is change in left ventricular ejection fraction at 3 months as measured by cardiac CT.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Dilated Cardiomyopathy

Eligibility

Sex: ALLMin age: 18 YearsMax age: 85 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients with a confirmed diagnosis of dilated cardiomyopathy under the supervision of a physician or a heart failure nurse specialist. * NYHA class ≥ 2 symptoms despite having received optimal medical therapy and appropriate device therapy, as per clinical guidelines for an interval of at least 3 months. * No other treatment options available as part of the current best standard of care. * LVEF ≤35% on any imaging modality performed as part of the screening phase. Exclusion Criteria: * Congenital heart disease. * Clinically significant valvular heart disease. * Patients who are not suitable for a Percutaneous Mechanical Support Device (E.g. unsuitable femoral artery anatomy, unable able to lie flat for prolonged time to accommodate the stem cell infusion \& presence of LV thrombus) * Weight of patient that exceeds the maximum limit of the cardiac catheterisation laboratory table / CT scanner. * Cardiomyopathy 2o to a reversible cause that has not been treated e.g. thyroid disease, alcohol abuse, hypophosphataemia, hypocalcaemia, cocaine abuse, selenium toxicity \& chronic uncontrolled tachycardia. * Cardiomyopathy in association with a neuromuscular disorder e.g. Duchenne's progressive muscular dystrophy. * Previous cardiac surgery. * Contra-indication for bone marrow aspiration (thrombocytopaenia - platelet count \<80 x 10(9)/L or extensive surgical scarring/anatomical deformity at site of bone marrow puncture). * Known active infection on admission as defined by a temperature \>37.5°C or on a short course of antibiotics. * An active infection of hepatitis B, hepatitis C, syphilis or HTLV * Known HIV infection * Chronic inflammatory disease requiring on-going medication. * Concomitant disease with a life expectancy of less than one year * Follow-up impossible (no fixed abode, etc.) * Neoplastic disease without documented remission within the past 5 years. * Patients on renal replacement therapy. * Subjects of childbearing potential unless βHCG negative and are on adequate contraception during the trial. * Patients falling into the vulnerable category or lacking capacity * Patients who are unable to understand or read written English will be excluded from the trial. * Killip Class III or above

Outcomes

Primary Outcomes

Change in left ventricular ejection fraction

Change in left ventricular ejection fraction as measured by cardiac CT

Time frame: Baseline to 3 months

Secondary Outcomes

Change in left ventricular ejection fraction

Change in left ventricular ejection fraction as measured by cardiac CT

Time frame: Baseline to 12 months

Change in exercise capacity

Change in exercise capacity as assessed by a 6-minute walk test

Time frame: Baseline to 3 and 12 months

Change in heart failure symptoms

Change in heart failure symptoms as measured by NYHA classification

Time frame: Baseline to 3 and 12 months

Change in quality of life as assessed by Minnesota Living with Heart Failure Questionnaire scores

Change in quality of life as measured by MLHFQ (The 21-item MLHFQ uses a 6-point Likert scale, where 0 = no, 1= very little and 5= very much. The questions are intended to be representative of the ways heart failure can affect physical and emotional dimensions of quality of life)

Time frame: Baseline to 3 and 12 months

Change in quality of life as measured by EuroQol-5 Dimension 5 Levels questionnaires

Change in quality of life as measured by EQ-5D-5L questionnaires (the scale measures quality of life on a 5-component scale including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression)

Time frame: Baseline to 3 and 12 months

Procedural safety as assessed by in-hospital procedural related morbidity/mortality

Time frame: In-hospital procedural time

Change in biochemical markers of heart failure

Change in biochemical markers of heart failure as measured by change in NT-proBNP

Time frame: Baseline to 3 and 12 months

Assessment of rates of MACE (cumulative & individual components)

Rates of MACE (all-cause death, myocardial infarction, hospitalisation for heart failure, major arrhythmias \[defined as VT and VF\])

Time frame: 3 and 12 months

Assessment of rates of stroke

Time frame: 3 and 12 months

Assessment of peri-procedural myocardial infarction

Assessment of peri-procedural myocardial infarction as per SCAI definition measured by change in troponin (MI defined by increase in troponin \>70 times upper limit of normal from baseline).

Time frame: Day 0 and Day 6

Change in renal function

Change in renal function from baseline at 3 and 12 months as measured by creatinine levels.

Time frame: Baseline to 3 and 12 months

Change in inflammatory markers

Change in inflammatory markers as measured by change in C-reactive protein

Time frame: Baseline to 3 and 12 months

Use of Bone Marrow Derived Stem Cell and G-CSF With Circulatory Assistance in the Treatment of DCM

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes