Use of Bone Marrow Derived Stem Cell and G-CSF With Circulatory Assistance in the Treatment of DCM (DCM-Support)
Familial Dilated Cardiomyopathy / Posted 2 weeks ago
Brief Summary
The DCM SUPPORT study is a single centre, single arm study evaluating the change in left ventricular ejection fraction with the combines use of bone marrow derived stem cells and G-CSF with a percutaneous circulatory assistance for the treatment of dilated cardiomyopathy.
Detailed Description
The DCM SUPPORT study is a single centre, single arm, phase II pilot study. The primary endpoint will be a change in LVEF from baseline measured by a CT scan of the heart at 3 months. The secondary endpoints include change in LVEF from baseline measured by a CT scan of the heart at 12 months, in-hospital procedural related morbidity / mortality and assessment of major adverse cardiac events (MACE; death, heart attack, need for repeat heart revascularisation), change in exercise capacity and NYHA class at 3 months and 12 months respectively. The NYHA classification is a quantitative way of assessing a patient’s heart failure symptoms. It places patients into one of four categories (I – IV).
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Inclusion Criteria :
- Ages Eligible for Study: 18 Years to 85 Years (Adult, Older Adult)
- Sexes Eligible for Study: All
- Accepts Healthy Volunteers: No
- Patients with a confirmed diagnosis of dilated cardiomyopathy under the supervision of a physician or a heart failure nurse specialist.
- NYHA class III or IV 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 current best standard care.
- LVEF ≤30% on the cardiac CT scan performed as part of the screening phase.
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Exclusion Criteria :
- NYHA I-II.
- Documented latest ejection fraction >30% (any imaging modality)
- 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 catheter 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.
- Known active infection at time of randomisation.
- Positive virology tests.
- 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
- Study start date : December 24, 2018
- Study end date : July 30, 2024
- Wales-Based Study Contact : Please speak to your clinician
- Principal Investigator : Anthony Mathur (Barts & The London NHS Trust)