Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy (REGEN-DCM)

Barts & The London NHS Trust

Status and phase: Completed; Phase 2

Conditions: Dilated Cardiomyopathy

Treatments: Drug: granulocyte colony stimulating factor (GCSF); Procedure: bone marrow mononuclear cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01302171

2009-013112-12

Details and patient eligibility

About

A randomised, double-blind, placebo-controlled trial to evaluate the role of intracoronary injection of progenitor cells compared to placebo injection in patients with Dilated Cardiomyopathy who have been pre-treated with G-CSF (Granocyte™) injections for 5 days, and patients treated with a 5 day course of G-CSF (Granocyte™) injection only compared to placebo injection

Enrollment: 60 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Symptomatic patients with a confirmed diagnosis of dilated cardiomyopathy (NYHA II-III) attending their local 'Heart Failure clinic' who are on optimal heart failure treatment, under supervision from their physician or heart failure nurse specialist, and have no other treatment options
• Patients who are NYHA II that have been hospitalised with a dilated cardiomyopathy related condition
• Coronary angiography will be performed where necessary to confirm the diagnosis and ensure no other conventional treatment options are indicated
• Prior to recruitment to the study patients at risk of ventricular arrhythmia will have undergone electrophysiological assessment and appropriate clinical management (including implantable defibrillator insertion) where indicated (as per NICE guidelines)

Exclusion criteria

• NYHA I
• Referral hospitals most recent documented ejection fraction of >45% (any imaging modality)
• The presence of cardiogenic shock
• The presence of acute left and/or right sided pump failure as judged by the presence of pulmonary oedema and/or new peripheral oedema
• Known severe pre-existent left ventricular dysfunction (with a documented ejection fraction of <10% from referral hospital) prior to randomisation
• Congenital cardiac disease
• Cardiomyopathy secondary 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
• Known infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), HTLV or syphilis.
• Chronic inflammatory disease requiring ongoing medication
• Serious known concomitant disease with a life expectancy of less than one year
• Follow-up impossible (no fixed abode, etc)
• Patients with an irregular heart rhythm (AF allowed if paced in a regular rhythm)
• Patients with renal impairment (Creatinine >200mmol/L)
• Neoplastic disease without documented remission within the past 5 years
• Weight>140kg
• Subjects of childbearing potential

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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60 participants in 2 patient groups

Peripheral

Experimental group

Description:

Half the patients will be randomised to the non-interventional part of the trial. In this subgroup of patients will be randomised 1:1 to 5 day course of subcutaneous placebo injections or a 5 day course of G-CSF(Granocyte™) subcutaneous injections

Treatment:

Drug: granulocyte colony stimulating factor (GCSF)

Interventional arm

Experimental group

Description:

In the subgroup of the interventional arm patients will be randomised 1:1 to receive a 5 day course of subcutaneous G-CSF (Granocyte™) injections and bone marrow aspiration at day 5, they will then receive either stem cells or placebo via intracoronary injection

Treatment:

Procedure: bone marrow mononuclear cells