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

This study has been completed.
Sponsor:
Collaborators:
Royal Brompton & Harefield NHS Foundation Trust
University College London Hospitals
Information provided by (Responsible Party):
Anthony Mathur, Barts & The London NHS Trust
ClinicalTrials.gov Identifier:
NCT01302171
First received: February 21, 2011
Last updated: November 14, 2013
Last verified: November 2013

February 21, 2011
November 14, 2013
August 2010
December 2012   (final data collection date for primary outcome measure)
Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography [ Time Frame: 3 months ] [ Designated as safety issue: No ]
Change in left ventricular ejection fraction as measured by cardiac MRI at 3 months [ Time Frame: 3 months ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01302171 on ClinicalTrials.gov Archive Site
  • Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme) [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • Changes in V02 max (exercise capacity) [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • Changes in left ventricular ejection fraction, ventricular dimensions as measured by cardiac magnetic resonance imaging or computerised tomography [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • Occurrence of a Major Adverse Cardiac Event (MACE) defined as cardiac death, myocardial infarction (CK / CK-MB over 2 times the upper limit of normal) [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • Hospitalization for Heart failure & the occurrence of major arrhythmias defined as symptomatic ventricular tachycardia or survived sudden death [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death [ Time Frame: 3 months and 12 months ] [ Designated as safety issue: No ]
  • Change in: Concentrations of NT-proBNP (cardiac enzyme) [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Changes in V02 max (exercise capacity) [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Changes in ejection fraction, ventricular dimensions as measured by cardiac MRI/ CT [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Occurrence of a Major Adverse Cardiac Event (MACE) defined as cardiac death, myocardial infarction (CK / CK-MB over 2 times the upper limit of normal) [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Hospitalization for Heart failure & the occurrence of major arrhythmias defined as symptomatic ventricular tachycardia or survived sudden death [ Time Frame: 3 months ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy
Randomised Controlled Trial to Compare the Effects of G-CSF (Granocyte™) and Autologous Bone Marrow Progenitor Cells on Quality of Life and Left Ventricular Function in Patients With Idiopathic Dilated Cardiomyopathy

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

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Dilated Cardiomyopathy
  • Drug: granulocyte colony stimulating factor (GCSF)
    10mcg/kg per day 5 days
    Other Name: Lenograstim, Granocyte™, Chugai Pharma UK, Limited
  • Procedure: bone marrow mononuclear cells
    intra coronary injection of stem cells or placebo
  • Experimental: Peripheral
    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
    Intervention: Drug: granulocyte colony stimulating factor (GCSF)
  • Experimental: Interventional arm
    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
    Intervention: Procedure: bone marrow mononuclear cells
Arnous S, Mozid A, Mathur A. The Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy (REGENERATE-DCM) trial: study design. Regen Med. 2011 Jul;6(4):525-33. doi: 10.2217/rme.11.29.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
60
July 2013
December 2012   (final data collection date for primary outcome measure)

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
Both
18 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
United Kingdom
 
NCT01302171
2009-013112-12
Yes
Anthony Mathur, Barts & The London NHS Trust
Barts & The London NHS Trust
  • Royal Brompton & Harefield NHS Foundation Trust
  • University College London Hospitals
Principal Investigator: Anthony Mathur, MD FRCP FESC Barts & The London NHS Trust
Barts & The London NHS Trust
November 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP