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
  Purpose

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


Condition Intervention Phase
Dilated Cardiomyopathy
Drug: granulocyte colony stimulating factor (GCSF)
Procedure: bone marrow mononuclear cells
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Barts & The London NHS Trust:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 60
Study Start Date: August 2010
Study Completion Date: July 2013
Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
Drug: granulocyte colony stimulating factor (GCSF)
10mcg/kg per day 5 days
Other Name: Lenograstim, Granocyte™, Chugai Pharma UK, Limited
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
Procedure: bone marrow mononuclear cells
intra coronary injection of stem cells or placebo

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01302171

Locations
United Kingdom
London Chest Hospital
London, United Kingdom, E2 9JX
Sponsors and Collaborators
Barts & The London NHS Trust
Royal Brompton & Harefield NHS Foundation Trust
University College London Hospitals
Investigators
Principal Investigator: Anthony Mathur, MD FRCP FESC Barts & The London NHS Trust
  More Information

Publications:
Responsible Party: Anthony Mathur, Clinical and Research Lead (Cardiology), Barts & The London NHS Trust
ClinicalTrials.gov Identifier: NCT01302171     History of Changes
Other Study ID Numbers: 2009-013112-12
Study First Received: February 21, 2011
Last Updated: November 14, 2013
Health Authority: United Kingdom: Research Ethics Committee

Keywords provided by Barts & The London NHS Trust:
dilated cardiomyopathy
adult stem cells
bone marrow progenitor cells
bone marrow stem cells
autologous
granulocyte-colony stimulating factor
intracoronary injection
left ventricular function
To determine if patient's own bone marrow derived stem cells can be used to improve cardiac function

Additional relevant MeSH terms:
Cardiomyopathies
Cardiomyopathy, Dilated
Cardiomegaly
Cardiovascular Diseases
Heart Diseases
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on October 21, 2014