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

Primary Purpose

Dilated Cardiomyopathy

Status
Completed
Phase
Phase 2
Locations
United Kingdom
Study Type
Interventional
Intervention
granulocyte colony stimulating factor (GCSF)
bone marrow mononuclear cells
Sponsored by
Barts & The London NHS Trust
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dilated Cardiomyopathy focused on measuring 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

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • London Chest Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Peripheral

Interventional arm

Arm 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

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

Outcomes

Primary Outcome Measures

Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography

Secondary Outcome Measures

Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)
Changes in V02 max (exercise capacity)
Changes in left ventricular ejection fraction, ventricular dimensions as measured by cardiac magnetic resonance imaging or computerised tomography
Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires
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)
Hospitalization for Heart failure & the occurrence of major arrhythmias defined as symptomatic ventricular tachycardia or survived sudden death
The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death

Full Information

First Posted
February 21, 2011
Last Updated
November 14, 2013
Sponsor
Barts & The London NHS Trust
Collaborators
Royal Brompton & Harefield NHS Foundation Trust, University College London Hospitals
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1. Study Identification

Unique Protocol Identification Number
NCT01302171
Brief Title
Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy
Acronym
REGEN-DCM
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
Study Type
Interventional

2. Study Status

Record Verification Date
November 2013
Overall Recruitment Status
Completed
Study Start Date
August 2010 (undefined)
Primary Completion Date
December 2012 (Actual)
Study Completion Date
July 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Barts & The London NHS Trust
Collaborators
Royal Brompton & Harefield NHS Foundation Trust, University College London Hospitals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dilated Cardiomyopathy
Keywords
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
60 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Peripheral
Arm Type
Experimental
Arm 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
Arm Title
Interventional arm
Arm Type
Experimental
Arm 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
Intervention Type
Drug
Intervention Name(s)
granulocyte colony stimulating factor (GCSF)
Other Intervention Name(s)
Lenograstim, Granocyte™, Chugai Pharma UK, Limited
Intervention Description
10mcg/kg per day 5 days
Intervention Type
Procedure
Intervention Name(s)
bone marrow mononuclear cells
Intervention Description
intra coronary injection of stem cells or placebo
Primary Outcome Measure Information:
Title
Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)
Time Frame
3 months and 12 months
Title
Changes in V02 max (exercise capacity)
Time Frame
3 months and 12 months
Title
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
Title
Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires
Time Frame
3 months and 12 months
Title
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
Title
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
Title
The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death
Time Frame
3 months and 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anthony Mathur, MD FRCP FESC
Organizational Affiliation
Barts & The London NHS Trust
Official's Role
Principal Investigator
Facility Information:
Facility Name
London Chest Hospital
City
London
ZIP/Postal Code
E2 9JX
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
21749209
Citation
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.
Results Reference
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Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy

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