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

November 14, 2013 updated by: Anthony Mathur, Barts & The London NHS Trust

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

60

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography
Time Frame: 3 months
3 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)
Time Frame: 3 months and 12 months
3 months and 12 months
Changes in V02 max (exercise capacity)
Time Frame: 3 months and 12 months
3 months and 12 months
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
3 months and 12 months
Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires
Time Frame: 3 months and 12 months
3 months and 12 months
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
3 months and 12 months
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
3 months and 12 months
The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death
Time Frame: 3 months and 12 months
3 months and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Barts & The London NHS Trust

Collaborators

University College London Hospitals
Royal Brompton & Harefield NHS Foundation Trust

Investigators

  • Principal Investigator: Anthony Mathur, MD FRCP FESC, Barts & The London NHS Trust

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2010

Primary Completion (Actual)

December 1, 2012

Study Completion (Actual)

July 1, 2013

Study Registration Dates

First Submitted

February 21, 2011

First Submitted That Met QC Criteria

February 22, 2011

First Posted (Estimate)

February 24, 2011

Study Record Updates

Last Update Posted (Estimate)

November 15, 2013

Last Update Submitted That Met QC Criteria

November 14, 2013

Last Verified

November 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2009-013112-12

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Dilated Cardiomyopathy

Clinical Trials on granulocyte colony stimulating factor (GCSF)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Brazil

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe