Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy

NCT01302171 | Completed Phase 2 DMC

• 1 other identifier: 2009-013112-12
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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

Conditions

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

Outcome Measures

Primary Outcomes (1)

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

  3 months

Secondary Outcomes (7)

• Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)

  3 months and 12 months

• Changes in V02 max (exercise capacity)

  3 months and 12 months

• Changes in left ventricular ejection fraction, ventricular dimensions as measured by cardiac magnetic resonance imaging or computerised tomography

  3 months and 12 months

• Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires

  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)

  3 months and 12 months

Study Arms (2)

Peripheral

EXPERIMENTAL

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)

Interventional arm

EXPERIMENTAL

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

Interventions

granulocyte colony stimulating factor (GCSF) DRUG

10mcg/kg per day 5 days

Also known as: Lenograstim, Granocyte™, Chugai Pharma UK, Limited

Peripheral

bone marrow mononuclear cells PROCEDURE

intra coronary injection of stem cells or placebo

Interventional arm

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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)

Sponsors & Collaborators

• Barts & The London NHS Trust: lead
• Royal Brompton & Harefield NHS Foundation Trust: collaborator
• University College London Hospitals: collaborator

Study Sites (1)

London Chest Hospital

London, E2 9JX, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 21749209 BACKGROUND

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Interventions

Granulocyte Colony-Stimulating Factor; Lenograstim

Condition Hierarchy (Ancestors)

Cardiomegaly; Heart Diseases; Cardiovascular Diseases; Cardiomyopathies; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Anthony Mathur, MD FRCP FESC

  Barts & The London NHS Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical and Research Lead (Cardiology)

Study Record Dates

• First Submitted: February 21, 2011
• First Posted: February 24, 2011
• Study Start: August 1, 2010
• Primary Completion: December 1, 2012
• Study Completion: July 1, 2013
• Last Updated: November 15, 2013

Record last verified: 2013-11

Locations

GB (1)