Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy

Dilated Cardiomyopathy Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01302171
• Other study ID #: 2009-013112-12
• Status: Completed
• Phase: Phase 2
• First received: February 21, 2011
• Last updated: November 14, 2013
• Start date: August 2010
• Est. completion date: July 2013

Study information

• Verified date: November 2013
• Source: Barts & The London NHS Trust
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Research Ethics Committee
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: July 2013
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 80 Years

Eligibility

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 Design

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

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Dilated
• Dilated Cardiomyopathy

Intervention

Drug:
• granulocyte colony stimulating factor (GCSF)
10mcg/kg per day 5 days

Procedure:
• bone marrow mononuclear cells
intra coronary injection of stem cells or placebo

Locations

Country	Name	City	State
United Kingdom	London Chest Hospital	London

Sponsors (3)

Lead Sponsor	Collaborator
Barts & The London NHS Trust	Royal Brompton & Harefield NHS Foundation Trust, University College London Hospitals

Country where clinical trial is conducted

United Kingdom, 

References & 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography		3 months	No
Secondary	Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)		3 months and 12 months	No
Secondary	Changes in V02 max (exercise capacity)		3 months and 12 months	No
Secondary	Changes in left ventricular ejection fraction, ventricular dimensions as measured by cardiac magnetic resonance imaging or computerised tomography		3 months and 12 months	No
Secondary	Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires		3 months and 12 months	No
Secondary	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	No
Secondary	Hospitalization for Heart failure & the occurrence of major arrhythmias defined as symptomatic ventricular tachycardia or survived sudden death		3 months and 12 months	No
Secondary	The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death		3 months and 12 months	No