Clinical trial NCT03572660: Use of Bone Marrow Derived Stem Cell and G-CSF With Circulatory Assistance in the Treatment of DCM
Official title: Phase II Study Assessing the Combined Use of Autologous Bone Marrow Derived Mononuclear Cells and G-csf With Percutaneous Circulatory Assistance in the Treatment of Dilated Cardiomyopathy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Dilated Cardiomyopathy
Keywords: Stem Cell
Circulatory Support
Heart Failure
G-CSF
Lead sponsor: Barts & The London NHS Trust
Sponsor class: OTHER
First posted: 2018-06-28
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single arm intervention study
Primary purpose: TREATMENT

Brief summary: DCM Support is recruiting patients with dilated cardiomyopathy and heart failure symptoms. The goal of this clinical trial is to examine whether treatment with a patient's own stem cells can improve their heart function and alleviate heart failure symptoms.

* Stem cells will be collected from bone marrow in the patient's hip under local anaesthetic.
* The stem cells will be infused into the arteries that supply blood to the heart under local anaesthetic.
* A mini heart pump will be used to take the strain off the heart during the procedure.
* The follow-up involves a phone call at 1 month and clinic visits at 3 and 12 months

Detailed description: DCM SUPPORT is a single centre, single arm clinical trial taking place at St Bartholomew's Hospital in London, UK.

* It is recruiting patients with dilated cardiomyopathy and ongoing heart failure symptoms
* All patients undergo a bone marrow aspiration after 5 days of subcutaneous G-CSF injections
* After cell processing, bone marrow-derived mononuclear cells are infused into the coronary arteries using the stop-flow technique. An intra-procedural Impella CP device is used to support the circulation.
* The primary endpoint is change in left ventricular ejection fraction at 3 months as measured by cardiac CT.

Eligibility criteria: Inclusion Criteria:

* Patients with a confirmed diagnosis of dilated cardiomyopathy under the supervision of a physician or a heart failure nurse specialist.
* NYHA class ≥ 2 symptoms despite having received optimal medical therapy and appropriate device therapy, as per clinical guidelines for an interval of at least 3 months.
* No other treatment options available as part of the current best standard of care.
* LVEF ≤35% on any imaging modality performed as part of the screening phase.

Exclusion Criteria:

* Congenital heart disease.
* Clinically significant valvular heart disease.
* Patients who are not suitable for a Percutaneous Mechanical Support Device (E.g. unsuitable femoral artery anatomy, unable able to lie flat for prolonged time to accommodate the stem cell infusion \& presence of LV thrombus)
* Weight of patient that exceeds the maximum limit of the cardiac catheterisation laboratory table / CT scanner.
* Cardiomyopathy 2o 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 (thrombocytopaenia - platelet count \<80 x 10(9)/L or extensive surgical scarring/anatomical deformity at site of bone marrow puncture).
* Known active infection on admission as defined by a temperature \>37.5°C or on a short course of antibiotics.
* An active infection of hepatitis B, hepatitis C, syphilis or HTLV
* Known HIV infection
* Chronic inflammatory disease requiring on-going medication.
* Concomitant disease with a life expectancy of less than one year
* Follow-up impossible (no fixed abode, etc.)
* Neoplastic disease without documented remission within the past 5 years.
* Patients on renal replacement therapy.
* Subjects of childbearing potential unless βHCG negative and are on adequate contraception during the trial.
* Patients falling into the vulnerable category or lacking capacity
* Patients who are unable to understand or read written English will be excluded from the trial.
* Killip Class III or above

Primary outcomes:
measure: Change in left ventricular ejection fraction
timeFrame: Baseline to 3 months
Secondary outcomes:
measure: Change in left ventricular ejection fraction
timeFrame: Baseline to 12 months
measure: Change in exercise capacity
timeFrame: Baseline to 3 and 12 months
measure: Change in heart failure symptoms
timeFrame: Baseline to 3 and 12 months
measure: Change in quality of life as assessed by Minnesota Living with Heart Failure Questionnaire scores
timeFrame: Baseline to 3 and 12 months
measure: Change in quality of life as measured by EuroQol-5 Dimension 5 Levels questionnaires
timeFrame: Baseline to 3 and 12 months
measure: Procedural safety as assessed by in-hospital procedural related morbidity/mortality
timeFrame: In-hospital procedural time
measure: Change in biochemical markers of heart failure
timeFrame: Baseline to 3 and 12 months
measure: Assessment of rates of MACE (cumulative & individual components)
timeFrame: 3 and 12 months
measure: Assessment of rates of stroke
timeFrame: 3 and 12 months
measure: Assessment of peri-procedural myocardial infarction
timeFrame: Day 0 and Day 6
measure: Change in renal function
timeFrame: Baseline to 3 and 12 months
measure: Change in inflammatory markers
timeFrame: Baseline to 3 and 12 months
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: St Bartholomew's Hospital
Study officials:
affiliation: Queen Mary University of London
name: Anthony Mathur
role: PRINCIPAL_INVESTIGATOR
References: