Ischemic Heart Failure
Conditions
Interventions
Product Name: Cultured Autologous Skeletal Myoblast Cells
Product Code: N/A
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: N/A
CAS Number: N/A
Current Sponsor code: N/A
Other descr
Sponsors
Genzyme Europe B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients must meet the following criteria to be enrolled in this study: 1. Patient must be > = 18 and = 15% (determined by echocardiography and confirmed by the core laboratory). 4.Patient must have significant myocardial dysfunction from a previous myocardial infarction demonstrated by the existence of akinesia affecting more than 2 accessible, contiguous left ventricular segments (out of 16) on a basal state echocardiogram, with no viability after stimulation with low dose dobutamine. 5.The left ventricular myocardial infarction (Q wave or positive enzyme) targeted for study injections must have occurred > = 4 weeks prior to screening. 6.Patient must qualify for insertion of an ICD device. Patient must have an ICD upon enrollment, receive an ICD before CABG surgery and study treatment or receive an ICD prior to discharge from the hospital following CABG surgery. 7.Patient should be a NYHA functional class I-III, who is receiving optimal contemporary medical management (e.g., ACE inhibitors, beta-blockers, diuretics, etc.). In Canada, only patients classified as NYHA Class II or III will be eligible for enrollment. 8.Fertile female patients with negative urine pregnancy test at screening and on the day of treatment prior to autologous skeletal myoblast administration. 9.Fertile female patients who have agreed to follow an approved method of contraception to avoid pregnancy for 60 days after receiving autologous skeletal myoblast administration (or longer if required by concomitant medication (e.g., amiodarone). 10.Patient must be committed to following the protocol requirements for 2 years as evidenced by written informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Need for a rapid surgical coronary revascularization (< = 21 days) 2.Need for any other related cardio-surgical measure during coronary surgery (e.g., mitral valve repair or valve replacement) 3.Patient with a left ventricular aneurysm who is a candidate for left ventricular aneurysmectomy or left ventricular reduction surgery 4.Patients receiving left or biventricular (BiV) pacing therapy for heart failure (unless the patient has stabilized after 6 or more months of this therapy) 5.Patient with cardiomyopathy presumed to be of non-ischemic origin (e.g., hypertrophic cardiomyopathy) 6.Patient for whom adequate echocardiography, or other required study procedures, cannot be performed for technical reasons 7.Patient for whom low dose of dobutamine, required for the identification of non-viable, scarred myocardium, is contra-indicated (e.g., recent sustained ventricular tachycardia and ventricular fibrillation) 8.Patient with advanced heart failure (e.g., NYHA IV heart failure symptoms, or in need of a heart transplant) who does not respond to optimal medical therapy and does not improve to at least Class III symptoms 30 days prior to skeletal muscle biopsy 9.Patient infected with the hepatitis C virus; the hepatitis B virus (HBs + antigen); patient who is HIV-1 or HIV-2 positive; HTLV-1 positive; Ag P24 positive (this exclusion criteria refers only to regions where this testing is required (e.g. France) 10.Patient with hemophilia; long-term immunosuppressive treatment, including with corticosteroids; stage III-IV arteriopathy of the lower limbs; significant muscular amyotrophia; peripheral muscular illness; serious intellectual deterioration or neuro-psychiatric disorders that would make follow-up difficult 11.Patient with allergies to gentamicin or other aminoglycosides, allergies to study required medications or imaging agents 12.Female patient who is pregnant, nursing, or fertile and using either no or an inadequate form of contraception 13.Illness other than ischemic heart failure that makes the short-range prognosis for survival questionable 14.Simultaneous participation in another study with an investigational study agent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The coprimary objectives of this study are to assess the recovery of contractility in akinetic myocardial segments that have received transplanted skeletal myoblasts as measured by echocardiogram, and to evaluate the change in left ventricular ejection fraction (LVEF).;Secondary Objective: •Assess freedom from Major Adverse Cardiac Events (MACE). •Monitor the evolution of overall left ventricular function as measured by echocardiogram and assessed by ejection fraction and diastolic and valvular function. •Monitor recovery of metabolic viability and blood flow in the transplant area as evaluated by positron emission tomography (PET) in a subset of patients and at selected sites. •Assess change in functional state, assessed using the New York Heart Association (NYHA) classification. •Evaluate change in quality of life, assessed using the SF-36® Health Survey. •Evaluate the safety and efficacy of 2 different doses of skeletal myoblasts for further testing in a confirmatory Phase 3 trial. ;Primary end point(s): The coprimary efficacy endpoints are: •the recovery of contractility in within previously akinetic myocardial segments at Month 6 •the absolute change from baseline to Month 6 in LVEF. | — |
Countries
Denmark
Outcome results
None listed