Status:
COMPLETED
Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure
Lead Sponsor:
Celladon Corporation
Conditions:
Heart Failure, Congestive
Dilated Cardiomyopathy
Eligibility:
All Genders
18-75 years
Phase:
PHASE1
PHASE2
Brief Summary
The study is divided into 2 parts. In the first part, the safety of the gene transfer agent MYDICAR® will be evaluated. In the second part, the ability of MYDICAR® to improve heart function will be st...
Detailed Description
The American Heart Association (AHA) 2006 update on heart disease reported that 5 million Americans are believed to have symptomatic heart failure (HF), and 550,000 patients are newly diagnosed each y...
Eligibility Criteria
Inclusion
- Chronic ischemic or non-ischemic cardiomyopathy. Subjects with ischemic cardiomyopathy must have at least one major coronary vessel with Thrombolysis in Myocardial Infarction (TIMI) grade 3 flow.
- Left ventricular ejection fraction (LVEF) ≤35%
- Diagnosis of New York Heart Association (NYHA) Class III/IV heart failure for a minimum of 3 months prior to screening
- Maximal oxygen consumption (VO2 max) ≤20 mL/kg/min within 90 days prior to enrollment
- An implantable cardioverter defibrillator (ICD) implanted a minimum of 30 days prior to enrollment
- Treatment with appropriate heart failure therapy as tolerated
- All women of childbearing potential must have a negative urine pregnancy test prior to administration of investigational product and agree to use adequate contraception. Men capable of fathering a child must agree to use barrier contraception or limit activity to post-menopausal, surgically sterilized, or a contraception-practicing partner, for 3 months after administration of investigational product.
- Ability to sign Informed Consent Form (ICF) and Release of Medical Information Form
Exclusion
- Any intravenous therapy with positive inotropes, vasodilators, or diuretics within 30 days prior to enrollment
- Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
- Cardiac surgery, percutaneous coronary intervention, or valvuloplasty within 30 days prior to enrollment
- Clinically significant myocardial infarction (e.g., ST elevation MI \[STEMI\] or large non-STEMI) within 6 months prior to enrollment
- Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), surgically implanted LVAD or cardiac shunt
- Likely to receive cardiac resynchronization therapy, cardiomyoplasty, LVRS, heart transplant, conventional revascularization procedure, or valvular repair within 6 months following enrollment
- Patients with prior coronary artery bypass graft(s) (CABG) will reviewed on a case-by-case basis
- No evidence of functional or viable myocardium
- Exercise capacity primarily limited by obesity, peripheral vascular disease, intrinsic pulmonary disease or orthopedic problems and not by underlying heart failure
- Known hypersensitivity to octafluoropropane (component of the intravenous echocardiography contrast agent, DEFINITY®) or other contrast dyes used for angiography; history of, or likely need for, high dose steroid pretreatment prior to contrast angiography
- A left ventricle that is difficult to image or high quality echocardiography is not obtainable at screening
- Significant left main or ostial right coronary lumenal stenosis in the opinion of the investigator
- Expected survival \<1 year in the investigator's medical opinion
- Suspected or active viral, bacterial, fungal, or parasitic infection within 48 hours prior to enrollment
- Liver function tests (alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], alkaline phosphatase) \>2x Upper Limit of Normal (ULN) within 30 days prior to enrollment or known intrinsic liver disease (e.g., cirrhosis, chronic hepatitis B or hepatitis C virus infection)
- Current or likely need for hemodialysis within 12 months following enrollment
- Bleeding diathesis or thrombocytopenia defined as platelet count \<50,000 platelets/μL
- Anemia defined as hemoglobin \<10 g/dL
- Known AIDS or HIV-positive status, or a previous diagnosis of immunodeficiency with an absolute neutrophil count \<1000 cells/mm3
- Previous participation in a study of gene transfer
- Presence of neutralizing anti-AAV1 antibodies at titer ≥1:2 within 3 months of screening
- Receiving investigational intervention or participating in another clinical study within 30 days or within 5 half-lives of the investigational drug administration prior to enrollment
- Pregnancy or lactation
- Recent history of psychiatric disease (including drug or alcohol abuse) that is likely to impair subject's ability to comply with protocol-mandated procedures, in the opinion of the investigator
- Other concurrent medical condition(s) that, while not explicitly excluded by the protocol, could jeopardize the safety of the patient or objectives of the study
Key Trial Info
Start Date :
March 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2012
Estimated Enrollment :
51 Patients enrolled
Trial Details
Trial ID
NCT00454818
Start Date
March 1 2007
End Date
August 1 2012
Last Update
August 20 2014
Active Locations (22)
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1
University of California at San Diego Medical Center
San Diego, California, United States, 92103
2
San Diego Cardiac Center
San Diego, California, United States, 92123
3
Shands Hospital at University of Florida
Gainesville, Florida, United States, 32608
4
Northwestern University
Chicago, Illinois, United States, 60611