Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure
Official Title
A Phase 1/2 Trial of Intracoronary Administration of MYDICAR® (AAV1/SERCA2a) in Subjects With Heart Failure in Two Stages (Open-Label, Sequential Dose-Escalation Cohorts and Randomized, Double-Blind, Placebo-Controlled, Parallel Cohorts)
Acronym
CUPID
Organization
Celladon CorporationINDUSTRY
Status Module
Record Verification Date
Aug 2014
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Mar 2007
Primary Completion Date
Aug 2010Actual
Completion Date
Aug 2012Actual
First Submitted Date
Mar 30, 2007
First Submission Date that Met QC Criteria
Mar 30, 2007
First Posted Date
Apr 2, 2007Estimated
Results Waived
Not provided
Results First Submitted Date
Jun 25, 2014
Results First Submitted that Met QC Criteria
Aug 19, 2014
Results First Posted Date
Aug 20, 2014Estimated
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Aug 19, 2014
Last Update Posted Date
Aug 20, 2014Estimated
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Celladon CorporationINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
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 studied.
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 year. The estimated direct and indirect cost of HF in the United States (U.S.) for 2006 will be ~$29.6 billion. Heart failure is a disabling chronic disease and the most frequent discharge diagnosis for hospitalization among older adults. Despite the significant resources expended on the treatment of this disease, outcomes remain poor. The five-year survival for individuals diagnosed with heart failure is less than 50%, and in end-stage heart failure, the one-year survival may be as low as 25% regardless of medical therapy.
Recent studies suggest that the failing heart is not refractory to treatment, as was previously believed. For example, the observation that a small percentage of subjects with left ventricular assist devices (LVADs) can be permanently weaned from their device strongly suggests that damaged hearts are capable of recovering lost function.
Clinical studies of MYDICAR® have not yet been conducted in humans. Celladon Corporation (Celladon) proposes to investigate gene transfer as a method to restore SERCA2a function in heart failure (HF) patients using a recombinant adeno-associated viral vector (AAV), which consists of an AAV serotype 1 capsid and contains the human SERCA2a complementary DNA (cDNA) flanked by Inverted Terminal Repeats (ITR) derived from AAV serotype 2 (AAV1/SERCA2a). MYDICAR® refers to AAV1/SERCA2a drug product intended for administration by percutaneous delivery.
Conditions Module
Conditions
Heart Failure, Congestive
Dilated Cardiomyopathy
Keywords
Not provided
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 1Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
51Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
MYDICAR Very Low Dose
Experimental
Single dose of MYDICAR, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1.4x10e11 DNAase resistant particles administered by antegrade epicardial coronary artery infusion. Used in MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study) only.
Genetic: MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)
MYDICAR Low Dose
Experimental
Single dose of MYDICAR, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6x10e11 DNAase resistant particles administered by antegrade epicardial coronary artery infusion. Used in MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study) and MYDICAR Phase 2 (Placebo-controlled, Randomized Study)
Genetic: MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)
Single dose of MYDICAR, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3x10e12 DNAase resistant particles administered by antegrade epicardial coronary artery infusion. Used in MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study) and MYDICAR Phase 2 (Placebo-controlled, Randomized Study).
Genetic: MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)
MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)
Genetic
MYDICAR administered by antegrade epicardial coronary artery infusion
MYDICAR High Dose
MYDICAR Low Dose
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Phase 2: Incidence of Treatment-emergent Adverse Events (TEAE) at 12 Months
Includes all adverse events that occurred from the time of first infusion of the investigational product or placebo to the 12-month visit. The category of "TEAEs related to the investigational product (IP)" includes TEAEs considered by the investigator to be possibly, probably, or definitely related to the IP.
12 months
Phase 2: Length of Cardiovascular-related Hospitalizations at 6 Months
Mean number of days in the hospital for cardiovascular-related complications. All hospitalizations were evaluated and classified by the blinded Clinical Endpoints Committee.
6 months
Phase 2: Change in Symptomatic Efficacy Domains From Baseline to Month 6: New York Heart Association (NYHA) Class and Minnesota Living With Heart Failure Questionnaire (MLWHFQ) Score
NYHA classification is a symptomatic assessment in which the investigator evaluates subjects on a scale ranging from Class I (subjects with no limitation of activities, no symptoms from ordinary activities) to Class IV (subjects who should be at complete rest, confined to bed or chair; any physical activity brings on discomfort and symptoms occur at rest).
The MLWHFQ is a patient-reported quality of life (QoL) measure in which patients assess the impact of their heart condition on activities in the past month using a Likert scale ranging from 0 (no effect) to 5 (very much effect). Higher scores thus indicate a lower QoL. The maximum (worst) score is 105 and the minimum (best) score is 0.
For both measures, changes from baseline with positive values indicate a worsening in symptoms and changes from baseline with negative values indicate an improvement in symptoms.
Baseline to 6 months
Phase 2: Change in 6-minute Walk Test (6MWT) From Baseline to Month 6
The 6MWT measures the distance walked in meters during a 6-minute test. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
Secondary Outcomes
Not provided
Other Outcomes
Measure
Description
Time Frame
Phase 2: Length of Cardiovascular-related Hospitalizations at 12 Months
Mean number of days in the hospital for cardiovascular-related complications. All hospitalizations were evaluated and classified by the blinded Clinical Endpoints Committee.
12 months
Phase 2: Change in Symptomatic Efficacy Domains From Baseline to Month 12: New York Heart Association (NYHA) Class and Minnesota Living With Heart Failure Questionnaire (MLWHFQ) Score
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
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 Criteria:
Any intravenous therapy with positive inotropes, vasodilators, or diuretics within 30 days prior to enrollment
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
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
18 Years
Maximum Age
75 Years
Standard Ages
AdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Brian Jaski, MD
San Diego Cardiac Center
Principal Investigator
Donna Mancini, MD
Columbia University Hospital
Principal Investigator
Randall Starling, MD
The Cleveland Clinic
Principal Investigator
Mariell Jessup, MD
University of Pennsylvania
Study Chair
Thomas Cappola, MD, ScM
University of Pennsylvania
Principal Investigator
Daniel Pauly, MD
Shands Hospital, University of Florida at Gainesville
Principal Investigator
Barry London, MD
University of Pittsburgh Medical Center
Principal Investigator
Barry Greenberg, MD
University of California at San Diego Medical Center
Principal Investigator
A. G. Kfoury, MD
Intermountain Health Care, Inc.
Principal Investigator
Locations
Facility
Status
City
State
ZIP
Country
Contacts
University of California at San Diego Medical Center
Hajjar RJ, Zsebo K, Deckelbaum L, Thompson C, Rudy J, Yaroshinsky A, Ly H, Kawase Y, Wagner K, Borow K, Jaski B, London B, Greenberg B, Pauly DF, Patten R, Starling R, Mancini D, Jessup M. Design of a phase 1/2 trial of intracoronary administration of AAV1/SERCA2a in patients with heart failure. J Card Fail. 2008 Jun;14(5):355-67. doi: 10.1016/j.cardfail.2008.02.005. Epub 2008 May 27.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
No data available
No data is available for this block.
Results Section
Participant Flow Module
Pre-assignment Details
Not provided
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
MYDICAR® Very Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1.4E11 DNAase resistant particles (DRP) administered by antegrade epicardial coronary artery infusion.
This arm was included only in the Phase I open-label dose-escalation period.
Single dose of MYDICAR, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1x10e13 DNAase resistant particles administered by antegrade epicardial coronary artery infusion. Used in MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study) and MYDICAR Phase 2 (Placebo-controlled, Randomized Study).
Genetic: MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)
MYDICAR administered by antegrade epicardial coronary artery infusion
MYDICAR High Dose
MYDICAR Low Dose
MYDICAR Mid Dose
AAV1/SERCA2a
Baseline to 6 months
Phase 2: Change in Peak Maximum Oxygen Consumption (VO2) From Baseline to Month 6
Peak VO2 is a measure of maximal oxygen consumption during cardiopulmonary exercise testing; this study used the modified Naughton treadmill protocol. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
Baseline to 6 months
Phase 2: Change in Absolute Levels of N-terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) From Baseline to Month 6
NT-proBNP is a biomarker for heart failure. Increased levels of this biomarker are associated with increased mortality and cardiovascular hospitalization in patients with heart failure.
Baseline to 6 months
Phase 2: Change in Percentage of Blood Ejected From the Left Ventricle (LV) (i.e., Left Ventricular Ejection Fraction [LVEF]) From Baseline to Month 6
Contrast echocardiography was used to determine LVEF. Increases in LVEF are associated with reduced mortality. Changes from baseline with positive values indicate an improvement in heart function and changes from baseline with negative values indicate a worsening of heart function.
Baseline to 6 months
Phase 2: Change in Absolute Left Ventricular End Systolic Volume (LVESV) Frm Baseline to Month 6
Contrast echocardiography was used to determine LVESV. Decreases in LVESV are associated with reduced mortality. Changes from baseline with positive values indicate a worsening in heart function and changes from baseline with negative values indicate an improvement in symptoms.
Baseline to 6 months
NYHA classification is a symptomatic assessment in which the investigator evaluates subjects on a scale ranging from Class I (subjects with no limitation of activities, no symptoms from ordinary activities) to Class IV (subjects who should be at complete rest, confined to bed or chair; any physical activity brings on discomfort and symptoms occur at rest).
The MLWHFQ is a patient-reported quality of life (QoL) measure in which patients assess the impact of their heart condition on activities in the past month using a Likert scale ranging from 0 (no effect) to 5 (very much effect). Higher scores thus indicate a lower QoL. The maximum (worst) score is 105 and the minimum (best) score is 0.
For both measures, changes from baseline with positive values indicate a worsening in symptoms and changes from baseline with negative values indicate an improvement in symptoms.
Baseline to 12 months
Phase 2: Change in 6-minute Walk Test (6MWT) From Baseline to Month 12
The 6MWT measures the distance walked in meters during a 6-minute test. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
Baseline to 12 months
Phase 2: Change in Peak Maximum Oxygen Consumption (VO2) From Baseline to Month 12
Peak VO2 is a measure of maximal oxygen consumption during cardiopulmonary exercise testing; this study used the modified Naughton treadmill protocol. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
Baseline to 12 months
Phase 2: Change in Absolute Levels of N-terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) From Baseline to Month 12
NT-proBNP is a biomarker for heart failure. Increased levels of this biomarker are associated with increased mortality and cardiovascular hospitalization in patients with heart failure.
Baseline to 12 months
Phase 2: Change in Percentage of Blood Ejected From the Left Ventricle (LV) (i.e., Left Ventricular Ejection Fraction [LVEF]) From Baseline to Month 12
Contrast echocardiography was used to determine LVEF. Increases in LVEF are associated with reduced mortality. Changes from baseline with positive values indicate an improvement in heart function and changes from baseline with negative values indicate a worsening of heart function.
Baseline to 12 months
Phase 2: Change in Absolute Left Ventricular End Systolic Volume (LVESV) From Baseline to Month 12
Contrast echocardiography was used to determine LVESV. Decreases in LVESV are associated with reduced mortality. Changes from baseline with positive values indicate a worsening in heart function and changes from baseline with negative values indicate an improvement in symptoms.
Baseline to 12 months
Phase 1 and Phase 2: All Subject Deaths Through 36 Months
All subject deaths that occurred during the 12-month study or the 24-month follow-up in subjects enrolled in either the Phase 1 or Phase 2 trial. Events occurring after early termination from the trial are listed as occurring during long-term follow-up, but may have been within 12 months. Specifically, two cardiovascular (CV) deaths in placebo subjects occurred following early study termination, but within 12 months of study initiation. These deaths are therefore included under "Deaths within 12 months" but also listed as "Cardiovascular deaths in long-term follow-up." Accordingly, the number of "Cardiovascular deaths in long-term follow-up" for the placebo group is greater than the number of "Deaths after 12 months," as 2 of the deaths occurred within 12 months but after early termination.
36 months
Stephen Archer, MD
University of Chicago
Principal Investigator
Andrew Kao, MD
Mid America Heart Institute, Saint Luke's Hospital
Principal Investigator
Paul J. Hauptman, MD
St. Louis University Hospital
Principal Investigator
Jill Kalman, MD
Icahn School of Medicine at Mount Sinai
Principal Investigator
Douglas W. Losordo, MD
Northwestern University
Principal Investigator
Eric J. Eichhorn, MD, FACC
Cardiopulmonary Research Science and Technology Institutte, Medical City Dallas Hospital
Principal Investigator
Stephanie H. Dunlap, DO
University of Cincinnati
Principal Investigator
Vinay Thohan, MD
Wake Forest University
Principal Investigator
Maryl R. Johnson, MD
University of Wisconsin, Madison
Principal Investigator
Mark Dunlap, MD
MetroHealth Medical Center
Principal Investigator
Joaquin E. Cigarroa, MD
Oregon Health and Science University
Principal Investigator
Dinesh K. Gupta, MD
Tennessee Center for Clinical Trials, Harton Regional Medical Center
Principal Investigator
Marc Klapholz, MD
University of Medicine and Dentistry of New Jersey
Principal Investigator
Guillermo Torre, MD
The Methodist Hospital Research Institute
Principal Investigator
San Diego
California
92123
United States
Shands Hospital at University of Florida
Gainesville
Florida
32608
United States
Northwestern University
Chicago
Illinois
60611
United States
University of Chicago Medical Center
Chicago
Illinois
60637
United States
Mid America Heart Institute, Saint Luke's Hospital
Kansas City
Missouri
64111
United States
St. Louis University Hospital
St Louis
Missouri
63110
United States
University of Medicine and Dentistry of New Jersey
Newark
New Jersey
07101
United States
Mount Sinai Medical Center
New York
New York
10029
United States
Columbia University Hospital
New York
New York
10032
United States
Wake Forest University
Winston-Salem
North Carolina
27157
United States
University of Cincinnati
Cincinnati
Ohio
45267
United States
MetroHealth Medical Center
Cleveland
Ohio
44109
United States
Cleveland Clinic Foundation
Cleveland
Ohio
44195
United States
Oregon Health and Science University
Portland
Oregon
97239
United States
Hospital of the University of Pennsylvania
Philadelphia
Pennsylvania
19104
United States
University of Pittsburgh Medical Center, Presbyterian-Shadyside Hospital
Pittsburgh
Pennsylvania
15213
United States
Tennessee Center for Clinical Trials & Harton Regional Medical Center
Tullahoma
Tennessee
37388
United States
Cardiopulmonary Research Science and Technology Institute, Medical City Dallas Hospital
Jessup M, Greenberg B, Mancini D, Cappola T, Pauly DF, Jaski B, Yaroshinsky A, Zsebo KM, Dittrich H, Hajjar RJ; Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID) Investigators. Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+-ATPase in patients with advanced heart failure. Circulation. 2011 Jul 19;124(3):304-13. doi: 10.1161/CIRCULATIONAHA.111.022889. Epub 2011 Jun 27.
Zsebo K, Yaroshinsky A, Rudy JJ, Wagner K, Greenberg B, Jessup M, Hajjar RJ. Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality. Circ Res. 2014 Jan 3;114(1):101-8. doi: 10.1161/CIRCRESAHA.113.302421. Epub 2013 Sep 24.
FG001
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DNAase resistant particles (DRP) administered by antegrade epicardial coronary artery infusion.
FG002
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DNAase resistant particles (DRP) administered by antegrade epicardial coronary artery infusion.
FG003
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DNAase resistant particles (DRP) administered by antegrade epicardial coronary artery infusion.
FG004
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
The placebo arm was included only in the Phase 2 randomized double-blind period.
FG0003 subjects
FG0013 subjects
FG0023 subjects
FG0033 subjects
FG0040 subjects
COMPLETED
FG0002 subjects
FG0012 subjects
FG0022 subjects
FG0032 subjects
FG0040 subjects
NOT COMPLETED
FG0001 subjects
FG0011 subjects
FG0021 subjects
FG0031 subjects
FG0040 subjects
Type
Comment
Reasons
Received heart transplant
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
Death
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0030 subjects
FG004
Received left ventricular assist device
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0031 subjects
FG004
Phase 2: Randomized Double-blind
Type
Comment
Milestone Data
STARTED
FG0000 subjects
FG0018 subjects
FG0028 subjects
FG0039 subjects
FG00414 subjects
COMPLETED
FG0000 subjects
FG0016 subjects
FG0025 subjects
FG0038 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0012 subjects
FG0023 subjects
FG0031 subjects
FG004
Type
Comment
Reasons
Received milrinone or dobutamine
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG003
Baseline data are provided for subjects enrolled in the Phase 1 or Phase 2 trials.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Phase 1: MYDICAR® Very Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1.4E11 DRP administered by antegrade epicardial coronary artery infusion.
BG001
Phase 1: MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
BG002
Phase 1: MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
BG003
Phase 1: MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
BG004
Phase 2: MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
BG005
Phase 2: MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
BG006
Phase 2: MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
BG007
Phase 2: Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
BG008
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG0003
BG0013
BG0023
BG0033
BG0048
BG0058
BG0069
BG00714
BG00851
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00053.7± 7.02
BG00155.7± 4.51
BG00248.0± 8.54
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0000
BG0011
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Phase 2: Incidence of Treatment-emergent Adverse Events (TEAE) at 12 Months
Includes all adverse events that occurred from the time of first infusion of the investigational product or placebo to the 12-month visit. The category of "TEAEs related to the investigational product (IP)" includes TEAEs considered by the investigator to be possibly, probably, or definitely related to the IP.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Number
percentage of participants
12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG003
Title
Denominators
Categories
Any TEAE
Title
Measurements
OG000100
OG001100
OG00288.9
OG003
Primary
Phase 2: Length of Cardiovascular-related Hospitalizations at 6 Months
Mean number of days in the hospital for cardiovascular-related complications. All hospitalizations were evaluated and classified by the blinded Clinical Endpoints Committee.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
days
6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
Primary
Phase 2: Change in Symptomatic Efficacy Domains From Baseline to Month 6: New York Heart Association (NYHA) Class and Minnesota Living With Heart Failure Questionnaire (MLWHFQ) Score
NYHA classification is a symptomatic assessment in which the investigator evaluates subjects on a scale ranging from Class I (subjects with no limitation of activities, no symptoms from ordinary activities) to Class IV (subjects who should be at complete rest, confined to bed or chair; any physical activity brings on discomfort and symptoms occur at rest).
The MLWHFQ is a patient-reported quality of life (QoL) measure in which patients assess the impact of their heart condition on activities in the past month using a Likert scale ranging from 0 (no effect) to 5 (very much effect). Higher scores thus indicate a lower QoL. The maximum (worst) score is 105 and the minimum (best) score is 0.
For both measures, changes from baseline with positive values indicate a worsening in symptoms and changes from baseline with negative values indicate an improvement in symptoms.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
units on a scale
Baseline to 6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
Primary
Phase 2: Change in 6-minute Walk Test (6MWT) From Baseline to Month 6
The 6MWT measures the distance walked in meters during a 6-minute test. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
meters
Baseline to 6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
Primary
Phase 2: Change in Peak Maximum Oxygen Consumption (VO2) From Baseline to Month 6
Peak VO2 is a measure of maximal oxygen consumption during cardiopulmonary exercise testing; this study used the modified Naughton treadmill protocol. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
mL/kg per minute
Baseline to 6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Primary
Phase 2: Change in Absolute Levels of N-terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) From Baseline to Month 6
NT-proBNP is a biomarker for heart failure. Increased levels of this biomarker are associated with increased mortality and cardiovascular hospitalization in patients with heart failure.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial. NT-proBNP data were not available for 1 patient in the MYDICAR high dose group.
Posted
Mean
Standard Deviation
pg/mL
Baseline to 6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Other Pre-specified
Phase 2: Length of Cardiovascular-related Hospitalizations at 12 Months
Mean number of days in the hospital for cardiovascular-related complications. All hospitalizations were evaluated and classified by the blinded Clinical Endpoints Committee.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
days
12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
Other Pre-specified
Phase 2: Change in Symptomatic Efficacy Domains From Baseline to Month 12: New York Heart Association (NYHA) Class and Minnesota Living With Heart Failure Questionnaire (MLWHFQ) Score
NYHA classification is a symptomatic assessment in which the investigator evaluates subjects on a scale ranging from Class I (subjects with no limitation of activities, no symptoms from ordinary activities) to Class IV (subjects who should be at complete rest, confined to bed or chair; any physical activity brings on discomfort and symptoms occur at rest).
The MLWHFQ is a patient-reported quality of life (QoL) measure in which patients assess the impact of their heart condition on activities in the past month using a Likert scale ranging from 0 (no effect) to 5 (very much effect). Higher scores thus indicate a lower QoL. The maximum (worst) score is 105 and the minimum (best) score is 0.
For both measures, changes from baseline with positive values indicate a worsening in symptoms and changes from baseline with negative values indicate an improvement in symptoms.
This analysis was performed on the intention to treat population, which included all patients randomized to treatment.
Posted
Mean
Standard Deviation
units on a scale
Baseline to 12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
Other Pre-specified
Phase 2: Change in 6-minute Walk Test (6MWT) From Baseline to Month 12
The 6MWT measures the distance walked in meters during a 6-minute test. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
meters
Baseline to 12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Other Pre-specified
Phase 2: Change in Peak Maximum Oxygen Consumption (VO2) From Baseline to Month 12
Peak VO2 is a measure of maximal oxygen consumption during cardiopulmonary exercise testing; this study used the modified Naughton treadmill protocol. Higher values indicate a better functional status. Changes from baseline with negative values indicate a worsening in function and changes from baseline with positive values indicate an improvement in function.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial. Peak VO2 data were not available for one patient in the placebo group.
Posted
Mean
Standard Deviation
mL/kg per minute
Baseline to 12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
Other Pre-specified
Phase 2: Change in Absolute Levels of N-terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) From Baseline to Month 12
NT-proBNP is a biomarker for heart failure. Increased levels of this biomarker are associated with increased mortality and cardiovascular hospitalization in patients with heart failure.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial. NT-proBNP data were not available for 1 patient in the MYDICAR high dose group.
Posted
Mean
Standard Deviation
pg/mL
Baseline to 12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Post-Hoc
Phase 2: Selected Clinical Outcomes During 12-month Study Period
Incidences of key clinical endpoints as adjudicated by the blinded Clinical Endpoint Committee.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Number
percentage of participants
12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
Other Pre-specified
Phase 2: Change in Percentage of Blood Ejected From the Left Ventricle (LV) (i.e., Left Ventricular Ejection Fraction [LVEF]) From Baseline to Month 12
Contrast echocardiography was used to determine LVEF. Increases in LVEF are associated with reduced mortality. Changes from baseline with positive values indicate an improvement in heart function and changes from baseline with negative values indicate a worsening of heart function.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
Percentage of blood ejected from the LV
Baseline to 12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Primary
Phase 2: Change in Percentage of Blood Ejected From the Left Ventricle (LV) (i.e., Left Ventricular Ejection Fraction [LVEF]) From Baseline to Month 6
Contrast echocardiography was used to determine LVEF. Increases in LVEF are associated with reduced mortality. Changes from baseline with positive values indicate an improvement in heart function and changes from baseline with negative values indicate a worsening of heart function.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
Percentage of blood ejected from the LV
Baseline to 6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Primary
Phase 2: Change in Absolute Left Ventricular End Systolic Volume (LVESV) Frm Baseline to Month 6
Contrast echocardiography was used to determine LVESV. Decreases in LVESV are associated with reduced mortality. Changes from baseline with positive values indicate a worsening in heart function and changes from baseline with negative values indicate an improvement in symptoms.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
mL
Baseline to 6 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Other Pre-specified
Phase 2: Change in Absolute Left Ventricular End Systolic Volume (LVESV) From Baseline to Month 12
Contrast echocardiography was used to determine LVESV. Decreases in LVESV are associated with reduced mortality. Changes from baseline with positive values indicate a worsening in heart function and changes from baseline with negative values indicate an improvement in symptoms.
This analysis was performed on the intention to treat population of the Phase 2 period, which included all patients randomized to treatment in the Phase 2 trial.
Posted
Mean
Standard Deviation
mL
Baseline to 12 months
ID
Title
Description
OG000
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Other Pre-specified
Phase 1 and Phase 2: All Subject Deaths Through 36 Months
All subject deaths that occurred during the 12-month study or the 24-month follow-up in subjects enrolled in either the Phase 1 or Phase 2 trial. Events occurring after early termination from the trial are listed as occurring during long-term follow-up, but may have been within 12 months. Specifically, two cardiovascular (CV) deaths in placebo subjects occurred following early study termination, but within 12 months of study initiation. These deaths are therefore included under "Deaths within 12 months" but also listed as "Cardiovascular deaths in long-term follow-up." Accordingly, the number of "Cardiovascular deaths in long-term follow-up" for the placebo group is greater than the number of "Deaths after 12 months," as 2 of the deaths occurred within 12 months but after early termination.
Includes all participants enrolled in the Phase 1 or Phase 2 trial. Events occurring after early termination are listed under long-term follow-up. The number of "CV deaths in long-term follow-up" for the placebo group is greater than the number of "Deaths after 12 months," as 2 deaths occurred within 12 months but after early termination.
Posted
Number
participants
36 months
ID
Title
Description
OG000
MYDICAR® Very Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1.4E11 DRP administered by antegrade epicardial coronary artery infusion.
OG001
Time Frame
All treatment-emergent adverse events (TEAEs) that occurred from the time of first infusion of the investigational product or placebo through the 12-month visit.
Description
These data include all TEAEs occurring during the Phase 1 or Phase 2 parts of this study.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
MYDICAR® Very Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1.4E11DRP administered by antegrade epicardial coronary artery infusion.
2
3
2
3
EG001
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11DRP administered by antegrade epicardial coronary artery infusion.
6
11
11
11
EG002
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
5
11
11
11
EG003
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
4
12
11
12
EG004
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
9
14
13
14
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Cardiac failure
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0024 affected11 at risk
EG0032 affected12 at risk
EG0043 affected14 at risk
Cardiac failure congestive
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0001 affected3 at risk
EG0012 affected11 at risk
EG0021 affected11 at risk
EG003
Cardiac failure acute
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Acute myocardial infarction
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Arrhythmia
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Cardiorenal syndrome
Cardiac disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Chest pain
General disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Chest discomfort
General disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Pyrexia
General disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Gastric ulcer haemorrhage
Gastrointestinal disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Melaena
Gastrointestinal disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Pancreatitis
Gastrointestinal disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Brain stem stroke
Nervous system disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Syncope
Nervous system disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Transient ischaemic attack
Nervous system disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Pneumonia
Infections and infestations
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Pneumonia staphylococcal
Infections and infestations
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Overdose
Injury, poisoning and procedural complications
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Chronic obstructive pulmonary disease
Respiratory, thoracic and mediastinal disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Respiratory failure
Respiratory, thoracic and mediastinal disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Catheterization cardiac
Surgical and medical procedures
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Heart transplant
Surgical and medical procedures
MedDRA v 11.1
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Vision blurred
Eye disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Colonoscopy
Investigations
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Colon cancer metastatic
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Renal failure acute
Renal and urinary disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Rash maculo-papular
Skin and subcutaneous tissue disorders
MedDRA v 11.1
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Angina unstable
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Cardiogenic shock
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Sudden death
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Diabetes mellitus
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Orthopnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anemia
Blood and lymphatic system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG0030 affected12 at risk
EG0041 affected14 at risk
Angina pectoris
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Arrhythmia
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Cardiac aneurysm
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Cardiac failure
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0022 affected11 at risk
EG003
Cardiac failure congestive
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0023 affected11 at risk
EG003
Cardiogenic shock
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Extrasystoles
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Intracardiac thrombus
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Palpitations
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Supraventricular tachycardia
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Tachycardia
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Ventricular fibrillation
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Ventricular tachycardia
Cardiac disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0022 affected11 at risk
EG003
Hydrocele
Congenital, familial and genetic disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
External ear disorder
Ear and labyrinth disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Hypothyroidism
Endocrine disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Vision blurred
Eye disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Abdominal distension
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Ascites
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Diarrhea
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Dyspepsia
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Epigastric discomfort
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Gastric ulcer
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Catheter site hematoma
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Catheter site hemorrhage
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Chest discomfort
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Chest pain
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Chills
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Fatigue
General disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0013 affected11 at risk
EG0022 affected11 at risk
EG003
Feeling jittery
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Gait disturbance
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Influenza like illness
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Infusion site pain
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Edema
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Edema peripheral
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Puncture site pain
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Pyrexia
General disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Venipuncture site thrombosis
General disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Jaundice
Hepatobiliary disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Bronchitis
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Candidiasis
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Cellulitis
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Central line infection
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Gastric ulcer Helicobacter
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Herpes zoster
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Influenza
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Respiratory tract infection
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Sinusitis
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0013 affected11 at risk
EG0021 affected11 at risk
EG003
Viral infection
Infections and infestations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0022 affected11 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Excoriation
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Eye injury
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0013 affected11 at risk
EG0020 affected11 at risk
EG003
Head injury
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Mouth injury
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Post procedural discharge
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Tendon injury
Injury, poisoning and procedural complications
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Blood creatinine phosphokinase increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Blood creatinine phosphokinase MB increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Blood magnesium decreased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Blood triglycerides increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Blood uric acid increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Carcinoembryonic antigen increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Gallop rhythm present
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Hepatic enzyme increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
International normalized ratio increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Liver function test abnormal
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Mean cell volume increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Muscle enzyme increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Prostatic specific antigen increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Transaminases increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Troponin increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Vitamin D decreased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Weight increased
Investigations
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Anorexia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Fluid overload
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0021 affected11 at risk
EG003
Fluid retention
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Gout
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Hyperkalemia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Hyperlipidemia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Hyperuricemia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Hypervolemia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Hypokalemia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0013 affected11 at risk
EG0022 affected11 at risk
EG003
Hyponatremia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Hypovolemia
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Type 2 diabetes mellitus
Metabolism and nutrition disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Bone disorder
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Bursitis
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Groin pain
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Muscle spasms
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0001 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Muscle tightness
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Muscular weakness
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Musculoskeletal chest pain
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Musculoskeletal discomfort
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Asterixis
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0022 affected11 at risk
EG003
Dizziness postural
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Headache
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Hepatic encephalopathy
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Hypoesthesia
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Neurological symptom
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Nystagmus
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Paresthesia
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Restless leg syndrome
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Somnolence
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Syncope
Nervous system disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Alcoholism
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Anxiety
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Bruxism
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Depression
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Mental status changes
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Somnambulism
Psychiatric disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Azotemia
Renal and urinary disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Hematuria
Renal and urinary disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Renal failure
Renal and urinary disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Renal failure acute
Renal and urinary disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Renal impairment
Renal and urinary disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0022 affected11 at risk
EG003
Breast disorder
Reproductive system and breast disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Gynecomastia
Reproductive system and breast disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Variocele
Reproductive system and breast disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Dyspnea
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Dyspnea exertional
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Hemoptysis
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Nasal congestion
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0022 affected11 at risk
EG003
Oropharyngeal discomfort
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Orthopnea
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Productive cough
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Pulmonary hypertension
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Rales
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Sleep apnea syndrome
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Wheezing
Respiratory, thoracic and mediastinal disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Cold sweat
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Ecchymosis
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0012 affected11 at risk
EG0020 affected11 at risk
EG003
Hemorrhage subcutaneous
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Rash generalized
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Skin ulcer
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Stevens-Johnson syndrome
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Cataract operation
Surgical and medical procedures
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0020 affected11 at risk
EG003
Deep vein thrombosis
Vascular disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0020 affected11 at risk
EG003
Hematoma
Vascular disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Hypertension
Vascular disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected11 at risk
EG0021 affected11 at risk
EG003
Hypotension
Vascular disorders
MedDRA (11.1)
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected11 at risk
EG0021 affected11 at risk
EG003
Limitations of this study include its small sample size and the inability to conclusively prove that the delivered gene was responsible for the observed clinical effects. Larger confirmatory trials are needed.
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
GT60
Results Disclosure Restriction on PI(s)?
Yes
Other Details
Not provided
Point of Contact
Title
Organization
Phone
Extension
Email
Jeffrey J. Rudy, Vice President
Celladon Corporation
1 858-366-4288
jrudy@celladon.net
ID
Term
D006333
Heart Failure
D002311
Cardiomyopathy, Dilated
Ancestor Terms
ID
Term
D006331
Heart Diseases
D002318
Cardiovascular Diseases
D006332
Cardiomegaly
D009202
Cardiomyopathies
D000083083
Laminopathies
D030342
Genetic Diseases, Inborn
D009358
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Browse Leaves
Not provided
Browse Branches
Not provided
0 subjects
0 subjects
10 subjects
4 subjects
1 subjects
FG0040 subjects
Death
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0030 subjects
FG0041 subjects
Received a heart transplant
FG0000 subjects
FG0010 subjects
FG0022 subjects
FG0030 subjects
FG0041 subjects
Received left ventricular assist device
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG0042 subjects
58.77
± 19.01
BG00460.3± 10.27
BG00563.9± 8.85
BG00656.6± 13.96
BG00761.0± 11.94
BG00860.5± 11.39
1
BG0031
BG0041
BG0050
BG0063
BG0071
BG0088
Male
BG0003
BG0012
BG0022
BG0032
BG0047
BG0058
BG0066
BG00713
BG00843
0
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Asian
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Native Hawaiian or Other Pacific Islander
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Black or African American
BG0000
BG0010
BG0022
BG0030
BG0040
BG0050
BG0063
BG0071
BG0086
White
BG0003
BG0013
BG0021
BG0033
BG0048
BG0058
BG0066
BG00713
BG00845
More than one race
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Unknown or Not Reported
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
14
92.9
TEAEs related to the investigational product (IP)
Title
Measurements
OG00050
OG00112.5
OG00211.1
OG00357.1
TEAEs related to administration of the IP
Title
Measurements
OG00012.5
OG00125
OG00211.1
OG00357.1
Serious TEAEs
Title
Measurements
OG00062.5
OG00150
OG00233.3
OG00364.3
Serious TEAEs related to the IP
Title
Measurements
OG0000
OG0010
OG0020
OG00321.4
Serious TEAEs related to IP administration
Title
Measurements
OG0000
OG0010
OG0020
OG00328.6
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG0000.3± 0.46
OG0011.3± 2.55
OG0020.2± 0.67
OG0032.1± 2.92
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG002
OG003
t-test, 2 sided
0.078
The a priori threshold for statistical significance was P < 0.2. There were no adjustments for multiple comparisons.
No
Superiority or Other
OG001
OG003
t-test, 2 sided
0.515
The a priori threshold for statistical significance was P < 0.2. There were no adjustments for multiple comparisons.
No
Superiority or Other
OG000
OG003
t-test, 2 sided
0.098
The a priori threshold for statistical significance was P < 0.2. There were no adjustments for multiple comparisons.
No
Superiority or Other
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Change in NHYA class
Title
Measurements
OG000-0.8± 0.71
OG001-0.8± 0.89
OG002-0.6± 0.73
OG003-0.2± 0.70
Change in MLWHFQ score
Title
Measurements
OG000-7.6± 20.99
OG0017.9± 27.28
OG002-10.3± 12.21
OG003
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG00013.0± 61.40
OG001-59.5± 213.64
OG0021.0± 99.69
OG003-86.6± 164.30
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG000-0.73± 4.88
OG001-1.07± 5.076
OG002-0.43± 0,802
OG003-2.10± 4.462
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0028
OG00314
Title
Denominators
Categories
Title
Measurements
OG000694.1± 1444.94
OG0012073.1± 4224.22
OG002-13.5± 928.48
OG0035540.0± 11,873.46
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG00010.1± 12.71
OG0017.4± 11.80
OG0020.4± 1.33
OG0034.5± 5.80
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Change in NHYA class
Title
Measurements
OG000-0.1± 0.99
OG0010.1± 0.83
OG002-0.3± 0.71
OG0030.1± 0.73
Change in MLWHFQ score
Title
Measurements
OG00024.4± 37.3
OG00122.5± 32.56
OG0020.1± 23.80
OG003
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG000-167.9± 191.02
OG001-115.9± 226.56
OG002-23.7± 151.08
OG003-120.4± 181.41
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00313
Title
Denominators
Categories
Title
Measurements
OG000-5.00± 4.733
OG001-3.31± 5.473
OG002-1.57± 3.677
OG003-2.75± 5.084
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0028
OG00314
Title
Denominators
Categories
Title
Measurements
OG0003689.1± 5109.27
OG0018440.4± 11270.44
OG0021756.3± 4331.00
OG00311464.3± 16866.55
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Fatal cardiovascular event
Title
Measurements
OG00012.5
OG0010
OG0020
OG0037.1
Worsening heart failure
Title
Measurements
OG00050.0
OG00137.5
OG00222.2
OG003
Myocardial infarction
Title
Measurements
OG0000
OG0010
OG0020
OG003
Heart failure-related hospitalization
Title
Measurements
OG00050.0
OG00137.5
OG00222.2
OG003
Silent myocardial infarction
Title
Measurements
OG0000
OG0010
OG0020
OG003
Receipt of left ventricular assist device
Title
Measurements
OG0000
OG00112.5
OG0020
OG003
Heart transplant
Title
Measurements
OG0000
OG00125.0
OG00211.1
OG003
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG000-6.5± 7.26
OG001-5.6± 10.52
OG0020.3± 8.87
OG003-2.5± 9.96
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG0000.0± 1.87
OG001-1.5± 6.35
OG002-0.7± 3.76
OG003-2.1± 6.90
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG0000.4± 26.16
OG00110.5± 45.91
OG002-9.6± 27.55
OG00318.2± 39.45
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG003
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Units
Counts
Participants
OG0008
OG0018
OG0029
OG00314
Title
Denominators
Categories
Title
Measurements
OG00040.7± 59.79
OG00166.8± 103.12
OG0029.9± 49.27
OG00337.7± 69.09
MYDICAR® Low Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 6E11 DRP administered by antegrade epicardial coronary artery infusion.
OG002
MYDICAR® Mid Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 3E12 DRP administered by antegrade epicardial coronary artery infusion.
OG003
MYDICAR® High Dose
Single dose of MYDICAR®, a viral vector (adeno-associated virus serotype 1 [AAV1]) carrying the gene for sarcoplasmic reticulum Ca++-adenosine triphosphatase (SERCA2a), at a dose of 1E13 DRP administered by antegrade epicardial coronary artery infusion.
OG004
All MYDICAR®
All participants who received a single infusion of MYDICAR® at any dose during the Phase 1 or Phase 2 studies.
OG005
Placebo
Single dose of placebo administered by antegrade epicardial coronary artery infusion.