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Trial record 95 of 157 for:    Idiopathic Dilated Cardiomyopathy

Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (CUPID)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00454818
Recruitment Status : Completed
First Posted : April 2, 2007
Results First Posted : August 20, 2014
Last Update Posted : August 20, 2014
Sponsor:
Information provided by (Responsible Party):
Celladon Corporation

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Heart Failure, Congestive
Dilated Cardiomyopathy
Interventions Genetic: MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)
Procedure: Placebo Infusion
Genetic: MYDICAR Phase 2 (Placebo-controlled, Randomized Study)
Enrollment 51
Recruitment Details  
Pre-assignment Details  
Arm/Group Title MYDICAR® Very Low Dose MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description

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 6E11 DNAase resistant particles (DRP) administered by antegrade epicardial coronary artery infusion. 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. 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.

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.

Period Title: Phase I: Open-label Dose-escalation
Started 3 3 3 3 0
Completed 2 2 2 2 0
Not Completed 1 1 1 1 0
Reason Not Completed
Received heart transplant             1             0             0             0             0
Death             0             1             0             0             0
Received left ventricular assist device             0             0             1             1             0
Period Title: Phase 2: Randomized Double-blind
Started 0 8 8 9 14
Completed 0 6 5 8 10
Not Completed 0 2 3 1 4
Reason Not Completed
Received milrinone or dobutamine             0             1             0             1             0
Death             0             1             0             0             1
Received a heart transplant             0             0             2             0             1
Received left ventricular assist device             0             0             1             0             2
Arm/Group Title Phase 1: MYDICAR® Very Low Dose Phase 1: MYDICAR® Low Dose Phase 1: MYDICAR® Mid Dose Phase 1: MYDICAR® High Dose Phase 2: MYDICAR® Low Dose Phase 2: MYDICAR® Mid Dose Phase 2: MYDICAR® High Dose Phase 2: Placebo Total
Hide Arm/Group Description 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. 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. 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. 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. 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. 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. 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. Single dose of placebo administered by antegrade epicardial coronary artery infusion. Total of all reporting groups
Overall Number of Baseline Participants 3 3 3 3 8 8 9 14 51
Hide Baseline Analysis Population Description
Baseline data are provided for subjects enrolled in the Phase 1 or Phase 2 trials.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 3 participants 3 participants 8 participants 8 participants 9 participants 14 participants 51 participants
53.7  (7.02) 55.7  (4.51) 48.0  (8.54) 58.77  (19.01) 60.3  (10.27) 63.9  (8.85) 56.6  (13.96) 61.0  (11.94) 60.5  (11.39)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 3 participants 8 participants 8 participants 9 participants 14 participants 51 participants
Female
0
   0.0%
1
  33.3%
1
  33.3%
1
  33.3%
1
  12.5%
0
   0.0%
3
  33.3%
1
   7.1%
8
  15.7%
Male
3
 100.0%
2
  66.7%
2
  66.7%
2
  66.7%
7
  87.5%
8
 100.0%
6
  66.7%
13
  92.9%
43
  84.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 3 participants 8 participants 8 participants 9 participants 14 participants 51 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
2
  66.7%
0
   0.0%
0
   0.0%
0
   0.0%
3
  33.3%
1
   7.1%
6
  11.8%
White
3
 100.0%
3
 100.0%
1
  33.3%
3
 100.0%
8
 100.0%
8
 100.0%
6
  66.7%
13
  92.9%
45
  88.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Phase 2: Incidence of Treatment-emergent Adverse Events (TEAE) at 12 Months
Hide Description 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.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Measure Type: Number
Unit of Measure: percentage of participants
Any TEAE 100 100 88.9 92.9
TEAEs related to the investigational product (IP) 50 12.5 11.1 57.1
TEAEs related to administration of the IP 12.5 25 11.1 57.1
Serious TEAEs 62.5 50 33.3 64.3
Serious TEAEs related to the IP 0 0 0 21.4
Serious TEAEs related to IP administration 0 0 0 28.6
2.Primary Outcome
Title Phase 2: Length of Cardiovascular-related Hospitalizations at 6 Months
Hide Description Mean number of days in the hospital for cardiovascular-related complications. All hospitalizations were evaluated and classified by the blinded Clinical Endpoints Committee.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: days
0.3  (0.46) 1.3  (2.55) 0.2  (0.67) 2.1  (2.92)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MYDICAR® High Dose, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.078
Comments The a priori threshold for statistical significance was P < 0.2. There were no adjustments for multiple comparisons.
Method t-test, 2 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MYDICAR® Mid Dose, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.515
Comments The a priori threshold for statistical significance was P < 0.2. There were no adjustments for multiple comparisons.
Method t-test, 2 sided
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection MYDICAR® Low Dose, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.098
Comments The a priori threshold for statistical significance was P < 0.2. There were no adjustments for multiple comparisons.
Method t-test, 2 sided
Comments [Not Specified]
3.Primary Outcome
Title 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
Hide Description

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.

Time Frame Baseline to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: units on a scale
Change in NHYA class -0.8  (0.71) -0.8  (0.89) -0.6  (0.73) -0.2  (0.70)
Change in MLWHFQ score -7.6  (20.99) 7.9  (27.28) -10.3  (12.21) 3.4  (36.00)
4.Primary Outcome
Title Phase 2: Change in 6-minute Walk Test (6MWT) From Baseline to Month 6
Hide Description 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.
Time Frame Baseline to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: meters
13.0  (61.40) -59.5  (213.64) 1.0  (99.69) -86.6  (164.30)
5.Primary Outcome
Title Phase 2: Change in Peak Maximum Oxygen Consumption (VO2) From Baseline to Month 6
Hide Description 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.
Time Frame Baseline to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: mL/kg per minute
-0.73  (4.88) -1.07  (5.076) -0.43  (0,802) -2.10  (4.462)
6.Primary Outcome
Title Phase 2: Change in Absolute Levels of N-terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) From Baseline to Month 6
Hide Description 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.
Time Frame Baseline to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 8 14
Mean (Standard Deviation)
Unit of Measure: pg/mL
694.1  (1444.94) 2073.1  (4224.22) -13.5  (928.48) 5540.0  (11,873.46)
7.Primary Outcome
Title 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
Hide Description 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.
Time Frame Baseline to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: Percentage of blood ejected from the LV
0.0  (1.87) -1.5  (6.35) -0.7  (3.76) -2.1  (6.90)
8.Primary Outcome
Title Phase 2: Change in Absolute Left Ventricular End Systolic Volume (LVESV) Frm Baseline to Month 6
Hide Description 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.
Time Frame Baseline to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: mL
0.4  (26.16) 10.5  (45.91) -9.6  (27.55) 18.2  (39.45)
9.Other Pre-specified Outcome
Title Phase 2: Length of Cardiovascular-related Hospitalizations at 12 Months
Hide Description Mean number of days in the hospital for cardiovascular-related complications. All hospitalizations were evaluated and classified by the blinded Clinical Endpoints Committee.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: days
10.1  (12.71) 7.4  (11.80) 0.4  (1.33) 4.5  (5.80)
10.Other Pre-specified Outcome
Title 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
Hide Description

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.

Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
This analysis was performed on the intention to treat population, which included all patients randomized to treatment.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: units on a scale
Change in NHYA class -0.1  (0.99) 0.1  (0.83) -0.3  (0.71) 0.1  (0.73)
Change in MLWHFQ score 24.4  (37.3) 22.5  (32.56) 0.1  (23.80) 14.7  (34.89)
11.Other Pre-specified Outcome
Title Phase 2: Change in 6-minute Walk Test (6MWT) From Baseline to Month 12
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: meters
-167.9  (191.02) -115.9  (226.56) -23.7  (151.08) -120.4  (181.41)
12.Other Pre-specified Outcome
Title Phase 2: Change in Peak Maximum Oxygen Consumption (VO2) From Baseline to Month 12
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 13
Mean (Standard Deviation)
Unit of Measure: mL/kg per minute
-5.00  (4.733) -3.31  (5.473) -1.57  (3.677) -2.75  (5.084)
13.Other Pre-specified Outcome
Title Phase 2: Change in Absolute Levels of N-terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) From Baseline to Month 12
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 8 14
Mean (Standard Deviation)
Unit of Measure: pg/mL
3689.1  (5109.27) 8440.4  (11270.44) 1756.3  (4331.00) 11464.3  (16866.55)
14.Other Pre-specified Outcome
Title 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
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: Percentage of blood ejected from the LV
-6.5  (7.26) -5.6  (10.52) 0.3  (8.87) -2.5  (9.96)
15.Other Pre-specified Outcome
Title Phase 2: Change in Absolute Left Ventricular End Systolic Volume (LVESV) From Baseline to Month 12
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Mean (Standard Deviation)
Unit of Measure: mL
40.7  (59.79) 66.8  (103.12) 9.9  (49.27) 37.7  (69.09)
16.Other Pre-specified Outcome
Title Phase 1 and Phase 2: All Subject Deaths Through 36 Months
Hide Description 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.
Time Frame 36 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Very Low Dose MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose All MYDICAR® Placebo
Hide Arm/Group Description:
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.
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.
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.
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.
All participants who received a single infusion of MYDICAR® at any dose during the Phase 1 or Phase 2 studies.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 3 11 11 12 37 14
Measure Type: Number
Unit of Measure: participants
Deaths within 12 months 0 2 0 0 2 3
Deaths after 12 months 1 2 3 2 8 3
Cardiovascular deaths on study 0 2 0 0 2 1
Cardiovascular deaths in long-term follow-up 1 2 1 2 6 4
All deaths during 36 months 1 4 3 2 10 6
17.Post-Hoc Outcome
Title Phase 2: Selected Clinical Outcomes During 12-month Study Period
Hide Description Incidences of key clinical endpoints as adjudicated by the blinded Clinical Endpoint Committee.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description:
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.
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.
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.
Single dose of placebo administered by antegrade epicardial coronary artery infusion.
Overall Number of Participants Analyzed 8 8 9 14
Measure Type: Number
Unit of Measure: percentage of participants
Fatal cardiovascular event 12.5 0 0 7.1
Worsening heart failure 50.0 37.5 22.2 50.0
Myocardial infarction 0 0 0 14.3
Heart failure-related hospitalization 50.0 37.5 22.2 42.9
Silent myocardial infarction 0 0 0 0
Receipt of left ventricular assist device 0 12.5 0 14.3
Heart transplant 0 25.0 11.1 7.1
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.
Adverse Event Reporting Description These data include all TEAEs occurring during the Phase 1 or Phase 2 parts of this study.
 
Arm/Group Title MYDICAR® Very Low Dose MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Hide Arm/Group Description 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. 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. 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. 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. Single dose of placebo administered by antegrade epicardial coronary artery infusion.
All-Cause Mortality
MYDICAR® Very Low Dose MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
MYDICAR® Very Low Dose MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/3 (66.67%)   6/11 (54.55%)   5/11 (45.45%)   4/12 (33.33%)   9/14 (64.29%) 
Cardiac disorders           
Cardiac failure  1  0/3 (0.00%)  1/11 (9.09%)  4/11 (36.36%)  2/12 (16.67%)  3/14 (21.43%) 
Cardiac failure congestive  1  1/3 (33.33%)  2/11 (18.18%)  1/11 (9.09%)  1/12 (8.33%)  3/14 (21.43%) 
Cardiac failure acute  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Acute myocardial infarction  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Arrhythmia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Atrial fibrillation  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Cardiorenal syndrome  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Angina unstable  2  1/3 (33.33%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Cardiogenic shock  2  1/3 (33.33%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Eye disorders           
Vision blurred  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Gastrointestinal disorders           
Gastric ulcer haemorrhage  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Melaena  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Pancreatitis  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
General disorders           
Chest pain  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  2/14 (14.29%) 
Chest discomfort  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Pyrexia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Sudden death  2  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Infections and infestations           
Pneumonia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Pneumonia staphylococcal  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Injury, poisoning and procedural complications           
Overdose  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Fall  2  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Investigations           
Colonoscopy  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Metabolism and nutrition disorders           
Diabetes mellitus  2  1/3 (33.33%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)           
Colon cancer metastatic  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Nervous system disorders           
Brain stem stroke  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Syncope  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Transient ischaemic attack  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Renal and urinary disorders           
Renal failure acute  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Respiratory, thoracic and mediastinal disorders           
Chronic obstructive pulmonary disease  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Respiratory failure  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Orthopnoea  2  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Skin and subcutaneous tissue disorders           
Rash maculo-papular  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Surgical and medical procedures           
Catheterization cardiac  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Heart transplant  1  1/3 (33.33%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA v 11.1
2
Term from vocabulary, MedDRA (11.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
MYDICAR® Very Low Dose MYDICAR® Low Dose MYDICAR® Mid Dose MYDICAR® High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/3 (66.67%)   11/11 (100.00%)   11/11 (100.00%)   11/12 (91.67%)   13/14 (92.86%) 
Blood and lymphatic system disorders           
Anemia  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Cardiac disorders           
Angina pectoris  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
Arrhythmia  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Atrial fibrillation  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Cardiac aneurysm  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Cardiac failure  1  0/3 (0.00%)  0/11 (0.00%)  2/11 (18.18%)  2/12 (16.67%)  3/14 (21.43%) 
Cardiac failure congestive  1  0/3 (0.00%)  0/11 (0.00%)  3/11 (27.27%)  0/12 (0.00%)  0/14 (0.00%) 
Cardiogenic shock  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Extrasystoles  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Intracardiac thrombus  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Palpitations  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Supraventricular tachycardia  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Tachycardia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Ventricular fibrillation  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  3/12 (25.00%)  1/14 (7.14%) 
Ventricular tachycardia  1  0/3 (0.00%)  2/11 (18.18%)  2/11 (18.18%)  2/12 (16.67%)  2/14 (14.29%) 
Congenital, familial and genetic disorders           
Hydrocele  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Ear and labyrinth disorders           
External ear disorder  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Endocrine disorders           
Hypothyroidism  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Eye disorders           
Vision blurred  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Gastrointestinal disorders           
Abdominal distension  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Abdominal pain  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Ascites  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Constipation  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  4/14 (28.57%) 
Diarrhea  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Dyspepsia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Epigastric discomfort  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Gastric ulcer  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Nausea  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
General disorders           
Catheter site hematoma  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Catheter site hemorrhage  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  1/14 (7.14%) 
Chest discomfort  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  1/14 (7.14%) 
Chest pain  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  3/12 (25.00%)  1/14 (7.14%) 
Chills  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  1/14 (7.14%) 
Fatigue  1  1/3 (33.33%)  3/11 (27.27%)  2/11 (18.18%)  2/12 (16.67%)  4/14 (28.57%) 
Feeling jittery  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Gait disturbance  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Influenza like illness  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Infusion site pain  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Edema  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Edema peripheral  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Puncture site pain  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Pyrexia  1  1/3 (33.33%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
Venipuncture site thrombosis  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Hepatobiliary disorders           
Jaundice  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Infections and infestations           
Bronchitis  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  1/12 (8.33%)  2/14 (14.29%) 
Candidiasis  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Cellulitis  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Central line infection  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Gastric ulcer Helicobacter  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Herpes zoster  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Influenza  1  1/3 (33.33%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Pneumonia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Respiratory tract infection  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Sinusitis  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
Upper respiratory tract infection  1  0/3 (0.00%)  3/11 (27.27%)  1/11 (9.09%)  1/12 (8.33%)  2/14 (14.29%) 
Viral infection  1  0/3 (0.00%)  0/11 (0.00%)  2/11 (18.18%)  0/12 (0.00%)  0/14 (0.00%) 
Injury, poisoning and procedural complications           
Contusion  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  2/12 (16.67%)  1/14 (7.14%) 
Excoriation  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  2/14 (14.29%) 
Eye injury  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Fall  1  0/3 (0.00%)  3/11 (27.27%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Head injury  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Mouth injury  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Post procedural discharge  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Tendon injury  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Investigations           
Blood bilirubin increased  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Blood creatinine phosphokinase increased  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Blood creatinine phosphokinase MB increased  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Blood magnesium decreased  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Blood triglycerides increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Blood uric acid increased  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Carcinoembryonic antigen increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Gallop rhythm present  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
Hepatic enzyme increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  1/14 (7.14%) 
International normalized ratio increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Liver function test abnormal  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  1/14 (7.14%) 
Mean cell volume increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Muscle enzyme increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Prostatic specific antigen increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Transaminases increased  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Troponin increased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Vitamin D decreased  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Weight increased  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Metabolism and nutrition disorders           
Anorexia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Decreased appetite  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Dehydration  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Fluid overload  1  0/3 (0.00%)  2/11 (18.18%)  1/11 (9.09%)  2/12 (16.67%)  1/14 (7.14%) 
Fluid retention  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Gout  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  1/12 (8.33%)  4/14 (28.57%) 
Hyperkalemia  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
Hyperlipidemia  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Hyperuricemia  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Hypervolemia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Hypokalemia  1  1/3 (33.33%)  3/11 (27.27%)  2/11 (18.18%)  0/12 (0.00%)  0/14 (0.00%) 
Hyponatremia  1  1/3 (33.33%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Hypovolemia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Type 2 diabetes mellitus  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Musculoskeletal and connective tissue disorders           
Arthralgia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  2/14 (14.29%) 
Back pain  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Bone disorder  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Bursitis  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Groin pain  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Muscle spasms  1  1/3 (33.33%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Muscle tightness  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Muscular weakness  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Musculoskeletal chest pain  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  1/14 (7.14%) 
Musculoskeletal discomfort  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  1/14 (7.14%) 
Myalgia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Pain in extremity  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  1/14 (7.14%) 
Nervous system disorders           
Asterixis  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Dizziness  1  0/3 (0.00%)  2/11 (18.18%)  2/11 (18.18%)  1/12 (8.33%)  4/14 (28.57%) 
Dizziness postural  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Headache  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  2/14 (14.29%) 
Hepatic encephalopathy  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Hypoesthesia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Neurological symptom  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Nystagmus  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Paresthesia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Restless leg syndrome  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Somnolence  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Syncope  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Psychiatric disorders           
Alcoholism  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Anxiety  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  2/14 (14.29%) 
Bruxism  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Depression  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  3/14 (21.43%) 
Insomnia  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  1/14 (7.14%) 
Mental status changes  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Somnambulism  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Renal and urinary disorders           
Azotemia  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  2/14 (14.29%) 
Hematuria  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Renal failure  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Renal failure acute  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Renal impairment  1  0/3 (0.00%)  0/11 (0.00%)  2/11 (18.18%)  1/12 (8.33%)  1/14 (7.14%) 
Reproductive system and breast disorders           
Breast disorder  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Gynecomastia  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Variocele  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Respiratory, thoracic and mediastinal disorders           
Asthma  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Cough  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  2/14 (14.29%) 
Dyspnea  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  2/12 (16.67%)  1/14 (7.14%) 
Dyspnea exertional  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Epistaxis  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Hemoptysis  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Nasal congestion  1  0/3 (0.00%)  1/11 (9.09%)  2/11 (18.18%)  1/12 (8.33%)  1/14 (7.14%) 
Oropharyngeal discomfort  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Oropharyngeal pain  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Orthopnea  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Productive cough  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Pulmonary hypertension  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Rales  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  1/12 (8.33%)  0/14 (0.00%) 
Sleep apnea syndrome  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Wheezing  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  1/14 (7.14%) 
Skin and subcutaneous tissue disorders           
Cold sweat  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Ecchymosis  1  0/3 (0.00%)  2/11 (18.18%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Hemorrhage subcutaneous  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Pruritus  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Rash  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Rash generalized  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Skin ulcer  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Stevens-Johnson syndrome  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Urticaria  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Surgical and medical procedures           
Cataract operation  1  0/3 (0.00%)  0/11 (0.00%)  0/11 (0.00%)  0/12 (0.00%)  1/14 (7.14%) 
Vascular disorders           
Deep vein thrombosis  1  0/3 (0.00%)  1/11 (9.09%)  0/11 (0.00%)  0/12 (0.00%)  0/14 (0.00%) 
Hematoma  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  2/14 (14.29%) 
Hypertension  1  0/3 (0.00%)  0/11 (0.00%)  1/11 (9.09%)  0/12 (0.00%)  0/14 (0.00%) 
Hypotension  1  0/3 (0.00%)  1/11 (9.09%)  1/11 (9.09%)  2/12 (16.67%)  1/14 (7.14%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (11.1)
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
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jeffrey J. Rudy, Vice President
Organization: Celladon Corporation
Phone: 1 858-366-4288
EMail: jrudy@celladon.net
Layout table for additonal information
Responsible Party: Celladon Corporation
ClinicalTrials.gov Identifier: NCT00454818     History of Changes
Other Study ID Numbers: CELL-001
CUPID Trial ( Registry Identifier: NCT00454818 )
First Submitted: March 30, 2007
First Posted: April 2, 2007
Results First Submitted: June 25, 2014
Results First Posted: August 20, 2014
Last Update Posted: August 20, 2014