Ignite Creation Date:
2024-05-05 @ 5:25 PM
Last Modification Date:
2024-10-26 @ 9:31 AM
Study NCT ID:
NCT00454818
Status:
COMPLETED
Last Update Posted:
2014-08-20
First Post:
2007-03-30
Brief Title:
Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure
Sponsor:
Celladon Corporation
Organization:
Celladon Corporation
Study Overview
Official Title:
A Phase 12 Trial of Intracoronary Administration of MYDICAR AAV1SERCA2a in Subjects With Heart Failure in Two Stages Open-Label Sequential Dose-Escalation Cohorts and Randomized Double-Blind Placebo-Controlled Parallel Cohorts
Status:
COMPLETED
Status Verified Date:
2014-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CUPID
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 550000 patients are newly diagnosed each year The estimated direct and indirect cost of HF in the United States US for 2006 will be 296 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 AAV1SERCA2a MYDICAR refers to AAV1SERCA2a drug product intended for administration by percutaneous delivery
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 AAV1SERCA2a MYDICAR refers to AAV1SERCA2a drug product intended for administration by percutaneous delivery
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| CUPID Trial | REGISTRY | NCT00454818 | None |