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Summary
A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure
NCTID
NCT01643330
(View at clinicaltrials.gov)
Description
The purpose of this trial is to assess whether MYDICAR can reduce the frequency and/or delay heart failure related hospitalizations in persons with advanced heart failure when added to their maximal and optimized therapy.
(Show More)
Development Status
🔄
Inactive
Indication
Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies
Disease Ontology Term
🔄
DOID:9651
Compound Name
🔄
AAV1-CMV-Serca2a
Sponsor
Celladon Corporation
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
250 (ACTUAL)
Results Posted
Not Available
Therapy Information
Target Gene/Variant
🔄
SERCA2A
Therapy Type
🔄
Gene transfer
Therapy Route
🔄
In-vivo
Mechanism of Action
🔄
Overexpression of protective allele/gene
Route of Administration
🔄
Intracoronary
Drug Product Type
🔄
Viral vector
Delivery System
🔄
Viral transduction
Vector Type
🔄
AAV1
Dose 1
🔄
6E11 DRP
Dose 2
🔄
3E12 DRP
Dose 3
🔄
1E13 DRP
Study Record Dates
Current Stage
Phase2
Submit Date
2012-07-16
Completion Date
2016-02
Last Update
2016-03-24
Participation Criteria
Eligible Age
18 Years - 80 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
54
Locations
Netherlands,Sweden,Belgium,Hungary,United States,Denmark,Poland,United Kingdom,Israel,Germany
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Failed to demonstrate improvement in ventricular remodelling, dosing was too low, clinical trials sponsored by Sardocor (using related product) are using higher doses
Resources/Links
Clinical Publications
🔄
Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial
Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial
Effect of intracoronary administration of AAV1/SERCA2a on ventricular remodelling in patients with advanced systolic heart failure: results from the AGENT-HF randomized phase 2 trial
Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality
Design of a phase 2b trial of intracoronary administration of AAV1/SERCA2a in patients with advanced heart failure: the CUPID 2 trial (calcium up-regulation by percutaneous administration of gene therapy in cardiac disease phase 2b)
Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure
Preclinical Publications
🔄
Reversal of cardiac dysfunction after long-term expression of SERCA2a by gene transfer in a pre-clinical model of heart failure
Recirculating cardiac delivery of AAV2/1SERCA2a improves myocardial function in an experimental model of heart failure in large animals
Related NCTID
🔄
Phase 2: NCT01966887
Phase 1/2: NCT00454818