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Clinical Trial Report
Gene Therapy Trial Report
Summary
Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction
NCTID
NCT04703842
(View at clinicaltrials.gov)
Description
It is believed that targeted SERCA2a enzyme replacement in HFrEF patients will correct defective intracellular Ca2+ hemostasis, resulting in improved cardiac contractile function and energetics which will, in turn, translate to improved clinical outcomes. Additionally, it is hypothesized that correcting SERCA2a dysfunction will also improve coronary blood flow through correction of the impaired endothelium-dependent nitric oxide-mediated vasodilatation observed in heart failure.
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Development Status
Active
Indication
Congestive Heart Failure, Heart Failure With Reduced Ejection Fraction (HFrEF)
Disease Ontology Term
DOID:9651
Compound Name
SRD-001
Compound Description
AAV1-CMV-SERCA2a
Sponsor
Sardocor Corp.
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
57
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
Intraarterial
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV1
Editor Type
none
Dose 1
3E13 vg
Dose 2
4.5E13 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-01-07
Completion Date
2028-12
Last Update
2024-03-26
Participation Criteria
Eligible Age
18 Years - 80 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
5
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Anticipated Phase 2b in 4Q2024-1Q2025
Resources/Links
Clinical Publications
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
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)
Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial
Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality
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
Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device - the SERCA-LVAD TRIAL
Preclinical Publications
Reversal of cardiac dysfunction after long-term expression of SERCA2a by gene transfer in a pre-clinical model of heart failure
Primary Effect of SERCA 2a Gene Transfer on Conduction Reserve in Chronic Myocardial Infarction
SERCA2a gene transfer decreases sarcoplasmic reticulum calcium leak and reduces ventricular arrhythmias in a model of chronic heart failure
Related NCTID
Phase 2: NCT00534703