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. (Show More)
Indication	Heart Failure with Reduced Ejection Fraction
Compound Name	AAV1-CMV-SERCA2a (SRD-001)
Sponsor	Sardocor Corp.
Funder Type	Industry
Status	Recruiting
Enrollment Count	57

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
Dose 1	3 x 10^13 vg
Dose 2	4.5 x 10^13 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States

Has US IND	True
Recent Updates	Update given at ASGCT 2024