Ignite Creation Date:
2024-05-06 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 1:53 PM
Study NCT ID:
NCT04703842
Status:
RECRUITING
Last Update Posted:
2024-03-26
First Post:
2021-01-07
Brief Title:
Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction
Sponsor:
Sardocor Corp
Organization:
Sardocor Corp
Study Overview
Official Title:
A Phase 12 Trial of the Safety and Efficacy of SRD-001 AAV1SERCA2a in Subjects With Heart Failure With Reduced Ejection Fraction
Status:
RECRUITING
Status Verified Date:
2024-03
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:
MUSIC-HFrEF1
Brief Summary:
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
Detailed Description:
MUSIC-HFrEF1 is an interventional study of SRD-001 an adeno-associated virus serotype 1 AAV1 vector expressing the transgene for sarcoendoplasmic reticulum Ca2 ATPase 2a isoform SERCA2a in anti-AAV1 neutralizing antibody NAb negative subjects with ischemic or non-ischemic cardiomyopathy and New York Heart Association NYHA class IIIIV symptoms of heart failure with reduced ejection fraction HFrEF
The Phase 1 trial is an open-label uncontrolled study investigating two doses of SRD-001 at 3E13 and 45E13 viral genomes vg in 3-4 participants at each dose level
The Phase 2 trial is a randomized double-blind placebo-controlled trial with prospective assignment to a single dose of either SRD-001 or placebo in a 11 ratio and a total sample size of N50
The Phase 1 trial is an open-label uncontrolled study investigating two doses of SRD-001 at 3E13 and 45E13 viral genomes vg in 3-4 participants at each dose level
The Phase 2 trial is a randomized double-blind placebo-controlled trial with prospective assignment to a single dose of either SRD-001 or placebo in a 11 ratio and a total sample size of N50
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None