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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction
NCTID
NCT06061549
(View at clinicaltrials.gov)
Description
The goal of this clinical trial is to test an experimental gene therapy in participants with heart failure with preserved ejection fraction, also known as diastolic heart failure. The main questions it aims to answer are: * safety and tolerability of the gene therapy; and * whether the gene therapy helps the heart ventricles relax during filling. Participants will undergo a one-time infusion of the gene therapy in the cardiac catheterization laboratory and then be followed for safety and effects on left-sided filling pressures while exercising. The first year will have multiple in-person visits followed by 4 years of biannual phone calls.
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Development Status
Active
Indication
Heart Failure, Diastolic, Heart Failure With Preserved Ejection Fraction
Disease Ontology Term
DOID:9775
Compound Name
SRD-001
Compound Description
AAV1-CMV-SERCA2a
Sponsor
Sardocor Corp.
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
10
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
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2023-07-30
Completion Date
2029-08
Last Update
2023-09-29
Participation Criteria
Eligible Age
>=50 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Fast Track
Recent Updates
First patients dosed Q1 2024, product/indication combo granted Fast Track designation
Resources/Links
Clinical Publications
Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality
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
News and Press Releases
MEDERAâS SARDOCOR ANNOUNCES FAST TRACK DESIGNATION AND DOSING OF 3 PATIENTS IN FIRST-IN-HUMAN HFpEF GENE THERAPY TRIAL
Preclinical Publications
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
Reversal of cardiac dysfunction after long-term expression of SERCA2a by gene transfer in a pre-clinical model of heart failure