Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction

NCT06061549 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: SRD-001-1003
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Heart Failure, Diastolic; Heart Failure With Preserved Ejection Fraction

Outcome Measures

Primary Outcomes (1)

• Change in pulmonary capillary wedge pressure (PCWP)

  PCWP assessed by right heart catheterization; change in mmHg from baseline

  Week 24 and Week 52

Secondary Outcomes (11)

• Change in PCWP at 20W exercise

  Week 24 and Week 52

• Change in oxygen uptake (VO2) during exercise

  Week 24 and Week 52

• Change in left ventricular (LV) relaxation

  Week 24 and Week 52

• Change in NT-proBNP

  Week 24 and Week 52

• Change in left atrial end systolic volume

  Week 24 and Week 52

Study Arms (1)

SRD-001 Gene Therapy

EXPERIMENTAL

AAV1/SERCA2a 3E13 vg

Biological: AAV1/SERCA2a

Interventions

AAV1/SERCA2a BIOLOGICAL

3E13 viral genomes fixed dose

SRD-001 Gene Therapy

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Willing and able to provide informed consent
• Negative for anti-AAV1 neutralizing antibodies
• NYHA class II or III
• Left ventricular ejection fraction ≥ 50%
• Evidence of resting or exercise-induced left ventricle filling pressure
• On oral diuretic therapy
• Adequate birth control

You may not qualify if:

• NYHA class IV
• Heart failure requiring hospitalization in the past 3 months
• Manifested or provocable ischemic heart disease
• Atrial fibrillation
• History of congenital heart disease, restrictive or infiltrative cardiomyopathy, hypertrophic cardiomyopathy, acute myocarditis, pericardial disease, uncorrected thyroid disease or discrete left ventricular (LV) aneurysm
• History of amyloidosis
• Untreated left-sided valvular disease
• Severe COPD
• BMI \> 50 kg/m\^2
• Severe liver, kidney or hematologic dysfunction
• Cancer within the past 5 years
• Unstable concurrent conditions

Sponsors & Collaborators

• Sardocor Corp.: lead

Study Sites (2)

Duke University

Durham, North Carolina, 27705, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Heart Failure, Diastolic

Condition Hierarchy (Ancestors)

Heart Failure; Heart Diseases; Cardiovascular Diseases

Central Study Contacts

Sardocor

CONTACT

858-752-2941; info@sardocorcorp.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2023
• First Posted: September 29, 2023
• Study Start: August 24, 2023
• Primary Completion: August 1, 2025
• Study Completion (Estimated): August 1, 2029
• Last Updated: September 29, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)