NCT04179643

AB-1002 in Patients With Class III Heart Failure

Phase 1 Open Label, Dose Escalation Trial of Intracoronary Infusion of AB-1002 in Subjects With NYHA Class III Heart Failure

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 17 (estimated)
• Sponsor: AskBio Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1, prospective, multi-center, open-label, sequential dose escalation study to explore the safety, feasibility, and efficacy of a single intracoronary infusion of AB-1002 in patients with NYHA Class III heart failure. Patients with non-ischemic cardiomyopathy will be enrolled until up to 17 subjects have received infusions of investigational product. All patients will be followed until 12 months post treatment intervention, and then undergo long-term follow-up via semi-structured telephone questionnaires every 6 months for an additional 24 months (+/- 30 days).

Detailed description

The safety endpoints will be assessed over the 12-month follow-up period * Adverse Events * Clinical laboratory tests * Vital signs * Electrocardiograms Efficacy Endpoints The efficacy endpoints assessed from baseline to 6 and 12 months will include the following: * Observed and changes from baseline in peak VO2 assessed by cardiopulmonary exercise testing * Observed and changes from baseline in 6-minute walk test * Observed and changes from baseline in NYHA Classification * Total number of days alive out-of-hospital The secondary efficacy endpoints will explore efficacy. Functional endpoints will be assessed as changes from baseline to 6 and 12 months following investigational product administration as indicated. These endpoints include: Functional Status \& Hospitalizations * Peak VO2 assessed by cardiopulmonary exercise testing * 6-minute walk test * New York Heart Association (NYHA) Classification * Total number of days alive out-of-hospital (as well as total days out-of-hospital as a % of total days alive post study intervention) Physiologic Assessments at 6 and 12 months compared to baseline * Echocardiographic assessments of LVEF, LVEVD, LVEDVI, VLESV, LVEVI, SpI and GLSand degree of mitral regurgitation * NT-proBNP level Quality of Life at Week 8, 6 and 12 months compared to baseline o Health related quality of life as assessed by Minnesota Living with Heart Failure Questionnaire (MLWHFQ) and Kansas City Cardiomyopathy Questionnaire The following endpoints will also be measured over the 12 month follow-up period and long-term follow-up period (until month 36 post-intervention): * Survival * Cardiac transplantation * Left ventricular assist device (LVAD) implantation

Conditions

• Congestive Heart Failure
• Heart Failure
• Heart Disease, Ischemic
• Cardiovascular Diseases
• Heart Failure, Systolic
• Heart Failure，Congestive
• Heart Arrhythmia
• Heart Failure, Diastolic
• Heart; Complications

Interventions

Type	Name	Description
BIOLOGICAL	3 x 10e13vg AB-1002	There are 2 components to AB-1002. The first is an active I-1 transgene (AA 1-65 with T35D), and the second is the vector, BNP116, which delivers the gene selectively to the heart after intracoronary administration.

Timeline

• Start date: 2019-11-20
• Primary completion: 2024-08-20
• Completion: 2026-09-30
• First posted: 2019-11-27
• Last updated: 2025-04-08

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study