A Phase 2, adaptive, double-blinded, placebo-controlled, randomized, multi-centre trial to evaluate the efficacy, safety and tolerability of intracoronary infusion of AB-1002 in adult subjects with New York Heart Association (NYHA) Class III heart fail...
Recipient organisationNIHR Guy's and St Thomas' Clinical Research Facility
NIHR supportRecorded as supported by this research centre
PeriodMar 2025 — Ongoing
In plain English
AI plain-English summary
A single infusion of a gene therapy called AB-1002, delivered directly into the heart’s main arteries, is being tested in a placebo-controlled trial for people with severe congestive heart failure. This matters because congestive heart failure—where the heart cannot pump blood efficiently, causing breathlessness and fatigue—has limited treatment options for severe cases. Existing therapies often only manage symptoms, and many patients continue to decline. AB-1002 aims to address the underlying problem by delivering a corrective gene to heart muscle cells, potentially improving the heart’s ability to pump. If the therapy works, it could offer a new treatment pathway for patients who currently have few choices. Success would mean a single infusion might slow or reverse disease progression, reducing hospitalisations and improving quality of life. The findings will determine whether larger, confirmatory studies are warranted. This is a late-stage clinical trial, not fundamental science—it directly tests a candidate treatment in humans, with the goal of changing clinical practice for a common and debilitating condition.
View original technical description
The GenePHIT study aims to evaluate the effectiveness and safety of a new gene therapy, AB-1002, in individuals with congestive heart failure (CHF). CHF is a condition in which the heart is unable to pump blood efficiently and to meet the demands of the body, leading to symptoms like breathlessness and fatigue. With limited treatment options for severe cases, this study explores AB-1002 as a potential treatment for CHF. The study's primary objective is to assess how participants respond to 2 different dose levels of AB-1002 compared to a placebo over 52 weeks. Researchers will measure factors such as heart failure-related deaths, heart function, and participants' ability to perform physical activities. Additionally, the study will evaluate the safety of AB-1002 in CHF patients, monitoring any medical issues that arise after treatment. Participants will receive AB-1002 or a placebo through a single infusion into the heart's main arteries. Various tests and procedures, including imaging and regular health check-ups, will be conducted throughout the study period. The study will span over five years, including a screening period, a treatment period of one year, and long-term follow-up. Eligible participants must be at least 18 years old, diagnosed with heart failure, and meet specific health criteria. Those with certain heart conditions or recent heart surgeries may not qualify. Potential benefits include the possibility of a new treatment option for CHF, while risks include the uncertainty of AB-1002's effects and potential side effects. The study's findings may provide valuable insights into the potential of AB-1002 as a treatment for CHF and determine the need for further research in this area.
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