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Study of AB-1002 gene therapy given through coronary arteries in adults with severe (NYHA Class III) heart failure caused by non-ischemic cardiomyopathy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure, a condition where the heart muscle becomes weak and enlarged, making it difficult to pump blood effectively throughout the body. The research evaluates a new gene therapy called AB-1002, which is given as a single injection directly into the heart's arteries.

The purpose of this research is to determine how well AB-1002 works and how safe it is compared to placebo in treating heart failure. The treatment involves a one-time delivery of the medication through intracoronary infusion, which means the medicine is administered directly into the blood vessels that supply the heart muscle.

During the study, participants will be monitored for one year after receiving either AB-1002 or placebo. The research will track changes in heart function, exercise capacity, and overall well-being. AB-1002 is a type of gene therapy that uses a modified virus to deliver genetic material designed to help improve heart function in people with heart failure.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your heart function will be evaluated through a transthoracic echocardiography (heart ultrasound)

    You will need to complete a six-minute walk test to assess your exercise capacity

    Your heart failure symptoms will be classified according to the New York Heart Association (NYHA) Class III criteria

  2. Step 2

    Treatment administration

    You will receive a single dose of either AB-1002 or placebo through a coronary artery infusion

    The medication will be administered by a heart specialist in a controlled medical setting

    This procedure involves delivering the treatment directly to your heart through blood vessels

  3. Step 3

    Follow-up period - 52 weeks

    Your heart function will be monitored at weeks 4, 12, and 36 using heart ultrasound

    Regular assessments will include heart function tests, walking tests, and quality of life questionnaires

    Blood tests will be performed to measure heart-related substances

    Your heart failure symptoms will be regularly evaluated

    Any hospital visits related to heart failure will be recorded

  4. Step 4

    Safety monitoring

    Throughout the 52-week period, you will undergo regular health checks including:

    Blood tests to monitor your general health

    Vital signs measurements

    Heart rhythm recordings (electrocardiograms)

    Any health changes will be documented and assessed

  5. Step 5

    Final evaluation

    At the end of 52 weeks, a final assessment will include:

    Heart ultrasound to measure heart function

    Walking test to evaluate exercise capacity

    Quality of life questionnaire completion

    Final review of your heart failure symptoms

Who can join the trial?

19 criteria

  • Must be 18 years or older when signing the consent form
  • Have chronic non-ischemic cardiomyopathy (a heart muscle condition not caused by blocked arteries)
  • Have a left ventricular ejection fraction (a measure of heart pumping ability) between 15% and 35% as shown on heart ultrasound
  • Able to walk more than 50 meters during a 6-minute walking test
  • Have stable Class III heart failure (significant limitation in physical activity) for at least 4 weeks while on appropriate medications
  • Must be on stable doses of heart failure medications for at least 30 days, including:
    • Blood pressure medications
    • Beta blockers
    • Water pills
    • Diabetes medications (SGLT2 inhibitors)
    • If applicable, must have had a cardiac resynchronization device (special pacemaker) for at least 90 days or an implantable defibrillator for at least 30 days
    • Women who can become pregnant must use effective birth control methods, such as:
      • Intrauterine device in place for at least 90 days
      • Abstinence
      • Partner's surgical sterilization for more than 180 days
      • Hormonal contraceptives for more than 90 days
      • Male participants must agree to contraception requirements:
        • Not donate sperm for 6 months after treatment
        • Use barrier methods if sexually active
        • Previous vasectomy (at least 180 days prior) is acceptable
        • Must be suitable for coronary artery procedure according to heart specialist
        • All sexually active participants must use barrier methods of protection for 6 months after treatment

Who cannot join the trial?

15 criteria

  • Age below 18 years or above 65 years
  • Presence of ischemic cardiomyopathy (heart muscle disease caused by coronary artery disease)
  • Heart failure symptoms classified as NYHA Class I, II or IV (NYHA III is required for participation)
  • Previous heart transplant or presence of a mechanical heart support device
  • Active infections or severe inflammatory conditions
  • Pregnant or breastfeeding women
  • Participation in other clinical trials within the past 30 days
  • Significant kidney or liver dysfunction
  • History of cancer within the past 5 years
  • Uncontrolled high blood pressure
  • Severe heart valve disease
  • History of blood clotting disorders
  • Allergic reactions to similar medications
  • Inability to provide informed consent
  • Conditions that might interfere with study procedures or follow-up visits
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Investigated drugs

AB-1002 is an investigational therapy administered through intracoronary infusion (delivered directly into the heart's blood vessels). This medication is being studied for treating patients with heart failure, specifically those with non-ischemic cardiomyopathy (a type of heart muscle disease not caused by blocked arteries). The therapy aims to help patients who have severe symptoms of heart failure classified as New York Heart Association (NYHA) Class III, where patients experience marked limitations in physical activity.

What is already known about the treatment

AB-1002 - An investigational drug administered through intracoronary infusion (directly into the heart arteries) being studied for the treatment of non-ischemic cardiomyopathy in patients with Class III heart failure. The medication is currently in Phase 2 clinical trials to evaluate its efficacy, safety, and tolerability compared to placebo. While the exact molecular mechanism of action is still under investigation in clinical trials, this treatment is being developed to target heart failure symptoms in patients with weakened heart muscle not caused by blocked arteries (non-ischemic cardiomyopathy). The drug represents a novel therapeutic approach in cardiology, being tested through a single antegrade intracoronary delivery method.

Investigated diseases

  • Non-ischemic Cardiomyopathy

    A heart muscle disease not caused by coronary artery disease or heart attacks. In this condition, the heart muscle becomes enlarged, thick, or rigid, making it harder for the heart to pump blood effectively. The condition develops gradually and can affect people of any age. It often leads to heart chambers becoming dilated and weakened over time. The disease can be inherited or develop due to various factors including infections, certain medications, or other medical conditions.

  • Congestive Heart Failure

    A chronic condition where the heart muscle cannot pump blood as well as it should. The condition causes blood and fluid to back up in the body, leading to congestion in the lungs, legs, and other tissues. This results in symptoms such as shortness of breath, fatigue, and swelling in the legs and ankles. The condition typically progresses gradually, affecting the body's ability to meet its oxygen needs. The heart tries to compensate by enlarging and developing more muscle mass.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-510581-17-00Protocol codeASK-CHF2-CS201Estimated enrolment323 patientsSponsorAskbio Inc.

sourced from the EU Clinical Trials Register and site verification

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