Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure
This research study is looking at a new treatment called AB-1002 for adults with a specific type of heart failure known as non-ischemic cardiomyopathy. This is when the heart muscle is weakened, but not due to blocked arteries. Participants will have 'NYHA Class III' symptoms, meaning they experience significant limitations during everyday activities due to their heart failure. The study is in 'Phase 2', which means it's still an early stage of testing. Doctors want to find out if AB-1002 is safe and whether it can help improve heart failure symptoms. Some participants will receive the active treatment, while others will receive a placebo (a dummy treatment). This is done 'double-blinded,' so neither the patients nor their doctors will know who is getting which until the study ends. The treatment is given directly into the heart's arteries.
At a glance
What is this study about?
This study is investigating a new treatment called AB-1002 for a specific type of heart failure. Heart failure means your heart isn't pumping blood as well as it should, leading to symptoms like tiredness and breathlessness. This particular study focuses on 'non-ischemic heart failure,' which means the heart weakness isn't caused by problems with the blood supply to the heart itself, like blocked arteries. Participants in this study will have 'NYHA Class III' heart failure, meaning their daily activities are noticeably limited by their symptoms.
The main goal of this study is to see if AB-1002 is safe for people with this condition and if it helps improve their symptoms and overall heart health. The treatment is given directly into the arteries of the heart. To get clear results, the study is designed as 'double-blinded' and 'placebo-controlled.' This means some patients will receive AB-1002, and others will receive a placebo (a treatment with no active drug), but neither you nor your doctor will know which you are receiving until the study is over. This helps ensure the results are unbiased and reliable.
About 90 to 150 people will take part. Participants will be organised into three groups: two groups will receive different dose levels of AB-1002, and one group will receive the placebo. Your health will be carefully monitored for about a year after receiving the treatment, and then for another four years to track your progress long-term. This type of research is vital for developing new treatments that could improve the lives of people living with heart failure.
Key takeaways
- This study is testing a new treatment (AB-1002) for non-ischemic heart failure.
- It's for people with NYHA Class III heart failure symptoms.
- You might receive the new treatment or a dummy treatment (placebo).
- Neither you nor your doctor will know which you receive during the study.
- Participation involves regular check-ups for about a year, then annual checks for four more years.
- The study aims to check the treatment's safety and effectiveness.
Who may be eligible?
To be considered for this study, you need to be at least 18 years old and have a long-term condition called non-ischemic cardiomyopathy, which is a type of heart failure. Your heart's pumping ability (measured by LVEF) needs to be between 15% and 35%. You also must be able to walk more than 50 meters in a timed test despite your heart condition.
Your heart failure symptoms should be stable and categorised as NYHA Class III for at least four weeks. This means your daily activities are significantly limited by your condition, but you don't have symptoms at rest. You also need to have been on standard heart failure medications like beta blockers and ACE inhibitors (or similar) for a certain period, with stable doses. If you have any implanted heart devices like a pacemaker or defibrillator, they must have been put in place a certain time before the study.
Women who could become pregnant must agree to use effective birth control throughout the study and for six months after receiving the treatment. This includes various reliable methods like surgical sterilisation, IUDs, certain barrier methods, or hormonal birth control. Abstinence is also an option if it's your usual lifestyle.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have non-ischemic heart failure (weak heart muscle not due to blocked arteries)?
- Are your heart failure symptoms stable and generally limit your daily activities but not at rest (NYHA Class III)?
- Have you been on your usual heart failure medications with stable doses for a while?
- If you're a woman who could become pregnant, are you able and willing to use specific birth control methods throughout the study?
What does participation involve?
If you decide to take part, you'll be randomly assigned to one of three groups: either one of two different doses of the AB-1002 treatment, or a placebo (a dummy treatment). You won't know which group you're in, and neither will your doctors, until the study is finished. The treatment itself is given through an infusion directly into a heart artery.
The main part of the study will last about 52 weeks (one year) after you receive the treatment. During this time, you'll have regular check-ups and assessments to see how you're doing, check your safety, and see if the treatment is working. After this first year, there's a longer-term follow-up period of another four years. During these four years, study staff will contact you twice a year for the first two years, and then once a year for the remaining two years. These contacts will be to check on your health and safety. The total time you'll be involved with the study, including initial and long-term follow-up, is about five years.
Potential risks and benefits
Locations (96)
- Cardiology P.C. BirminghamVerified postcodeBirmingham, United States
- University of Arizona Sarvor Heart CenterVerified postcodeTucson, United States
- University of California Irvine Medical CenterVerified postcodeIrvine, United States
- University of California San DiegoVerified postcodeLa Jolla, United States
- University of California San FranciscoVerified postcodeSan Francisco, United States
- Baycare Medical GroupVerified postcodeClearwater, United States
- University of MiamiVerified postcodeCoral Gables, United States
- University of FloridaVerified postcodeGainesville, United States
- Augusta UniversityVerified postcodeAugusta, United States
- Loyola Medicine Burr RidgeVerified postcodeOakbrook Terrace, United States
- University of IowaVerified postcodeIowa City, United States
- University of Kansas Medical Center (KUMC)Verified postcodeKansas City, United States
Common questions
What is non-ischemic heart failure?
It's a type of heart failure where your heart muscle is weakened, but not because of blocked arteries or problems with its blood supply.
What does a 'placebo' mean?
A placebo is a fake treatment, looking exactly like the real one but without any active medicine in it. It helps researchers accurately see the effects of the active drug.
What is 'NYHA Class III' heart failure?
This means you have noticeably limited physical activity due to heart failure symptoms like tiredness or shortness of breath, but you don't have symptoms when resting.
Will I know if I'm getting the real medicine or the placebo?
No, neither you nor your doctors will know until the entire study has finished. This is called a 'double-blinded' study and helps the research be as fair as possible.
How long will I be involved in the study?
You'll be monitored for about one year after receiving the treatment, followed by another four years of long-term check-ups, making your total participation about five years.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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