Gene therapy injection aims to repair failing hearts

NCT ID NCT05598333

Disease control Sponsor: AskBio Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy called AB-1002, given directly into the heart arteries, for people with advanced heart failure (NYHA Class III). The goal is to see if it can improve heart function, reduce symptoms, and lower the risk of death or hospitalization. About 150 adults with non-ischemic cardiomyopathy will receive either the gene therapy or a placebo, and researchers will track their outcomes over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: AB-1002 (gene therapy)
What this could lead to: If successful, this gene therapy could improve heart function and reduce symptoms in people with advanced heart failure, potentially decreasing the need for hospitalizations.
What could go wrong: This is an early Phase 2 trial with only 150 participants, so results may not apply to all patients. Gene therapies can have unpredictable side effects, and the treatment requires a heart catheterization procedure, which carries its own risks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 150 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2023

Expected to finish

Dec 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subject must be age ≥18 years of age, at the time of signing the informed consent. 2. Chronic non-ischemic cardiomyopathy 3. 15% ≤ LVEF ≤ 35% by transthoracic echocardiography (TTE) at screening 4. 6MWT \>50 meters 5. Medically stable, NYHA Class III HF for a minimum of 4 weeks while on appropriate medical therapy (defined below) including, but not limited to: 1. Beta blocker therapy and ACE inhibitor or angiotensin receptor blocker (ARB) or sacubitril/valsartan combination therapy (Entresto) for ≥ 90 days prior to enrollment. May also receive aldosterone antagonist therapy. Doses of the above medications must be stable for ≥ 30 days prior to enrollment; and 2. Cardiac resynchronization therapy (Zareba et al 2011), if clinically indicated, must have been implanted ≥ 90 days prior to enrollment. Internal cardioverter defibrillator (ICD) must be implanted, if clinically indicated ≥ 30 days prior to enrollment. 6. Women of childbearing potential must use at least one of the following acceptable birth control methods throughout the study and for 6 months after IP administration: * Surgically sterile (bilateral tubal ligation, hysterectomy, bilateral oophorectomy) 6 months minimum prior to IP administration * Intrauterine device in place for at least 90 days prior to receiving IP * Barrier methods (diaphragm plus spermicide or condom) starting at least 30 days prior to receiving IP * Abstinence (the subject must be willing to remain abstinent from screening to 6 months after receiving IP). Females are allowed to claim abstinence as their method of contraception only when it is the preferred and usual lifestyle of the subject * Surgical sterilization of the partner(s) (vasectomy) for \>180 days prior to IP administration * Hormonal contraceptives starting \> 90 days prior to IP. If hormonal contraceptives are started less than 90 days prior to receiving IP, subjects must agree to use a barrier method (diaphragm plus spermicide or condom) from screening through 90 days after initiation of hormonal contraceptives 7. Males subjects capable of fathering a child: * Must agree to use a condom and should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak when having sexual intercourse with a woman of childbearing potential who is not currently pregnant from IP administration through 6 months after the time of IP administration * Must agree not to donate sperm for 6 months after time of receiving IP * Documented evidence of vasectomy in males for 180 days minimum prior to receiving IP is an acceptable form of contraception * Males who claim abstinence as their method of contraception are allowed, provided they agree to use barrier methods should they become sexually active from screening through 6 months after receiving IP. Males are allowed to claim abstinence as their method of contraception only when it is the preferred and usual lifestyle of the subject 8. Appropriate candidate for protocol-specified intracoronary infusion in the judgment of the infusing interventional cardiologist Exclusion Criteria: Subjects are excluded from the study if any of the following criteria apply: 9. Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease 10. Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous cardiac assist device therapy within 30 days prior to enrollment 11. Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm 12. Cardiac surgery or percutaneous coronary intervention (PCI) within 30 days prior to screening 13. Uncorrected Third degree heart block 14. Clinically significant myocardial infarction (MI) in the judgment of the subject's physician (e.g., ST elevation MI \[STEMI\] or large non-STEMI) within 6 months prior to enrollment 15. Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), surgically implanted LVAD or cardiac shunt 16. Likely to receive cardiac resynchronization therapy, cardiomyoplasty, LV reduction surgery, heart transplant, conventional revascularization procedure, or valvular repair within 3 months of IP dosing in judgement of investigator. 17. Known hypersensitivity to contrast dyes (not easily controlled by antihistamines) used for angiography; history of, or likely need for, high-dose steroid pretreatment prior to contrast angiography. 18. Expected survival \< 1 year in the judgment of the investigator 19. Active or suspected infection within 48 hours prior to intra-coronary infusion as evidenced by fever or positive culture 20. Known intrinsic liver disease (e.g., cirrhosis, hepatitis A, chronic hepatitis B or hepatitis C virus infection). If serology is positive and PCR is known to be negative, subject may be eligible (confirm with medical monitor). 21. Liver function tests (alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], alkaline phosphatase) \> 2x upper limit of normal (ULN) within 30 days prior to enrollment. 22. Chronic Kidney Disease Stage 5, dialysis dependent or eGFR\<15 within 30 days prior to enrollment 23. Bleeding diathesis or thrombocytopenia defined as platelets \<50,000 platelets/μL within 30 days prior to enrollment 24. Anemia defined as hemoglobin \<10 g/dL or transfusion dependent within 30 days prior to enrollment 25. Neutropenia defined as absolute neutrophils \<1500 mm3 within 30 days prior to enrollment 26. Known AIDS or HIV-positive status, or a previous diagnosis of immunodeficiency with an absolute neutrophil count \<1000 cells/mm3 27. Previous participation in a study of gene transfer 28. Receiving investigational intervention or participating in another clinical study within 30 days of another investigational drug administration prior to administration of AB-1002 that may impact the therapeutic potential of AB-1002. 29. Pregnancy or breastfeeding or plans to become pregnant within the next 12 months at the time of screening 30. Subjects with any other condition which in the opinion of the investigator would preclude participation in the study (including risk for non-compliance and any intercurrent conditions that pose an undue medical hazard, or which could interfere with the interpretation of the study results) 31. Malignant neoplasm within 5 years of dosing, with the exception of those with negligible risk of metastasis or death (such as adequately treated carcinoma in situs of the cervix, basal or squamous cell skin cancer, localized prostate cancer or ductal carcinoma in situ) 32. Any documented history of non-compliance with medications, illicit drug use or laboratory evidence of illicit drug use during screen period

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Conditions

The condition(s) this trial relates to.

congestive heart failure heart failure intrinsic cardiomyopathy moderate heart failure

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congestive heart failure

Contacts and locations

Locations

1 Wojskowy Szpital Kliniczny z Poliklinika SPZOZ w Lublinie

Lublin, 20-049, Poland
Acibadem City Clinic University Multiprofile Hospital For Active Treatment Tokuda EAD

Sofia, 1407, Bulgaria
American Heart of Poland S.A. - MCSN, PAKS w Chrzanowie

Chrzanów, 32-500, Poland
Amsterdam UMC - Locatie AMC (Academisch Medisch Centrum)

Amsterdam, North Holland, 1105 AZ, Netherlands
Augusta University

Augusta, Georgia, 30912, United States
Aurora Saint Luke's Health System

Kansas City, Missouri, 64111, United States
Baycare Medical Group

Clearwater, Florida, 33756, United States
Baylor College of Medicine (BCM) - Baylor Heart Clinic

Houston, Texas, 77030, United States
Baylor Scott & White Advanced Heart Failure Clinic - Dallas

Dallas, Texas, 75246, United States
Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States
Budapesti Uzsoki Utcai Korhaz

Budapest, H-1145, Hungary
CHU de Québec - Université Laval

Québec, Quebec, G1R2J6, Canada
CIUSSS Saguenay Lac-St-Jean

Chicoutimi, Quebec, G7H5H6, Canada
Cardiology P.C. Birmingham

Birmingham, Alabama, 35211, United States
Centre Hospitalier de l'Université de Montréal - CHUM

Montreal, Quebec, H2X3E4, Canada
Charité Universitaetsmedizin

Berlin, 15353, Germany
Complejo Hospitalario Universitario Santiago de Compostela (CHUS)

Santiago de Compostela, 15706, Spain
Duke University

Durham, North Carolina, 27705, United States
Erasmus Medisch Centrum

Rotterdam, 3015CE, Netherlands
Heidelberg University Hospital

Heidelberg, 69120, Germany
Hospital Clinico Universitario de Valencia (CHUV)

Valencia, 46010, Spain
Hospital Ramon y Cajal | Cardiology - Research Unit

Madrid, 28034, Spain
Hospital Universitario de Bellvitge

Barcelona, Catalonia, 8908, Spain
Houston Methodist Debakey Cardiology Associates

Houston, Texas, 77030, United States
Imperial College London - National Heart & Lung Institute (NHLI) - Royal Brompton Campus

London, SW3 6LY, United Kingdom
Institut Universitaire de Cardiologie et de Pneumologie de Québec - IUCPQ

Québec, Quebec, G1V4G5, Canada
Institut de Cardiologie de Montréal

Montreal, Quebec, H1T1C8, Canada
Institutul De Boli Cardiovasculare Timisoara- Clinica de Cardiologie

Timișoara, Romania
Jewish General Hospital

Montreal, Quebec, H3T1E2, Canada
Kardiologie & Intensivmedizin Campus III

Linz, 4021, Austria
LVH Cardiology

Allentown, Pennsylvania, 18103, United States
Leeds General Infirmary

Leeds, West Yorkshire, LS1 3EX, United Kingdom
London Health Sciences Centre

London, Ontario, N6A5A5, Canada
Loyola Medicine Burr Ridge

Oakbrook Terrace, Illinois, 60181, United States
Manchester Royal Infirmary

Manchester, Manchester, M13 9WL, United Kingdom
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Mayo Clinic - Minnesota

Rochester, Minnesota, 55905, United States
Medical University of South Carolina (MUSC) Medical Center

Charleston, South Carolina, 29425, United States
Medizinische Hochschule Hannover (MHH)

Hanover, 30625, Germany
Medizinische Universität Graz

Graz, 8010, Austria
Memorial University Hospital

St. John's, Newfoundland and Labrador, A1B3V6, Canada
MetroHealth Medical Center

Cleveland, Ohio, 44109, United States
Minneapolis Heart Institute Foundation

Minneapolis, Minnesota, 55407, United States
Montefiore Medical Center

The Bronx, New York, 10461, United States
Morristown Medical Center - Cardiology

Morristown, New Jersey, 07962, United States
Mt. Sinai New York

New York, New York, 10029, United States
Multiprofile Hospital for Active Treatment "South West Hospital" OOD

Sandanski, 2802, Bulgaria
Multiprofile Hospital for Active Treatment 'National Cardiology Hospital' EAD

Sofia, 1309, Bulgaria
Multiprofile Hospital for Active Treatment Sveti Georgi OOD

Pernik, 2300, Bulgaria
NHS Golden Jubilee

Glasgow, G81 4DY, United Kingdom
NÖ Landesgesundheitsagentur

Sankt Pölten, Austria
Pecsi Tudomanyegyetem Klinikai Kozpont, Szivgyogyaszati Klinika

Pécs, 7624, Hungary
Radboud University Medical Center

Nijmegen, 9101, Netherlands
Renown Health

Reno, Nevada, 89502, United States
SALK University Hospital

Salzburg, 5020, Austria
Semmelweis Egyetem, Varosmajori Sziv- és Ergyogyaszati Klinika

Budapest, 1122, Hungary
Somogy Varmegyei Kaposi Mor Oktato Korhaz - Kardiologiai Osztaly

Kaposvár, 7400, Hungary
Specialized Hospital For Active Cardiology Treatment Medica Kor EAD

Rousse, 7013, Bulgaria
Spitalul Clinic Judetean De Urgenta Brasov- Sectia Cardiologie 3

Brasov, Romania
Spitalul Clinic Judetean De Urgenta Craiova- Sectia de Cardiologie

Craiova, Romania
Spitalul Clinic Judetean De Urgenta Targu Mures

Târgu Mureş, County Mures, Romania
Spitalul Universitar de Urgenta Bucuresti- Sectia Cardiologie 2

Târgu Mureş, Romania
Spitalul Universitar de Urgenta Militar Central "Dr. Carol Davila", Sectia Clinica Cardiologie I

Bucharest, Romania
St. Boniface Hospital

Winnipeg, Manitoba, R2H2A6, Canada
St. Louis University

St Louis, Missouri, 63117, United States
St. Michael's Hospital

Toronto, Ontario, M5B1W8, Canada
Stern Cardiovascular

Germantown, Tennessee, 38138, United States
Stony Brook

Stony Brook, New York, 11794, United States
Szegedi Tudományegyetem, Szent-Györgyi Albert Klinikai Központ - Belgyógyászati Klinika

Szeged, 6725, Hungary
Technical University of Munich (TUM)

München, Bavaria, 81675, Germany
The Christ Hospital / The Linder Center for Research

Cincinnati, Ohio, 45219, United States
The Ohio State University

Columbus, Ohio, 43210, United States
Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States
UZ Antwerpen

Edegem, 2650, Belgium
Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz (HULP)

Madrid, 28046, Spain
Universidad de Navarra - Clinica Universidad de Navarra (CUN)

Pamplona, Navarre, 31008, Spain
Universitaetsklinikum Freiburg | Herz-Zentrum Bad Krozingen (Heart Centre Bad Krozingen)

Freiburg im Breisgau, 79106, Germany
Universitair Medisch Centrum (UMC) Utrecht

Utrecht, 3508GA, Netherlands
University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom
University Multiprofile Hospital For Active Treatment Sofiamed OOD

Sofia, 1797, Bulgaria
University Multiprofile Hospital for Active Treatment 'Sveta Ekaterina' EAD

Sofia, 1431, Bulgaria
University Multiprofile Hospital for Active Treatment and Emergency Medicine N. I. Pirogov EAD

Sofia, 1606, Bulgaria
University of Arizona Sarvor Heart Center

Tucson, Arizona, 85724, United States
University of California Irvine Medical Center

Irvine, California, 92697, United States
University of California San Diego

La Jolla, California, 92093, United States
University of California San Francisco

San Francisco, California, 94143, United States
University of Cincinnati

Cincinnati, Ohio, 45219, United States
University of Florida

Gainesville, Florida, 32610, United States
University of Iowa

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center (KUMC)

Kansas City, Kansas, 66160, United States
University of Miami

Coral Gables, Florida, 33146, United States
University of Washington

Seattle, Washington, 98195, United States
University of Wisconsin

Madison, Wisconsin, 53792, United States
Universitätsklinikum Schleswig-Holstein

Kiel, D-24105, Germany
Uniwersytecki Szpital Kliniczny (USK) Nr 4 w Lublinie

Lublin, 20-090, Poland
Észak-Pesti Centrumkórház - Honvédkórház

Budapest, 1134, Hungary

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