Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure

NCT ID: NCT05598333

Last Updated: 2025-12-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-10-20

Study Completion Date

2030-12-31

Brief Summary

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This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age \>18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF.

Subjects will be randomized into one of three treatment groups in a 1:1:1

Detailed Description

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Subjects will be randomized into one of three treatment groups in a 1:1:1 fashion to either 7.15E13vg (n=30-50), 1.43E14 (n=30-50) or placebo group (n=30-50).

Approximately 90 to 150 subjects will be randomly assigned to study intervention Placebo

Study duration until the primary analysis at 52 week will be approximately 37 months including 25 months of recruitment and 52-week Observation Period after dosing. Once all the subjects complete the 52 weeks Observation Period, the treatment groups will be unblinded and primary analysis performed. Study participation duration: The study will last 52 weeks from dosing, with another 4 years of long-term follow-up for a total of 5 years. During the 4 year long-term follow up sites will contact subjects twice a year for two years, then once a year for the remaining two years for safety, efficacy assessments, and survival

Conditions

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Congestive Heart Failure

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Adaptive, double blinded, placebo controlled, randomized
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
The IWRS will be programmed with blind-breaking instructions. In case of an emergency, the investigator has the responsibility for determining if unblinding of a subject's intervention assignment is warranted. If the investigator is unavailable, and a treating physician not associated with the study requests emergency unblinding, the emergency unblinding requests are forwarded to the emergency medical advice 24 hours/7 day service.

Study Groups

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Treatment Group 1 AB-1002

Randomized in 1:1:1 into one of three groups. Group 1: 7.15E13vg/subject (n=30-50)

Group Type EXPERIMENTAL

AB-1002

Intervention Type DRUG

Intracoronary Infusion of AB-1002 or placebo

Treatment Group 2 AB-1002

Randomized in 1:1:1 into one of three groups.

Group 2: 1.43E14vg/subject (n=30-50)

Group Type EXPERIMENTAL

AB-1002

Intervention Type DRUG

Intracoronary Infusion of AB-1002 or placebo

Treatment Group 3

Randomized in 1:1:1 into one of three groups.

Group 3: Placebo (n=30-50)

Group Type PLACEBO_COMPARATOR

AB-1002

Intervention Type DRUG

Intracoronary Infusion of AB-1002 or placebo

Interventions

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AB-1002

Intracoronary Infusion of AB-1002 or placebo

Intervention Type DRUG

Other Intervention Names

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Gene Therapy NAN-101

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AskBio Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Cardiology P.C. Birmingham

Birmingham, Alabama, United States

Site Status

University of Arizona Sarvor Heart Center

Tucson, Arizona, United States

Site Status

University of California Irvine Medical Center

Irvine, California, United States

Site Status

University of California San Diego

La Jolla, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Baycare Medical Group

Clearwater, Florida, United States

Site Status

University of Miami

Coral Gables, Florida, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Augusta University

Augusta, Georgia, United States

Site Status

Loyola Medicine Burr Ridge

Oakbrook Terrace, Illinois, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Minneapolis Heart Institute Foundation

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic - Minnesota

Rochester, Minnesota, United States

Site Status

Aurora Saint Luke's Health System

Kansas City, Missouri, United States

Site Status

St. Louis University

St Louis, Missouri, United States

Site Status

Renown Health

Reno, Nevada, United States

Site Status

Morristown Medical Center - Cardiology

Morristown, New Jersey, United States

Site Status

Mt. Sinai New York

New York, New York, United States

Site Status

Stony Brook

Stony Brook, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

The Christ Hospital / The Linder Center for Research

Cincinnati, Ohio, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

MetroHealth Medical Center

Cleveland, Ohio, United States

Site Status

The Ohio State University

Columbus, Ohio, United States

Site Status

LVH Cardiology

Allentown, Pennsylvania, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina (MUSC) Medical Center

Charleston, South Carolina, United States

Site Status

Stern Cardiovascular

Germantown, Tennessee, United States

Site Status

Baylor Scott & White Advanced Heart Failure Clinic - Dallas

Dallas, Texas, United States

Site Status

Baylor College of Medicine (BCM) - Baylor Heart Clinic

Houston, Texas, United States

Site Status

Houston Methodist Debakey Cardiology Associates

Houston, Texas, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

University of Wisconsin

Madison, Wisconsin, United States

Site Status

Medizinische Universität Graz

Graz, , Austria

Site Status

Kardiologie & Intensivmedizin Campus III

Linz, , Austria

Site Status

SALK University Hospital

Salzburg, , Austria

Site Status

NÖ Landesgesundheitsagentur

Sankt Pölten, , Austria

Site Status

UZ Antwerpen

Edegem, , Belgium

Site Status

Multiprofile Hospital for Active Treatment Sveti Georgi OOD

Pernik, , Bulgaria

Site Status

Specialized Hospital For Active Cardiology Treatment Medica Kor EAD

Rousse, , Bulgaria

Site Status

Multiprofile Hospital for Active Treatment "South West Hospital" OOD

Sandanski, , Bulgaria

Site Status

Multiprofile Hospital for Active Treatment 'National Cardiology Hospital' EAD

Sofia, , Bulgaria

Site Status

Acibadem City Clinic University Multiprofile Hospital For Active Treatment Tokuda EAD

Sofia, , Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment 'Sveta Ekaterina' EAD

Sofia, , Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment and Emergency Medicine N. I. Pirogov EAD

Sofia, , Bulgaria

Site Status

University Multiprofile Hospital For Active Treatment Sofiamed OOD

Sofia, , Bulgaria

Site Status

St. Boniface Hospital

Winnipeg, Manitoba, Canada

Site Status

Memorial University Hospital

St. John's, Newfoundland and Labrador, Canada

Site Status

London Health Sciences Centre

London, Ontario, Canada

Site Status

St. Michael's Hospital

Toronto, Ontario, Canada

Site Status

CIUSSS Saguenay Lac-St-Jean

Chicoutimi, Quebec, Canada

Site Status

Institut de Cardiologie de Montréal

Montreal, Quebec, Canada

Site Status

Centre Hospitalier de l'Université de Montréal - CHUM

Montreal, Quebec, Canada

Site Status

Jewish General Hospital

Montreal, Quebec, Canada

Site Status

CHU de Québec - Université Laval

Québec, Quebec, Canada

Site Status

Institut Universitaire de Cardiologie et de Pneumologie de Québec - IUCPQ

Québec, Quebec, Canada

Site Status

Technical University of Munich (TUM)

München, Bavaria, Germany

Site Status

Charité Universitaetsmedizin

Berlin, , Germany

Site Status

Universitaetsklinikum Freiburg | Herz-Zentrum Bad Krozingen (Heart Centre Bad Krozingen)

Freiburg im Breisgau, , Germany

Site Status

Medizinische Hochschule Hannover (MHH)

Hanover, , Germany

Site Status

Heidelberg University Hospital

Heidelberg, , Germany

Site Status

Universitätsklinikum Schleswig-Holstein

Kiel, , Germany

Site Status

Semmelweis Egyetem, Varosmajori Sziv- és Ergyogyaszati Klinika

Budapest, , Hungary

Site Status

Észak-Pesti Centrumkórház - Honvédkórház

Budapest, , Hungary

Site Status

Budapesti Uzsoki Utcai Korhaz

Budapest, , Hungary

Site Status

Somogy Varmegyei Kaposi Mor Oktato Korhaz - Kardiologiai Osztaly

Kaposvár, , Hungary

Site Status

Pecsi Tudomanyegyetem Klinikai Kozpont, Szivgyogyaszati Klinika

Pécs, , Hungary

Site Status

Szegedi Tudományegyetem, Szent-Györgyi Albert Klinikai Központ - Belgyógyászati Klinika

Szeged, , Hungary

Site Status

Amsterdam UMC - Locatie AMC (Academisch Medisch Centrum)

Amsterdam, North Holland, Netherlands

Site Status

Radboud University Medical Center

Nijmegen, , Netherlands

Site Status

Erasmus Medisch Centrum

Rotterdam, , Netherlands

Site Status

Universitair Medisch Centrum (UMC) Utrecht

Utrecht, , Netherlands

Site Status

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

Chrzanów, , Poland

Site Status

1 Wojskowy Szpital Kliniczny z Poliklinika SPZOZ w Lublinie

Lublin, , Poland

Site Status

Uniwersytecki Szpital Kliniczny (USK) Nr 4 w Lublinie

Lublin, , Poland

Site Status

Spitalul Clinic Judetean De Urgenta Targu Mures

Târgu Mureş, County Mures, Romania

Site Status

Spitalul Clinic Judetean De Urgenta Brasov- Sectia Cardiologie 3

Brasov, , Romania

Site Status

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

Bucharest, , Romania

Site Status

Spitalul Clinic Judetean De Urgenta Craiova- Sectia de Cardiologie

Craiova, , Romania

Site Status

Spitalul Universitar de Urgenta Bucuresti- Sectia Cardiologie 2

Târgu Mureş, , Romania

Site Status

Institutul De Boli Cardiovasculare Timisoara- Clinica de Cardiologie

Timișoara, , Romania

Site Status

Hospital Universitario de Bellvitge

Barcelona, Catalonia, Spain

Site Status

Universidad de Navarra - Clinica Universidad de Navarra (CUN)

Pamplona, Navarre, Spain

Site Status

Hospital Ramon y Cajal | Cardiology - Research Unit

Madrid, , Spain

Site Status

Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz (HULP)

Madrid, , Spain

Site Status

Complejo Hospitalario Universitario Santiago de Compostela (CHUS)

Santiago de Compostela, , Spain

Site Status

Hospital Clinico Universitario de Valencia (CHUV)

Valencia, , Spain

Site Status

Manchester Royal Infirmary

Manchester, Manchester, United Kingdom

Site Status

Leeds General Infirmary

Leeds, West Yorkshire, United Kingdom

Site Status

University Hospital of Wales

Cardiff, , United Kingdom

Site Status

NHS Golden Jubilee

Glasgow, , United Kingdom

Site Status

Imperial College London - National Heart & Lung Institute (NHLI) - Royal Brompton Campus

London, , United Kingdom

Site Status

Countries

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United States Austria Belgium Bulgaria Canada Germany Hungary Netherlands Poland Romania Spain United Kingdom

Other Identifiers

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ASK-CHF2-CS201

Identifier Type: -

Identifier Source: org_study_id

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