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

NCT05598333 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ASK-CHF2-CS201
• Study Type: interventional
• Target: 150
• Locations: 12 countries
• Sites: 96

Brief Summary

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

Conditions

Congestive Heart Failure

Outcome Measures

Primary Outcomes (4)

• Cardiovascular Related Death

  52 weeks

• NYHA Classification

  NYHA Classification Change from baseline

  52 weeks

• Left Ventricular Ejection Fraction

  LVEF change from baseline

  52 weeks

• Six Minute Walk Test

  6MWT distance change from baseline

  52 weeks

Secondary Outcomes (1)

• Heart Failure Related Hospitalizations

  52 weeks

Study Arms (3)

Treatment Group 1 AB-1002

EXPERIMENTAL

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

Drug: AB-1002

Treatment Group 2 AB-1002

EXPERIMENTAL

Randomized in 1:1:1 into one of three groups. Group 2: 1.43E14vg/subject (n=30-50)

Drug: AB-1002

Treatment Group 3

PLACEBO COMPARATOR

Randomized in 1:1:1 into one of three groups. Group 3: Placebo (n=30-50)

Drug: AB-1002

Interventions

AB-1002 DRUG

Intracoronary Infusion of AB-1002 or placebo

Also known as: Gene Therapy, NAN-101

Treatment Group 1 AB-1002; Treatment Group 2 AB-1002; Treatment Group 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject must be age ≥18 years of age, at the time of signing the informed consent.
• Chronic non-ischemic cardiomyopathy
• % ≤ LVEF ≤ 35% by transthoracic echocardiography (TTE) at screening
• MWT \>50 meters
• Medically stable, NYHA Class III HF for a minimum of 4 weeks while on appropriate medical therapy (defined below) including, but not limited to:
• 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
• 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.
• 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

You may not qualify if:

• Subjects are excluded from the study if any of the following criteria apply:
• Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease
• Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous cardiac assist device therapy within 30 days prior to enrollment
• Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
• Cardiac surgery or percutaneous coronary intervention (PCI) within 30 days prior to screening
• Uncorrected Third degree heart block
• 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
• Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), surgically implanted LVAD or cardiac shunt
• 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.
• 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.
• Expected survival \< 1 year in the judgment of the investigator
• Active or suspected infection within 48 hours prior to intra-coronary infusion as evidenced by fever or positive culture
• 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).
• Liver function tests (alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], alkaline phosphatase) \> 2x upper limit of normal (ULN) within 30 days prior to enrollment.
• Chronic Kidney Disease Stage 5, dialysis dependent or eGFR\<15 within 30 days prior to enrollment

Sponsors & Collaborators

• AskBio Inc: lead

Study Sites (96)

Cardiology P.C. Birmingham

Birmingham, Alabama, 35211, United States

Location

University of Arizona Sarvor Heart Center

Tucson, Arizona, 85724, United States

Location

University of California Irvine Medical Center

Irvine, California, 92697, United States

Location

University of California San Diego

La Jolla, California, 92093, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Baycare Medical Group

Clearwater, Florida, 33756, United States

Location

University of Miami

Coral Gables, Florida, 33146, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Loyola Medicine Burr Ridge

Oakbrook Terrace, Illinois, 60181, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, 66160, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Minneapolis Heart Institute Foundation

Minneapolis, Minnesota, 55407, United States

Location

Mayo Clinic - Minnesota

Rochester, Minnesota, 55905, United States

Location

Aurora Saint Luke's Health System

Kansas City, Missouri, 64111, United States

Location

St. Louis University

St Louis, Missouri, 63117, United States

Location

Renown Health

Reno, Nevada, 89502, United States

Location

Morristown Medical Center - Cardiology

Morristown, New Jersey, 07962, United States

Location

Mt. Sinai New York

New York, New York, 10029, United States

Location

Stony Brook

Stony Brook, New York, 11794, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

The Christ Hospital / The Linder Center for Research

Cincinnati, Ohio, 45219, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45219, United States

Location

MetroHealth Medical Center

Cleveland, Ohio, 44109, United States

Location

The Ohio State University

Columbus, Ohio, 43210, United States

Location

LVH Cardiology

Allentown, Pennsylvania, 18103, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Medical University of South Carolina (MUSC) Medical Center

Charleston, South Carolina, 29425, United States

Location

Stern Cardiovascular

Germantown, Tennessee, 38138, United States

Location

Baylor Scott & White Advanced Heart Failure Clinic - Dallas

Dallas, Texas, 75246, United States

Location

Baylor College of Medicine (BCM) - Baylor Heart Clinic

Houston, Texas, 77030, United States

Location

Houston Methodist Debakey Cardiology Associates

Houston, Texas, 77030, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

Medizinische Universität Graz

Graz, 8010, Austria

Location

Kardiologie & Intensivmedizin Campus III

Linz, 4021, Austria

Location

SALK University Hospital

Salzburg, 5020, Austria

Location

NÖ Landesgesundheitsagentur

Sankt Pölten, Austria

Location

UZ Antwerpen

Edegem, 2650, Belgium

Location

Multiprofile Hospital for Active Treatment Sveti Georgi OOD

Pernik, 2300, Bulgaria

Location

Specialized Hospital For Active Cardiology Treatment Medica Kor EAD

Rousse, 7013, Bulgaria

Location

Multiprofile Hospital for Active Treatment "South West Hospital" OOD

Sandanski, 2802, Bulgaria

Location

Multiprofile Hospital for Active Treatment 'National Cardiology Hospital' EAD

Sofia, 1309, Bulgaria

Location

Acibadem City Clinic University Multiprofile Hospital For Active Treatment Tokuda EAD

Sofia, 1407, Bulgaria

Location

University Multiprofile Hospital for Active Treatment 'Sveta Ekaterina' EAD

Sofia, 1431, Bulgaria

Location

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

Sofia, 1606, Bulgaria

Location

University Multiprofile Hospital For Active Treatment Sofiamed OOD

Sofia, 1797, Bulgaria

Location

St. Boniface Hospital

Winnipeg, Manitoba, R2H2A6, Canada

Location

Memorial University Hospital

St. John's, Newfoundland and Labrador, A1B3V6, Canada

Location

London Health Sciences Centre

London, Ontario, N6A5A5, Canada

Location

St. Michael's Hospital

Toronto, Ontario, M5B1W8, Canada

Location

CIUSSS Saguenay Lac-St-Jean

Chicoutimi, Quebec, G7H5H6, Canada

Location

Institut de Cardiologie de Montréal

Montreal, Quebec, H1T1C8, Canada

Location

Centre Hospitalier de l'Université de Montréal - CHUM

Montreal, Quebec, H2X3E4, Canada

Location

Jewish General Hospital

Montreal, Quebec, H3T1E2, Canada

Location

CHU de Québec - Université Laval

Québec, Quebec, G1R2J6, Canada

Location

Institut Universitaire de Cardiologie et de Pneumologie de Québec - IUCPQ

Québec, Quebec, G1V4G5, Canada

Location

Technical University of Munich (TUM)

München, Bavaria, 81675, Germany

Location

Charité Universitaetsmedizin

Berlin, 15353, Germany

Location

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

Freiburg im Breisgau, 79106, Germany

Location

Medizinische Hochschule Hannover (MHH)

Hanover, 30625, Germany

Location

Heidelberg University Hospital

Heidelberg, 69120, Germany

Location

Universitätsklinikum Schleswig-Holstein

Kiel, D-24105, Germany

Location

Semmelweis Egyetem, Varosmajori Sziv- és Ergyogyaszati Klinika

Budapest, 1122, Hungary

Location

Észak-Pesti Centrumkórház - Honvédkórház

Budapest, 1134, Hungary

Location

Budapesti Uzsoki Utcai Korhaz

Budapest, H-1145, Hungary

Location

Somogy Varmegyei Kaposi Mor Oktato Korhaz - Kardiologiai Osztaly

Kaposvár, 7400, Hungary

Location

Pecsi Tudomanyegyetem Klinikai Kozpont, Szivgyogyaszati Klinika

Pécs, 7624, Hungary

Location

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

Szeged, 6725, Hungary

Location

Amsterdam UMC - Locatie AMC (Academisch Medisch Centrum)

Amsterdam, North Holland, 1105 AZ, Netherlands

Location

Radboud University Medical Center

Nijmegen, 9101, Netherlands

Location

Erasmus Medisch Centrum

Rotterdam, 3015CE, Netherlands

Location

Universitair Medisch Centrum (UMC) Utrecht

Utrecht, 3508GA, Netherlands

Location

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

Chrzanów, 32-500, Poland

Location

1 Wojskowy Szpital Kliniczny z Poliklinika SPZOZ w Lublinie

Lublin, 20-049, Poland

Location

Uniwersytecki Szpital Kliniczny (USK) Nr 4 w Lublinie

Lublin, 20-090, Poland

Location

Spitalul Clinic Judetean De Urgenta Targu Mures

Târgu Mureş, County Mures, Romania

Location

Spitalul Clinic Judetean De Urgenta Brasov- Sectia Cardiologie 3

Brasov, Romania

Location

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

Bucharest, Romania

Location

Spitalul Clinic Judetean De Urgenta Craiova- Sectia de Cardiologie

Craiova, Romania

Location

Spitalul Universitar de Urgenta Bucuresti- Sectia Cardiologie 2

Târgu Mureş, Romania

Location

Institutul De Boli Cardiovasculare Timisoara- Clinica de Cardiologie

Timișoara, Romania

Location

Hospital Universitario de Bellvitge

Barcelona, Catalonia, 8908, Spain

Location

Universidad de Navarra - Clinica Universidad de Navarra (CUN)

Pamplona, Navarre, 31008, Spain

Location

Hospital Ramon y Cajal | Cardiology - Research Unit

Madrid, 28034, Spain

Location

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

Madrid, 28046, Spain

Location

Complejo Hospitalario Universitario Santiago de Compostela (CHUS)

Santiago de Compostela, 15706, Spain

Location

Hospital Clinico Universitario de Valencia (CHUV)

Valencia, 46010, Spain

Location

Manchester Royal Infirmary

Manchester, Manchester, M13 9WL, United Kingdom

Location

Leeds General Infirmary

Leeds, West Yorkshire, LS1 3EX, United Kingdom

Location

University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom

Location

NHS Golden Jubilee

Glasgow, G81 4DY, United Kingdom

Location

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

London, SW3 6LY, United Kingdom

Location

MeSH Terms

Conditions

Heart Failure

Interventions

Genetic Therapy

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics; Genetic Engineering; Genetic Techniques; Investigative Techniques

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Adaptive, double blinded, placebo controlled, randomized

Study Record Dates

• First Submitted: October 25, 2022
• First Posted: October 28, 2022
• Study Start: October 20, 2023
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): December 31, 2030
• Last Updated: December 3, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

PL (3); BE (1); AU (4); HU (6); BU (8); GB (5); DE (6); ES (6); RO (6); CA (10); US (37); NL (4)