Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

NCT04988386 | Active Not Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: AG10-3042020-005643-22
• Study Type: interventional
• Target: 389
• Locations: 17 countries
• Sites: 72

Brief Summary

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Conditions

Amyloid Cardiomyopathy, Transthyretin-Related

Keywords

ATTR-CM

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent Adverse Events [Safety and Tolerability]

  Incidence of each treatment-emergent adverse events measured over 60 Months or study completion

  60 months

Secondary Outcomes (4)

• Evaluate all-cause mortality and cardiovascular mortality

  60 months

• Evaluate the effect of acoramidis on the 6-minute walk test (6MWT)

  60 months

• Evaluate the effect of acoramidis on health-related quality of life Kansas City Cardiomyopathy Questionnaire

  60 months

• Evaluate the effect of acoramidis on the frequency of CV-related hospitalization

  60 months

Study Arms (1)

AG10

EXPERIMENTAL

Open-label study all participants will receive AG10 during this study.

Drug: Acoramidis (AG10)

Interventions

Acoramidis (AG10) DRUG

Acoramidis (AG10) twice daily

Also known as: AG10, ALXN 2060

AG10

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Completed 30 months of the blinded study treatment in Study AG10-301 and the Study AG10-301 Month 30 visit including assessments and procedures.
• Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures.
• Female participants of childbearing potential who engage in heterosexual intercourse must agree to use a highly effective method of contraception beginning with enrollment and continuing for 30 days after the last dose of acoramidis. Female participants using oral contraceptives must agree to use an additional birth control method. While not considered highly effective, a double-barrier method is acceptable. A male participant who is sexually active with a female of childbearing potential and has not had a vasectomy must agree to use a double-barrier method of birth control.

You may not qualify if:

• Acute myocardial infarction, acute coronary syndrome or coronary revascularization within 90 days prior to Day 1 stroke or transient ischemic attack (TIA) within 90 days prior to Day 1.
• Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study.
• Has had a heart and/or liver transplant or is on the heart transplantation list within the year prior to Day 1
• Has had implantation of a cardiac mechanical assist device (CMAD) or is scheduled for implantation of a CMAD
• Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301.
• Has estimated glomerular filtration rate (eGFR) by modification of diet for renal disease (MDRD) formula \< 15 mL/min/1.73 m2 at Month 27 of Study AG10-301 or at any subsequent central lab value prior to Day 1.
• Known hypersensitivity to acoramidis, its metabolites, or formulation excipients.
• At the end of Study AG10-301 or at Day 1 of Study AG10-304 (or any time during the study), participant is on prohibited medication.
• Females who are pregnant or breastfeeding. A negative urine pregnancy test at the Day 1 visit and at each study visit are required for female participants of childbearing potential.
• In the judgment of the Investigator or Medical Monitor, has any clinically important ongoing medical condition or laboratory abnormality or condition that might jeopardize the participant's safety, increase their risk from participation, or interfere with the study.
• Participation in another interventional clinical trial (with the exception of Study AG10-301) within 30 days prior to dosing. Participation in observational and/or registry studies should be discussed with the Medical Monitor.
• Has any condition that in the opinion of the Investigator or Medical Monitor would preclude compliance with the study protocol such as a history of substance abuse, alcoholism, or a psychiatric condition.

Sponsors & Collaborators

• Eidos Therapeutics, a BridgeBio company: lead

Study Sites (72)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

University of Colorado Hospital - Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

NorthShore University Health System

Evanston, Illinois, 60201, United States

Location

MedStar Medical Group Cardiology at Franklin Square

Baltimore, Maryland, 21237, United States

Location

Boston University Medical Center General Clinical Research Unit

Boston, Massachusetts, 02118, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Saint Luke's Hospital of Kansas City

Kansas City, Missouri, 64111, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

New York University Langone Health

New York, New York, 10016, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Laurelton Heart Specialists

Rosedale, New York, 11422, United States

Location

Montefiore Medical Center at Hutchinson

The Bronx, New York, 10461, United States

Location

Duke University Health System - Duke Clinic

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic Main Campus

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, 15213, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Virginia Commonwealth University Health System

Richmond, Virginia, 23219, United States

Location

Roanoke Heart Institute

Roanoke, Virginia, 24014, United States

Location

University of Washington School of Medicine

Seattle, Washington, 98195, United States

Location

Providence Sacred Heart Children's Hospital

Spokane, Washington, 99204, United States

Location

Saint Vincent's Hospital Sydney

Darlinghurst, New South Wales, 2010, Australia

Location

Princess Alexandra Hospital

Woolloongabba, Queensland, 4102, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

Royal Hobart Hospital

Hobart, Tasmania, 7000, Australia

Location

Box Hill Hospital

Box Hill, Victoria, 3128, Australia

Location

Fiona Stanley Hospital

Murdoch, Western Australia, 6150, Australia

Location

Ziekenhuis Oost-Limburg

Genk, Limburg, 3600, Belgium

Location

Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan

Bruges, West Vlaanderen, 8000, Belgium

Location

Instituto de Cardiologia do Rio Grande do Sul

Porto Alegre, Rio Grande do Sul, 90620-001, Brazil

Location

Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED

Ribeirão Preto, São Paulo, 14026-900, Brazil

Location

Instituto do Coração de São Paulo

São Paulo, São Paulo, 05403-000, Brazil

Location

Vancouver General Hospital

Vancouver, British Columbia, V5Z 1M9, Canada

Location

Saint Boniface Hospital

Winnipeg, Manitoba, R2H 2A6, Canada

Location

Queen Elizabeth II Health Sciences Centre - Halifax Infirmary

Halifax, Nova Scotia, B3H 3A7, Canada

Location

Toronto Heart Centre

Toronto, Ontario, M4P IE4, Canada

Location

L' Institut de Cardiologie de Montréal

Montreal, Quebec, H1T 1C8, Canada

Location

Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CHUM)

Montreal, Quebec, H2X 0A9, Canada

Location

Hôpital du Sacré-Coeur de Montréal

Montreal, Quebec, H4J 1C5, Canada

Location

Hôpital régional de Rimouski

Rimouski, Quebec, G5L 5T1, Canada

Location

Vseobecna Fakultni Nemocnice v Praze

Prague, Prague, 128 08, Czechia

Location

Institut Klinické a Experimentální Medicíny

Prague, Prague, 140 21, Czechia

Location

Fakultní Nemocnice u sv. Anny v Brně

Brno, South Moravian, 656 91, Czechia

Location

Aarhus Universitets Hospital

Aarhus N, Palle Juul-Jensens, 8200, Denmark

Location

Alexandra General Hospital of Athens

Athens, Attica, 11528, Greece

Location

Mater Misericordiae University Hospital

Dublin, Dublin, 7, Ireland

Location

The Chaim Sheba Medical Center

Ramat Gan, Tel Aviv, 5265601, Israel

Location

Hadassah University Hospital Ein Kerem

Jerusalem, 9112001, Israel

Location

Ospedale San Donato

Arezzo, Arezzo, 52100, Italy

Location

Azienda Ospedaliero - Universitaria Careggi

Florence, Florence, 50134, Italy

Location

Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica

Pisa, Pisa, 56124, Italy

Location

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

Location

Universitair Medisch Centrum Groningen

Groningen, Provincie Groningen, 9713 GZ, Netherlands

Location

Universitair Medisch Centrum Utrecht

Utrecht, Utrecht, 3584 CX, Netherlands

Location

Middlemore Clinical Trials - Middlemore Hospital

Papatoetoe, Auckland, 2025, New Zealand

Location

Waikato Hospital

Hamilton, Waikato Region, 3240, New Zealand

Location

Centro Hospitalar Universitário Lisboa Norte EPE- Hospital Santa Maria

Lisbon, Lisbon District, 1649-035, Portugal

Location

Seoul National University Bundang Hospital

Seongnam-si, Gyeonggi-do, 13620, South Korea

Location

Seoul National University Hospital

Seoul, Gyeonggi-do, 3080, South Korea

Location

Hospital Son Llàtzer

Palma de Mallorca, Balearic Islands, 7198, Spain

Location

Complejo Hospitalario Universitario de Santiago (CHUS)

Santiago de Compostela, La Coruña, 15706, Spain

Location

Clinica Universidad de Navarra Madrid

Madrid, Madrid, 28027, Spain

Location

Hospital Universitario Puerta de Hierro - Majadahonda

Majadahonda, Madrid, 28222, Spain

Location

Clínica Universidad de Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital Clínico Universitario de Valencia

Valencia, Valencia, 46010, Spain

Location

National Amyloidosis Centre, Division of Medicine - Royal Free

London, England, NW3 2PF, United Kingdom

Location

Richmond Pharmacology Ltd

London, England, SE1 1YR, United Kingdom

Location

Related Publications (2)

• Alexander KM, Davis MK, Akinboboye O, Berk J, Bhatt K, Cappelli F, Cuddy SAM, Fontana M, Garcia-Pavia P, Gillmore JD, Griffin JM, Grodin JL, Judge DP, Khouri MG, Lam K, Masri A, Maurer MS, Obici L, Ruberg FL, Sarswat N, Shah K, Soman P, Stern L, Wright R, Xiong K, Cao X, Lystig T, Tamby JF, Castano A, Katz L, Sinha U, Fox JC, Solomon SD, Grogan M. Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension. JAMA Cardiol. 2025 Nov 8:e254477. doi: 10.1001/jamacardio.2025.4477. Online ahead of print.

  PMID: 41205147 DERIVED

• Judge DP, Gillmore JD, Alexander KM, Ambardekar AV, Cappelli F, Fontana M, Garcia-Pavia P, Grodin JL, Grogan M, Hanna M, Masri A, Nativi-Nicolau J, Obici L, Hvitfeldt Poulsen S, Sarswat N, Shah K, Soman P, Lystig T, Cao X, Wang K, Pecoraro ML, Tamby JF, Katz L, Sinha U, Fox JC, Maurer MS. Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial. Circulation. 2025 Mar 4;151(9):601-611. doi: 10.1161/CIRCULATIONAHA.124.072771. Epub 2024 Nov 18.

  PMID: 39556242 DERIVED

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

attruby

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label study extension study from the AG10-301 double-blind study.

Study Record Dates

• First Submitted: July 13, 2021
• First Posted: August 3, 2021
• Study Start: October 27, 2021
• Primary Completion (Estimated): April 15, 2028
• Study Completion (Estimated): May 15, 2028
• Last Updated: October 14, 2025

Record last verified: 2025-10

Locations

IE (1); IL (2); BE (2); DK (1); PT (1); IT (4); CA (8); GB (2); GR (1); CZ (3); NL (2); KR (2); AU (6); US (26); NZ (2); ES (6); BR (3)