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Amyloid Cardiomyopathy, Transthyretin-RelatedPhase 3AG10NCT04988386

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

A Phase 3 study testing Acoramidis (AG10) in people with Amyloid Cardiomyopathy, Transthyretin-Related, run by Eidos Therapeutics, a BridgeBio company at 72 sites in 17 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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) Read more about this study

Who is it for?
Ages 18 to 90 years
What is being tested?
Acoramidis (AG10)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
389 participants
Where
72 sitesin 17 countries
Study dates
27 Oct 2021to 15 May 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 90 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 12Show fewer
  • 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.

Trial locations and contacts

72 sites in 17 countries.

72 sites in 17 countries

United States26 sites

  • University of Colorado Hospital - Anschutz Medical Campus

    Aurora, Colorado, 80045

  • MedStar Medical Group Cardiology at Franklin Square

    Baltimore, Maryland, 21237

  • Boston University Medical Center General Clinical Research Unit

    Boston, Massachusetts, 02118

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Northwestern University

    Chicago, Illinois, 60611

  • University of Chicago Medical Center

    Chicago, Illinois, 60637

  • Cleveland Clinic Main Campus

    Cleveland, Ohio, 44195

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Duke University Health System - Duke Clinic

    Durham, North Carolina, 27710

  • NorthShore University Health System

    Evanston, Illinois, 60201

  • Saint Luke's Hospital of Kansas City

    Kansas City, Missouri, 64111

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

  • New York University Langone Health

    New York, New York, 10016

  • Columbia University Irving Medical Center

    New York, New York, 10032

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania, 19104

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania, 15213

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Virginia Commonwealth University Health System

    Richmond, Virginia, 23219

  • Roanoke Heart Institute

    Roanoke, Virginia, 24014

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905

  • Laurelton Heart Specialists

    Rosedale, New York, 11422

  • University of Utah

    Salt Lake City, Utah, 84132

  • University of Washington School of Medicine

    Seattle, Washington, 98195

  • Providence Sacred Heart Children's Hospital

    Spokane, Washington, 99204

  • Washington University

    St Louis, Missouri, 63110

  • Montefiore Medical Center at Hutchinson

    The Bronx, New York, 10461

Other trials in United States

Canada8 sites

  • Queen Elizabeth II Health Sciences Centre - Halifax Infirmary

    Halifax, Nova Scotia, B3H 3A7

  • L' Institut de Cardiologie de Montréal

    Montreal, Quebec, H1T 1C8

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

    Montreal, Quebec, H2X 0A9

  • Hôpital du Sacré-Coeur de Montréal

    Montreal, Quebec, H4J 1C5

  • Hôpital régional de Rimouski

    Rimouski, Quebec, G5L 5T1

  • Toronto Heart Centre

    Toronto, Ontario, M4P IE4

  • Vancouver General Hospital

    Vancouver, British Columbia, V5Z 1M9

  • Saint Boniface Hospital

    Winnipeg, Manitoba, R2H 2A6

Other trials in Canada

Australia6 sites

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000

  • Box Hill Hospital

    Box Hill, Victoria, 3128

  • Saint Vincent's Hospital Sydney

    Darlinghurst, New South Wales, 2010

  • Royal Hobart Hospital

    Hobart, Tasmania, 7000

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150

  • Princess Alexandra Hospital

    Woolloongabba, Queensland, 4102

Other trials in Australia

Spain6 sites

  • Clinica Universidad de Navarra Madrid

    Madrid, Madrid, 28027

  • Hospital Universitario Puerta de Hierro - Majadahonda

    Majadahonda, Madrid, 28222

  • Hospital Son Llàtzer

    Palma de Mallorca, Balearic Islands, 7198

  • Clínica Universidad de Navarra

    Pamplona, Navarre, 31008

  • Complejo Hospitalario Universitario de Santiago (CHUS)

    Santiago de Compostela, La Coruña, 15706

  • Hospital Clínico Universitario de Valencia

    Valencia, Valencia, 46010

Other trials in Spain

Italy4 sites

  • Ospedale San Donato

    Arezzo, Arezzo, 52100

  • Azienda Ospedaliero - Universitaria Careggi

    Florence, Florence, 50134

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100

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

    Pisa, Pisa, 56124

Other trials in Italy

Brazil3 sites

  • Instituto de Cardiologia do Rio Grande do Sul

    Porto Alegre, Rio Grande do Sul, 90620-001

  • Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED

    Ribeirão Preto, São Paulo, 14026-900

  • Instituto do Coração de São Paulo

    São Paulo, São Paulo, 05403-000

Other trials in Brazil

Czechia3 sites

  • Fakultní Nemocnice u sv. Anny v Brně

    Brno, South Moravian, 656 91

  • Vseobecna Fakultni Nemocnice v Praze

    Prague, Prague, 128 08

  • Institut Klinické a Experimentální Medicíny

    Prague, Prague, 140 21

Other trials in Czechia

Belgium2 sites

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

    Bruges, West Vlaanderen, 8000

  • Ziekenhuis Oost-Limburg

    Genk, Limburg, 3600

Other trials in Belgium

Israel2 sites

  • Hadassah University Hospital Ein Kerem

    Jerusalem, 9112001

  • The Chaim Sheba Medical Center

    Ramat Gan, Tel Aviv, 5265601

Other trials in Israel

Netherlands2 sites

  • Universitair Medisch Centrum Groningen

    Groningen, Provincie Groningen, 9713 GZ

  • Universitair Medisch Centrum Utrecht

    Utrecht, Utrecht, 3584 CX

Other trials in Netherlands

New Zealand2 sites

  • Waikato Hospital

    Hamilton, Waikato Region, 3240

  • Middlemore Clinical Trials - Middlemore Hospital

    Papatoetoe, Auckland, 2025

Other trials in New Zealand

South Korea2 sites

  • Seoul National University Bundang Hospital

    Seongnam-si, Gyeonggi-do, 13620

  • Seoul National University Hospital

    Seoul, Gyeonggi-do, 3080

Other trials in South Korea

United Kingdom2 sites

  • National Amyloidosis Centre, Division of Medicine - Royal Free

    London, England, NW3 2PF

  • Richmond Pharmacology Ltd

    London, England, SE1 1YR

Other trials in United Kingdom

Denmark1 site

  • Aarhus Universitets Hospital

    Aarhus N, Palle Juul-Jensens, 8200

Other trials in Denmark

Greece1 site

  • Alexandra General Hospital of Athens

    Athens, Attica, 11528

Other trials in Greece

Ireland1 site

  • Mater Misericordiae University Hospital

    Dublin, Dublin, 7

Other trials in Ireland

Portugal1 site

  • Centro Hospitalar Universitário Lisboa Norte EPE- Hospital Santa Maria

    Lisbon, Lisbon District, 1649-035

Other trials in Portugal

About this study

Official title: An 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)

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)

Read the detailed description

The primary objective of this prospective, multi-center, open-label study is to evaluate the long-term safety and tolerability of acoramidis in patients with an established diagnosis of ATTR-CM and heart failure who are concomitantly treated with currently recommended heart failure therapies. Secondary efficacy and PD objectives will be assessed. Exploratory objectives may also be assessed.

All participants who complete 30 months of blinded study treatment and the Month 30 assessments of the double-blind treatment period of the Phase 3 ATTRibute-CM trial (AG10-301) may be eligible to participate in this Open Label Extension (OLE) study of acoramidis. The Day 1 visit in Study AG10-304 may be the same as the Month 30 visit in Study AG10-301. Under these circumstances, the last dose of Investigational Medicinal Product (IMP) in Study AG10-301 (either blinded acoramidis or matching placebo) will be the night before the day of the Month 30 visit. The first dose of open-label acoramidis in Study AG10-304 will be during the Study AG10-304 Day 1 visit after baseline assessments have been completed.

Currently, tafamidis is approved for the treatment of ATTR-CM in some regions. Participants are not allowed to be treated with tafamidis or any other ATTR-CM-specific approved or investigational treatment, or therapies used off-label or as non-prescription supplements for ATTR-CM at any time during the study. If participants choose treatment with an alternative treatment as described above, they will be asked to discontinue acoramidis and complete an End of Treatment (EoT) form, and they may be asked to discontinue/withdraw from the study. If a participant discontinues/withdraws from the study, the participant will be asked to complete an early termination visit and a safety follow-up visit.

Participants are not permitted to participate in another interventional clinical trial (except Study AG10-301) within 30 days prior to dosing and throughout Study AG10-304. Participants who choose to participate in another interventional clinical trial will be asked to withdraw from acoramidis and complete an EoT form, and they may be asked to discontinue/withdraw from the study. If a participant discontinues/withdraws from the study, the participant will be asked to complete an early termination visit and a safety follow-up visit. Participation in observational and/or registry studies should be discussed with the Medical Monitor.

Being tested
Acoramidis (AG10)
Keywords
ATTR-CM
Medical subject headings
Amyloid Neuropathies, Familial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Acoramidis (AG10)Drug

    Also called AG10, ALXN 2060

    Acoramidis (AG10) twice daily

Study groups in NCT04988386
Study groupRoleWhat they receive
AG10ExperimentalOpen-label study all participants will receive AG10 during this study.
Receives: Drug: Acoramidis (AG10)

What is being measured

The primary outcome is the main question the study is designed to answer.

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

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

    Measured: 60 months

Secondary outcomes (4)

  1. Evaluate all-cause mortality and cardiovascular mortality

    Assessment of All-cause mortality and CV mortality during study period

    Measured: 60 months

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

    Change from Baseline in distance walked during the 6MWT during study period

    Measured: 60 months

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

    Change from Baseline in Kansas City Cardiomyopathy Questionnaire Overall Score during study period [scale is scored from 0-100, where 0=Poor, 100=Excellent]

    Measured: 60 months

  4. Evaluate the effect of acoramidis on the frequency of CV-related hospitalization

    assess CV-related hospitalization during the study period

    Measured: 60 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Aug 2021
  2. Study start27 Oct 2021
  3. Primary completion15 Apr 2028 (estimated)
  4. Study completion15 May 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Open-label study extension study from the AG10-301 double-blind study.
Masking
None (open label)
Enrolment
389 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04988386
Sponsor's protocol ID
AG10-304
Other IDs
2020-005643-22
Acronym
AG10
Status last verified
Oct 2025
First submitted
13 Jul 2021
Last updated
14 Oct 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Testani JM, Judge DP, Borlaug BA, Cherney D, Cox ZL, Gillmore JD, Lewis JB, Adler SH, Cao X, Castano A, Fox JC, Katz L, Mathur V, Xiong K, Butler J, Masri A. Effects of Acoramidis on Kidney Function in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2026 Jul 2:e014656. doi: 10.1161/CIRCHEARTFAILURE.126.014656. Online ahead of print.

    PubMed 42389794

  • Soman P, Cuddy SAM, Gillmore JD, Sarswat N, Judge DP, Cappelli F, Stern LK, Wright R, Akinboboye O, Obici L, Bhatt K, Xiong K, Castano A, Chen C, Falvey H, Pecoraro ML, Tamby JF, Sinha U, Fox JC, Shah K, Grodin JL, Ambardekar AV, Alexander KM, Khouri MG, Cheng RK. Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial. JAMA Cardiol. 2026 May 1;11(5):446-454. doi: 10.1001/jamacardio.2026.0819.

    PubMed 41911489

  • 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. 2026 Jan 1;11(1):57-67. doi: 10.1001/jamacardio.2025.4477.

    PubMed 41205147

  • 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.

    PubMed 39556242

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Acoramidis (AG10) in people with Amyloid Cardiomyopathy, Transthyretin-Related, run by Eidos Therapeutics, a BridgeBio company at 72 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 14 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 72 sites in United States, Canada, Australia, Spain, Italy and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of treatment-emergent Adverse Events [Safety and Tolerability] (time frame: 60 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04988386, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2025.

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