Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
NCT ID: NCT04988386
Last Updated: 2025-10-14
Study Results
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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ACTIVE_NOT_RECRUITING
PHASE3
389 participants
INTERVENTIONAL
2021-10-27
2028-05-15
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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AG10
Open-label study all participants will receive AG10 during this study.
Acoramidis (AG10)
Acoramidis (AG10) twice daily
Interventions
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Acoramidis (AG10)
Acoramidis (AG10) twice daily
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures.
3. 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.
Exclusion Criteria
2. Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study.
3. Has had a heart and/or liver transplant or is on the heart transplantation list within the year prior to Day 1
4. Has had implantation of a cardiac mechanical assist device (CMAD) or is scheduled for implantation of a CMAD
5. Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301.
6. 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.
7. Known hypersensitivity to acoramidis, its metabolites, or formulation excipients.
8. 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.
9. 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.
10. 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.
11. 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.
12. 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.
18 Years
90 Years
ALL
No
Sponsors
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Eidos Therapeutics, a BridgeBio company
INDUSTRY
Responsible Party
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Locations
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Cedars-Sinai Medical Center
Los Angeles, California, United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, United States
Northwestern University
Chicago, Illinois, United States
University of Chicago Medical Center
Chicago, Illinois, United States
NorthShore University Health System
Evanston, Illinois, United States
MedStar Medical Group Cardiology at Franklin Square
Baltimore, Maryland, United States
Boston University Medical Center General Clinical Research Unit
Boston, Massachusetts, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Saint Luke's Hospital of Kansas City
Kansas City, Missouri, United States
Washington University
St Louis, Missouri, United States
New York University Langone Health
New York, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
Laurelton Heart Specialists
Rosedale, New York, United States
Montefiore Medical Center at Hutchinson
The Bronx, New York, United States
Duke University Health System - Duke Clinic
Durham, North Carolina, United States
Cleveland Clinic Main Campus
Cleveland, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University Health System
Richmond, Virginia, United States
Roanoke Heart Institute
Roanoke, Virginia, United States
University of Washington School of Medicine
Seattle, Washington, United States
Providence Sacred Heart Children's Hospital
Spokane, Washington, United States
Saint Vincent's Hospital Sydney
Darlinghurst, New South Wales, Australia
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
Royal Hobart Hospital
Hobart, Tasmania, Australia
Box Hill Hospital
Box Hill, Victoria, Australia
Fiona Stanley Hospital
Murdoch, Western Australia, Australia
Ziekenhuis Oost-Limburg
Genk, Limburg, Belgium
Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan
Bruges, West Vlaanderen, Belgium
Instituto de Cardiologia do Rio Grande do Sul
Porto Alegre, Rio Grande do Sul, Brazil
Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED
Ribeirão Preto, São Paulo, Brazil
Instituto do Coração de São Paulo
São Paulo, São Paulo, Brazil
Vancouver General Hospital
Vancouver, British Columbia, Canada
Saint Boniface Hospital
Winnipeg, Manitoba, Canada
Queen Elizabeth II Health Sciences Centre - Halifax Infirmary
Halifax, Nova Scotia, Canada
Toronto Heart Centre
Toronto, Ontario, Canada
L' Institut de Cardiologie de Montréal
Montreal, Quebec, Canada
Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, Canada
Hôpital du Sacré-Coeur de Montréal
Montreal, Quebec, Canada
Hôpital régional de Rimouski
Rimouski, Quebec, Canada
Vseobecna Fakultni Nemocnice v Praze
Prague, Prague, Czechia
Institut Klinické a Experimentální Medicíny
Prague, Prague, Czechia
Fakultní Nemocnice u sv. Anny v Brně
Brno, South Moravian, Czechia
Aarhus Universitets Hospital
Aarhus N, Palle Juul-Jensens, Denmark
Alexandra General Hospital of Athens
Athens, Attica, Greece
Mater Misericordiae University Hospital
Dublin, Dublin, Ireland
The Chaim Sheba Medical Center
Ramat Gan, Tel Aviv, Israel
Hadassah University Hospital Ein Kerem
Jerusalem, , Israel
Ospedale San Donato
Arezzo, Arezzo, Italy
Azienda Ospedaliero - Universitaria Careggi
Florence, Florence, Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica
Pisa, Pisa, Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
Universitair Medisch Centrum Groningen
Groningen, Provincie Groningen, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, Utrecht, Netherlands
Middlemore Clinical Trials - Middlemore Hospital
Papatoetoe, Auckland, New Zealand
Waikato Hospital
Hamilton, Waikato Region, New Zealand
Centro Hospitalar Universitário Lisboa Norte EPE- Hospital Santa Maria
Lisbon, Lisbon District, Portugal
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, South Korea
Seoul National University Hospital
Seoul, Gyeonggi-do, South Korea
Hospital Son Llàtzer
Palma de Mallorca, Balearic Islands, Spain
Complejo Hospitalario Universitario de Santiago (CHUS)
Santiago de Compostela, La Coruña, Spain
Clinica Universidad de Navarra Madrid
Madrid, Madrid, Spain
Hospital Universitario Puerta de Hierro - Majadahonda
Majadahonda, Madrid, Spain
Clínica Universidad de Navarra
Pamplona, Navarre, Spain
Hospital Clínico Universitario de Valencia
Valencia, Valencia, Spain
National Amyloidosis Centre, Division of Medicine - Royal Free
London, England, United Kingdom
Richmond Pharmacology Ltd
London, England, United Kingdom
Countries
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References
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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.
Other Identifiers
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2020-005643-22
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AG10-304
Identifier Type: -
Identifier Source: org_study_id
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