Clinical trial • Phase III • Cardiology|Neurology|Rare Disease

acoramidis hydrochloride for Transthyretin amyloidosis | Transthyretin amyloid cardiomyopathy (ATTR-CM)

Phase III trial of acoramidis hydrochloride for Transthyretin amyloidosis | Transthyretin amyloid cardiomyopathy (ATTR-CM).

Overview

Trial Therapeutic Area
Cardiology|Neurology|Rare Disease
Trial Disease
Transthyretin amyloidosis | Transthyretin amyloid cardiomyopathy (ATTR-CM)
Trial Stage
Phase III
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
14-07-2025
First CTIS Authorization Date
22-10-2025

Trial design

open-label, none/not specified-controlled Phase III trial in Germany, Italy, Portugal and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
155

Eligibility

Recruits 155 isVulnerablePopulationSelected is true. Inclusion criterion 2: "Participant must be willing and able to give signed informed consent for participation in the study. Informed consent must be obtained prior to initiation of study procedures." No provisions for assent or minor consent are specified in the available record..

Pregnancy Exclusion
9. Female participants who are pregnant or breastfeeding. A negative urine pregnancy test on Day 1 prior to dosing is required for WOCBP. A positive urine dipstick pregnancy test will need to be confirmed with a serum test and participants cannot commence AG10-504 study unless the serum test results return negative.
Vulnerable Population
isVulnerablePopulationSelected is true. Inclusion criterion 2: "Participant must be willing and able to give signed informed consent for participation in the study. Informed consent must be obtained prior to initiation of study procedures." No provisions for assent or minor consent are specified in the available record.

Inclusion criteria

  • {"criterion_text":"- 1. Participants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM)."}
  • {"criterion_text":"- 2. Participant must be willing and able to give signed informed consent for participation in the study. Informed consent must be obtained prior to initiation of study procedures."}
  • {"criterion_text":"- 3. Agree to the use of highly effective contraception: a. FEMALE: WOCBP (defined as all women physiologically capable of becoming pregnant) who engage in heterosexual intercourse must agree to use a highly effective method of contraception from Day 1 and continuing for 30 calendar days after the last dose of study drug. (see Section 8.3.6). b. MALE: A male participant who has not had a vasectomy and is sexually active with a female of childbearing potential must agree to use a double-barrier method of birth control during the study and continue for 30 calendar days after the last dose of study drug. Males must agree to refrain from sperm donation for a minimum of 30 calendar days post the last dose of the study drug."}

Exclusion criteria

  • {"criterion_text":"- 1. Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only or who permanently discontinued IMP prior to diagnosis of ATTR-CM in AG10-501."}
  • {"criterion_text":"- 10. In the judgment of the Investigator or Medical Monitor, has any clinically relevant ongoing medical condition or laboratory abnormality or other condition that might jeopardize the participant’s safety, increase the participant’s risk from participation, interfere with the study, or confound study results."}
  • {"criterion_text":"- 11. Participation in another investigational clinical trial. Participation in observational and/or registry studies must be discussed with the Medical Monitor."}
  • {"criterion_text":"- 12. Major surgery planned during the next 6 months. For participants who may have required major surgery during their participation in the AG10-501 study and the decision by the Investigator at that time was to continue the participant in the AG10-501 study, that participant may still qualify for Study AG10-504 provided study participation does not interfere with the surgical care plan and vice versa."}
  • {"criterion_text":"- 2. History of light-chain amyloidosis (AL) or another non-TTR amyloid subtype (eg, ApoA 1, gelsolin)."}
  • {"criterion_text":"- 3. History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, MGUS) in which AL has not been ruled out."}
  • {"criterion_text":"- 4. Stage IV or V chronic kidney disease (corresponding to an eGFR ≤ 29 mL/min/1.73 m2), or undergoing renal dialysis, or recipient of a kidney transplant."}
  • {"criterion_text":"- 5. Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated. In the event a participant developed a low-grade and treatable malignancy in the AG10-501 study (eg, low-grade, localized prostate cancer) and the decision by the Investigator at that time was to continue the participant in the AG10-501 study, that participant may still qualify for Study AG10-504 after documentation with the Medical Monitor."}
  • {"criterion_text":"- 6. History of any organ transplant (with the exception of corneal transplant)."}
  • {"criterion_text":"- 7. Known hypersensitivity to acoramidis or any of the excipients within the study drug."}
  • {"criterion_text":"- 8. Treatment for ATTR-CM with any ATTR-oriented on- or off-label or OTC product."}
  • {"criterion_text":"- 9. Female participants who are pregnant or breastfeeding. A negative urine pregnancy test on Day 1 prior to dosing is required for WOCBP. A positive urine dipstick pregnancy test will need to be confirmed with a serum test and participants cannot commence AG10-504 study unless the serum test results return negative."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of participants with: treatment-emergent AEs and SAEs, AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal ECG parameters ofclinical relevance, changes in clinical safety laboratory parameters of potential concern","definition_or_measurement_approach":"Proportion of participants experiencing each listed safety outcome during treatment (treatment-emergent adverse events [AEs], serious AEs [SAEs], AEs leading to discontinuation, clinically relevant abnormal physical exam findings, vital signs, ECG parameters, and changes in clinical safety laboratory parameters)."}

Secondary endpoints

  • {"endpoint_text":"- 1. All-cause mortality and cardiovascular mortality","definition_or_measurement_approach":"Assessment of deaths classified as all-cause and cardiovascular mortality (as stated)."}
  • {"endpoint_text":"- 2. Cardiovascular-related hospitalization","definition_or_measurement_approach":"Occurrence/count of hospitalizations related to cardiovascular causes (as stated)."}
  • {"endpoint_text":"- 3. Participants with: • Any HF event: O-HF and/or UV-HF and/or H-HF • UV-HF and/or H-HF • O-HF • UV-HF • H-HF • NOHF","definition_or_measurement_approach":"Recording of predefined heart failure (HF) events: outpatient HF event (O-HF), urgent visit for HF (UV-HF), hospitalization for HF (H-HF), and new onset HF (NOHF) as listed."}
  • {"endpoint_text":"- 4. Development of symptomatic ATTR-CM as defined by the investigator","definition_or_measurement_approach":"Investigator-defined development of symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM)."}
  • {"endpoint_text":"- 5. New arrhythmia and cardiac conduction complications (not otherwise attributable to a non-amyloid cause) as defined by: • New onset of atrial fibrillation or atrial flutter and/or • Sustained ventricular tachycardia or sudden cardiac death and/or • New onset 1st or 2nd degree atrioventricular block and/or • Conduction disease requiring temporary transvenous or permanent pacemaker implantation","definition_or_measurement_approach":"Identification of new arrhythmias or conduction disorders meeting the listed criteria (new AF/flutter, sustained VT or SCD, new 1st/2nd degree AV block, conduction disease requiring pacemaker)."}
  • {"endpoint_text":"- 6. New diagnosis of or therapeutic surgery (not otherwise attributable to a non-amyloid cause) for: • Carpal tunnel syndrome and/or • Brachial biceps tendon rupture or Achilles tendon rupture and/or • Lumbar spinal stenosis and/or • Hip and knee osteoarthritis and/or • Trigger finger and/or • Rotator cuff osteoarthritis","definition_or_measurement_approach":"Recording of new diagnoses or surgeries for listed musculoskeletal conditions not otherwise attributable to non-amyloid causes."}
  • {"endpoint_text":"- 7. Change from baseline in: o Echocardiographic and strain parameters that estimate myocardial structure and function o NT-proBNP and hsTnI","definition_or_measurement_approach":"Change from baseline for echocardiographic and strain parameters and biomarker levels (NT-proBNP, hsTnI)."}

Recruitment

Planned Sample Size
155
Recruitment Window Months
77
Consent Approach
"Participant must be willing and able to give signed informed consent for participation in the study. Informed consent must be obtained prior to initiation of study procedures." Subject information and ICF documents (including a Pregnant Participant ICF and materials in multiple languages) are provided; consent is signed by participant prior to study procedures. No assent procedures for minors are described.

Methods

  • Rollover recruitment from participants who completed the AG10-501 study (ACT-EARLY); inclusion criterion requires participants to have completed AG10-501 within the past 60 calendar days.

Geography

Total Number Of Sites
34
Total Number Of Participants
117

Germany

Earliest CTIS Part Ii Submission Date
06-10-2025
Latest Decision Or Authorization Date
28-10-2025
Processing Time Days
22
Number Of Sites
3
Number Of Participants
9

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Kardiologie, Angiologie und Intensivsmedizin
Principal Investigator Name
Katrin Hahn
Principal Investigator Email
katrin.hahn@charite.de
Contact Person Name
Katrin Hahn
Contact Person Email
katrin.hahn@charite.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
Studienzentrum der Klinik und Poliklinik für Neurologie
Principal Investigator Name
Frank Birklein
Principal Investigator Email
frank.birklein@unimedizin-mainz.de
Contact Person Name
Frank Birklein
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Neurologische Klinik
Principal Investigator Name
Markus Weiler
Principal Investigator Email
markus.weiler@med.uni-heidelberg.de
Contact Person Name
Markus Weiler

Italy

Earliest CTIS Part Ii Submission Date
01-08-2025
Latest Decision Or Authorization Date
27-10-2025
Processing Time Days
87
Number Of Sites
7
Number Of Participants
20

Sites

Site Name
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Department Name
Neurology
Principal Investigator Name
Anna Mazzeo
Principal Investigator Email
annamazzeo@yahoo.it
Contact Person Name
Anna Mazzeo
Contact Person Email
annamazzeo@yahoo.it
Site Name
Fondazione Toscana Gabriele Monasterio
Department Name
Cardiology
Principal Investigator Name
Alberto Aimo
Principal Investigator Email
aimoalb@ftgm.it
Contact Person Name
Alberto Aimo
Contact Person Email
aimoalb@ftgm.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
rare diseases
Principal Investigator Name
Laura Piera Obici
Principal Investigator Email
l.obici@smatteo.pv.it
Contact Person Name
Laura Piera Obici
Contact Person Email
l.obici@smatteo.pv.it
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Neurology
Principal Investigator Name
Luca Leonardi
Principal Investigator Email
leonardi.luca89@gmail.com
Contact Person Name
Luca Leonardi
Contact Person Email
leonardi.luca89@gmail.com
Site Name
Azienda Ospedaliero Universitaria Careggi
Department Name
Cardiology
Principal Investigator Name
Francesco Cappelli
Principal Investigator Email
f.cappelli@unifi.it
Contact Person Name
Francesco Cappelli
Contact Person Email
f.cappelli@unifi.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Cardiology
Principal Investigator Name
Simone Longhi
Principal Investigator Email
simone.longhi@aosp.bo.it
Contact Person Name
Simone Longhi
Contact Person Email
simone.longhi@aosp.bo.it
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Neurology
Principal Investigator Name
Marco Luigetti
Principal Investigator Email
marco.luigetti@policlinicogemelli.it
Contact Person Name
Marco Luigetti

Portugal

Earliest CTIS Part Ii Submission Date
01-08-2025
Latest Decision Or Authorization Date
23-10-2025
Processing Time Days
83
Number Of Sites
2
Number Of Participants
11

Sites

Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Department of Neurosciences
Principal Investigator Name
Isabel Conceição
Principal Investigator Email
imsconceicao@gmail.com
Contact Person Name
Isabel Conceição
Contact Person Email
imsconceicao@gmail.com
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Neurophysiology Department
Principal Investigator Name
Márcio Cardoso
Contact Person Name
Márcio Cardoso

Netherlands

Earliest CTIS Part Ii Submission Date
03-10-2025
Latest Decision Or Authorization Date
24-10-2025
Processing Time Days
21
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Cardiology
Principal Investigator Name
Michelle Michels
Principal Investigator Email
m.michels@erasmusmc.nl
Contact Person Name
Michelle Michels
Contact Person Email
m.michels@erasmusmc.nl
Site Name
Universitair Medisch Centrum Utrecht
Department Name
Cardiology
Principal Investigator Name
Marish Oerlemans
Principal Investigator Email
m.oerlemans@umcutrecht.nl
Contact Person Name
Marish Oerlemans
Contact Person Email
m.oerlemans@umcutrecht.nl
Site Name
Universitair Medisch Centrum Groningen
Department Name
Internal Medicine
Principal Investigator Name
Hans Nienhuis
Principal Investigator Email
h.l.a.nienhuis@umcg.nl
Contact Person Name
Hans Nienhuis
Contact Person Email
h.l.a.nienhuis@umcg.nl

Ireland

Earliest CTIS Part Ii Submission Date
26-09-2025
Latest Decision Or Authorization Date
31-10-2025
Processing Time Days
35
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Tallaght University Hospital
Department Name
Neurology
Principal Investigator Name
Sinead Murphy
Principal Investigator Email
Sinead.murphy@tuh.ie
Contact Person Name
Sinead Murphy
Contact Person Email
Sinead.murphy@tuh.ie
Site Name
Mater Misericordiae University Hospital
Department Name
Cardiology
Principal Investigator Name
Emer Joyce
Principal Investigator Email
emerjoyce@mater.ie
Contact Person Name
Emer Joyce
Contact Person Email
emerjoyce@mater.ie

France

Earliest CTIS Part Ii Submission Date
18-09-2025
Latest Decision Or Authorization Date
12-12-2025
Processing Time Days
85
Number Of Sites
6
Number Of Participants
24

Sites

Site Name
CHU De Martinique
Department Name
cardiology
Principal Investigator Name
Jocelyn Inamo
Principal Investigator Email
Inamo_jocelyn@yahoo.com
Contact Person Name
Jocelyn Inamo
Contact Person Email
Inamo_jocelyn@yahoo.com
Site Name
Bicetre Hospital
Department Name
Nephrology
Principal Investigator Name
Andoni Echaniz-Laguna
Principal Investigator Email
andoni.echaniz-laguna@aphp.fr
Contact Person Name
Andoni Echaniz-Laguna
Contact Person Email
andoni.echaniz-laguna@aphp.fr
Site Name
Pellegrin Hospital
Department Name
Neurology
Principal Investigator Name
Guilhem Sole
Principal Investigator Email
Guilhem.sole@chu-bordeaux.fr
Contact Person Name
Guilhem Sole
Contact Person Email
Guilhem.sole@chu-bordeaux.fr
Site Name
CHU de Rennes
Department Name
cardiology
Principal Investigator Name
Erwan Donal
Principal Investigator Email
erwan.donal@chu-rennes.fr
Contact Person Name
Erwan Donal
Contact Person Email
erwan.donal@chu-rennes.fr
Site Name
AP HP Hopital Henri Mondor
Department Name
cardiology
Principal Investigator Name
Thibaud Damy
Principal Investigator Email
thibaud.damy@aphp.fr
Contact Person Name
Thibaud Damy
Contact Person Email
thibaud.damy@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
cardiology
Principal Investigator Name
Olivier Lairez
Principal Investigator Email
lairez.o@chu-toulouse.fr
Contact Person Name
Olivier Lairez
Contact Person Email
lairez.o@chu-toulouse.fr

Sweden

Earliest CTIS Part Ii Submission Date
29-01-2026
Latest Decision Or Authorization Date
20-02-2026
Processing Time Days
22
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Region Vaesterbotten (Umea University)
Department Name
Norrlands Universitetssjukhus, Amyloidosis center E41, Forsorjningsvagen 1 C, 901 85, Umeå, Sweden
Principal Investigator Name
Jonas Wixner
Principal Investigator Email
Jonas.wixner@umu.se
Contact Person Name
Jonas Wixner
Contact Person Email
Jonas.wixner@umu.se

Spain

Earliest CTIS Part Ii Submission Date
02-10-2025
Latest Decision Or Authorization Date
14-01-2026
Processing Time Days
104
Number Of Sites
6
Number Of Participants
22

Sites

Site Name
Bellvitge University Hospital
Department Name
Cardiology
Principal Investigator Name
José González Costello
Principal Investigator Email
jgonzalez@bellvitgehospital.cat
Contact Person Name
José González Costello
Site Name
Hospital Universitario De Salamanca
Department Name
Cardiology
Principal Investigator Name
Rocío Eiros Bachiller
Principal Investigator Email
eirosbachiller@gmail.com
Contact Person Name
Rocío Eiros Bachiller
Contact Person Email
eirosbachiller@gmail.com
Site Name
Hospital Universitario Juan Ramon Jimenez
Department Name
Internal Medicine
Principal Investigator Name
Cristina Borrachero Garro
Principal Investigator Email
crborrachero@gmail.com
Contact Person Name
Cristina Borrachero Garro
Contact Person Email
crborrachero@gmail.com
Site Name
Hospital Son Llatzer
Department Name
Internal Medicine
Principal Investigator Name
Juan González-Moreno
Principal Investigator Email
jgonzalez4@hsll.es
Contact Person Name
Juan González-Moreno
Contact Person Email
jgonzalez4@hsll.es
Site Name
Hospital Universitario Puerta De Hierro De Majadahonda
Department Name
Cardiology
Principal Investigator Name
Esther González López
Principal Investigator Email
esthgonzalez@hotmail.com
Contact Person Name
Esther González López
Contact Person Email
esthgonzalez@hotmail.com
Site Name
Complexo Hospitalario Universitario A Coruna
Department Name
Cardiology
Principal Investigator Name
Roberto Barriales Villa
Principal Investigator Email
roberto.barriales.villa@sergas.es
Contact Person Name
Roberto Barriales Villa

Denmark

Earliest CTIS Part Ii Submission Date
29-01-2026
Latest Decision Or Authorization Date
06-03-2026
Processing Time Days
36
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Region Midtjylland
Department Name
Cardiology
Principal Investigator Name
Torsten Rasmussen
Principal Investigator Email
torasm@rm.dk
Contact Person Name
Torsten Rasmussen
Contact Person Email
torasm@rm.dk

Greece

Earliest CTIS Part Ii Submission Date
27-11-2025
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
67
Number Of Sites
2
Number Of Participants
10

Sites

Site Name
Hippokration Hospital
Department Name
1st Cardiology Department, National and Kapodistrian University of Athens
Principal Investigator Name
Charalambos Vlachopoulos
Principal Investigator Email
ekkan@med.uoa.gr
Contact Person Name
Charalambos Vlachopoulos
Contact Person Email
ekkan@med.uoa.gr
Site Name
General Hospital Of Athens Alexandra
Department Name
Center of Expertise in Amyloidosis,Plasma Cell Dyscrasias U/Hem-Onc U,Dpt Clinical Ther,Med Sch NKUA
Principal Investigator Name
Efstathios Kastritis
Principal Investigator Email
ekastritis@med.uoa.gr
Contact Person Name
Efstathios Kastritis
Contact Person Email
ekastritis@med.uoa.gr

Belgium

Earliest CTIS Part Ii Submission Date
30-01-2026
Latest Decision Or Authorization Date
13-03-2026
Processing Time Days
42
Number Of Sites
1
Number Of Participants
7

Sites

Site Name
UZ Leuven
Department Name
Neurology
Principal Investigator Name
Kristl Claeys
Principal Investigator Email
kristl.claeys@uzleuven.be
Contact Person Name
Kristl Claeys
Contact Person Email
kristl.claeys@uzleuven.be

Sponsor

Primary sponsor

Full Name
Eidos Therapeutics Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Medpace Ellas Monoprosopi I.K.E.
Responsibilities
Sponsor duties (codes 1, 12)
Name
Eresearchtechnology Inc. (Clario)
Responsibilities
Central ECG Reader
Name
United Biosource LLC
Responsibilities
Safety database management
Name
Almac Clinical Services LLC
Responsibilities
IVRS & Randomization, IP Depot Distribution and Packaging
Name
Medpace Finland Oy
Responsibilities
Safety labs, sample management and related services

Third parties

  • {"country":"Greece","full_name":"Medpace Ellas Monoprosopi I.K.E.","duties_or_roles":"sponsorDuties codes: 1, 12","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central ECG Reader","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"United Biosource LLC","duties_or_roles":"safety database","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Services LLC","duties_or_roles":"IVRS & Randomization, IP Depot Distribution and Packaging; other duties (codes 14,3,15)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Allucent (US) LLC","duties_or_roles":"Data Flow Mapping for GDPR","organisation_type":"Pharmaceutical company"}
  • {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"Safety Labs, sample management; other sponsor duties (codes 1,12,15,4)","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Acoramidis (AG10)
Active Substance
acoramidis hydrochloride
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Maximum Dose
1424 mg

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