Clinical trial • Phase III • Cardiology
Acoramidis hydrochloride for Transthyretin amyloid cardiomyopathy (ATTR-CM)
Phase III trial of Acoramidis hydrochloride for Transthyretin amyloid cardiomyopathy (ATTR-CM). open-label. 236 participants.
Overview
- Trial Therapeutic Area
- Cardiology
- Trial Disease
- Transthyretin amyloid cardiomyopathy (ATTR-CM)
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 31-07-2024
- First CTIS Authorization Date
- 29-08-2024
Trial design
open-label Phase III trial across 21 sites in Belgium, Czechia, Denmark and others.
- Open Label
- Yes
- Target Sample Size
- 236
- Trial Duration For Participant
- 3680
Eligibility
Recruits 236 The CTIS record indicates isVulnerablePopulationSelected=true. Inclusion criterion requires that participants "Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures." No pediatric assent or minor-specific consent procedures are described in the available documents..
- Pregnancy Exclusion
- 8. Females who are pregnant or breastfeeding. A negative urine pregnancy test at the Day 1 visit and at each study visit are required for WOCBP.
- Vulnerable Population
- The CTIS record indicates isVulnerablePopulationSelected=true. Inclusion criterion requires that participants "Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures." No pediatric assent or minor-specific consent procedures are described in the available documents.
Inclusion criteria
- {"criterion_text":"- 1. Completed 30 months of the blinded study treatment in Study AG10301 and the Study AG10-301 Month 30 visit including assessments and procedures."}
- {"criterion_text":"- 2. Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures."}
- {"criterion_text":"- 3. Women of childbearing potential (WOCBP) 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 women of childbearing potential and has not had a vasectomy must agree to use a double-barrier method of birth control."}
Exclusion criteria
- {"criterion_text":"- 1. Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study."}
- {"criterion_text":"- 10. 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."}
- {"criterion_text":"- 11. 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."}
- {"criterion_text":"- 2. Has had a heart and/or liver transplant within the year prior to Day 1."}
- {"criterion_text":"- 3. Has had implantation of a cardiac mechanical assist device (CMAD)."}
- {"criterion_text":"- 4. Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301."}
- {"criterion_text":"- 5. Is on dialysis or has a degree of renal impairment that in the opinion of the Investigator might jeopardize the participant's safety, increase their risk from participation, or interfere with the study."}
- {"criterion_text":"- 6. Known hypersensitivity to acoramidis, its metabolites, or formulation excipients."}
- {"criterion_text":"- 7. 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."}
- {"criterion_text":"- 8. Females who are pregnant or breastfeeding. A negative urine pregnancy test at the Day 1 visit and at each study visit are required for WOCBP."}
- {"criterion_text":"- 9. 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."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Safety parameters to be assessed: treatment- emergent serious adverse events (SAEs) and adverse events (AEs), AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal electrocardiogram (ECG) parameters of clinical relevance, and changes in clinical safety laboratory parameters of clinical relevance","definition_or_measurement_approach":"Safety parameters as listed in the endpoint text: assessment of treatment-emergent SAEs and AEs, AEs leading to treatment discontinuation, clinically relevant abnormal physical exam findings, vital signs, ECG parameters, and changes in clinical safety laboratory parameters."}
Secondary endpoints
- {"endpoint_text":"- All-cause mortality and CV mortality","definition_or_measurement_approach":"All-cause mortality and cardiovascular mortality as recorded during study follow-up."}
- {"endpoint_text":"- Change from Baseline in distance walked during the 6MWT (6MWD)","definition_or_measurement_approach":"Change from baseline in 6-minute walk test distance (6MWD)."}
- {"endpoint_text":"- Change from Baseline in KCCQ Overall Summary Score (KCCQ-OS)","definition_or_measurement_approach":"Change from baseline in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OS)."}
- {"endpoint_text":"- CV-related hospitalization","definition_or_measurement_approach":"Frequency/incidence of cardiovascular-related hospitalizations during the study."}
- {"endpoint_text":"- Change from baseline in TTR level (an in vivo measure of TTR stabilization)","definition_or_measurement_approach":"Change from baseline in circulating TTR concentration measured as an in vivo biomarker of TTR stabilization."}
- {"endpoint_text":"- TTR stabilization measured in established ex vivo assays (FPE and D39Western blot) in the PK-PD substudy","definition_or_measurement_approach":"TTR stabilization assessed using established ex vivo assays (FPE and D39 Western blot) in the PK-PD substudy."}
Recruitment
- Planned Sample Size
- 236
- Recruitment Window Months
- 137
- Consent Approach
- Written informed consent is required prior to initiation of study procedures: "Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures." Subject information and consent documents are available in multiple languages (examples in the CTIS documents: Dutch, French, Greek, Italian, Portuguese, Spanish, Danish, Czech and English). No pediatric assent is described; pregnancy/partner information forms are provided separately where relevant.
Geography
- Total Number Of Sites
- 21
- Total Number Of Participants
- 213
Belgium
- Latest Decision Or Authorization Date
- 29-08-2024
- Number Of Sites
- 2
- Number Of Participants
- 21
Sites
- Site Name
- Ziekenhuis Oost Limburg
- Department Name
- Department of Cardiology
- Principal Investigator Name
- Matthias Dupont
- Principal Investigator Email
- matthias.dupont@zol.be
- Contact Person Name
- Matthias Dupont
- Contact Person Email
- matthias.dupont@zol.be
- Site Name
- Az St-Jan Brugge-Oostende A.V.
- Department Name
- Department of Cardiology
- Principal Investigator Name
- Philippe Debonnaire
- Principal Investigator Email
- philippe.debonnaire@azsintjan.be
- Contact Person Name
- Philippe Debonnaire
- Contact Person Email
- philippe.debonnaire@azsintjan.be
Czechia
- Latest Decision Or Authorization Date
- 03-09-2024
- Number Of Sites
- 3
- Number Of Participants
- 22
Sites
- Site Name
- Institute For Clinical And Experimental Medicine
- Department Name
- Klinika kardiologie
- Principal Investigator Name
- Miloš Kubánek
- Principal Investigator Email
- mikb@ikem.cz
- Contact Person Name
- Miloš Kubánek
- Contact Person Email
- mikb@ikem.cz
- Site Name
- Vseobecna Fakultni Nemocnice V Praze
- Department Name
- II. Interní klinika Kardiologie a angiologie
- Principal Investigator Name
- Tomáš Paleček
- Principal Investigator Email
- tomas.palecek@vfn.cz
- Contact Person Name
- Tomáš Paleček
- Contact Person Email
- tomas.palecek@vfn.cz
- Site Name
- Fakultni Nemocnice U Sv Anny V Brne
- Department Name
- I. Interní kardioangiologická klinika
- Principal Investigator Name
- Jan Krejčí
- Principal Investigator Email
- jan.krejci@fnusa.cz
- Contact Person Name
- Jan Krejčí
- Contact Person Email
- jan.krejci@fnusa.cz
Denmark
- Latest Decision Or Authorization Date
- 30-08-2024
- Number Of Sites
- 1
- Number Of Participants
- 40
Sites
- Site Name
- Aarhus Universitetshospital
- Department Name
- Hjertesygdomme – Klinisk Forskning
- Principal Investigator Name
- Steen Hvitfeldt Poulsen
- Principal Investigator Email
- steen.hvitfeldt@rm.dk
- Contact Person Name
- Steen Hvitfeldt Poulsen
- Contact Person Email
- steen.hvitfeldt@rm.dk
Greece
- Latest Decision Or Authorization Date
- 20-09-2024
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Alexandra Hospital
- Department Name
- Department of Clinical Therapeutics, Plasma Cell Dyscrasias Unit
- Principal Investigator Name
- Efstathios Kastritis
- Principal Investigator Email
- ekastritis@med.uoa.gr
- Contact Person Name
- Efstathios Kastritis
- Contact Person Email
- ekastritis@med.uoa.gr
Ireland
- Latest Decision Or Authorization Date
- 29-08-2024
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Mater Misericordiae University Hospital
- Department Name
- Catherine McAuley Centre
- Principal Investigator Name
- Emer Joyce
- Principal Investigator Email
- mater.medicine@ucd.ie
- Contact Person Name
- Emer Joyce
- Contact Person Email
- mater.medicine@ucd.ie
Italy
- Latest Decision Or Authorization Date
- 16-10-2024
- Number Of Sites
- 4
- Number Of Participants
- 59
Sites
- Site Name
- Fondazione IRCCS Policlinico San Matteo
- Department Name
- U.O.C. Medicina Generale 2- Centro Amiloidosi Sistemiche e Malattie ad Alta Complessità
- 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
- Fondazione Toscana Gabriele Monasterio
- Department Name
- Cardio-Thoracic Department FTGM
- Principal Investigator Name
- Michele Emdin
- Principal Investigator Email
- emdin@ftgm.it
- Contact Person Name
- Michele Emdin
- Contact Person Email
- emdin@ftgm.it
- Site Name
- Careggi University Hospital
- Department Name
- Intensive Cardiac Care Unit- SOD Interventistica Cardiologica Strutturale
- Principal Investigator Name
- Francesco Cappelli
- Principal Investigator Email
- cappellif@aou-careggi.toscana.it
- Contact Person Name
- Francesco Cappelli
- Contact Person Email
- cappellif@aou-careggi.toscana.it
- Site Name
- Azienda Unita' Sanitaria Locale Toscana Sud Est
- Department Name
- Department Cardio Toraco Neuro Vascolare- Area Funzionale Cardiologica
- Principal Investigator Name
- Michele Ciabatti
- Principal Investigator Email
- michele.ciabatti1989@gmail.com
- Contact Person Name
- Michele Ciabatti
- Contact Person Email
- michele.ciabatti1989@gmail.com
Portugal
- Latest Decision Or Authorization Date
- 02-09-2024
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Unidade Local De Saude De Santa Maria E.P.E.
- Department Name
- Cardiology Department
- Principal Investigator Name
- João Agostinho
- Principal Investigator Email
- joaoragostinho@gmail.com
- Contact Person Name
- João Agostinho
- Contact Person Email
- joaoragostinho@gmail.com
Spain
- Latest Decision Or Authorization Date
- 29-08-2024
- Number Of Sites
- 6
- Number Of Participants
- 48
Sites
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- Cardiology
- Principal Investigator Name
- María Inés Gómez Otero
- Principal Investigator Email
- Maria.Ines.Gomez.Otero@sergas.es
- Contact Person Name
- María Inés Gómez Otero
- Contact Person Email
- Maria.Ines.Gomez.Otero@sergas.es
- Site Name
- Hospital Clinico Universitario De Valencia
- Department Name
- Cardiology Department
- Principal Investigator Name
- Julio Nuñez de Villota
- Principal Investigator Email
- yulnunez@gmail.com
- Contact Person Name
- Julio Nuñez de Villota
- Contact Person Email
- yulnunez@gmail.com
- Site Name
- Clinica Universidad De Navarra
- Department Name
- Hematology
- Principal Investigator Name
- Ramon Lecumberri Villamediana
- Principal Investigator Email
- eecc@unav.es
- Contact Person Name
- Ramon Lecumberri Villamediana
- Contact Person Email
- eecc@unav.es
- Site Name
- Hospital Universitario Puerta De Hierro De Majadahonda
- Department Name
- Cardiology
- Principal Investigator Name
- Pablo Garcia Pavia
- Principal Investigator Email
- pablogpavia@yahoo.es
- Contact Person Name
- Pablo Garcia Pavia
- Contact Person Email
- pablogpavia@yahoo.es
- Site Name
- Hospital Son Llatzer
- Department Name
- Cardiology
- Principal Investigator Name
- Tomas Ripoll Vera
- Principal Investigator Email
- tripoll@hsll.es
- Contact Person Name
- Tomas Ripoll Vera
- Contact Person Email
- tripoll@hsll.es
- Site Name
- Clinica Universidad De Navarra
- Department Name
- Hematology
- Principal Investigator Name
- Ramon Lecumberri Villamediana
- Principal Investigator Email
- eecc@unav.es
- Contact Person Name
- Ramon Lecumberri Villamediana
- Contact Person Email
- eecc@unav.es
Netherlands
- Latest Decision Or Authorization Date
- 02-09-2024
- Number Of Sites
- 2
- Number Of Participants
- 12
Sites
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- Cardiologie
- Principal Investigator Name
- Peter van der Meer
- Principal Investigator Email
- p.van.der.meer@umcg.nl
- Contact Person Name
- Peter van der Meer
- Contact Person Email
- p.van.der.meer@umcg.nl
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- Polikliniek Cardiologie
- Principal Investigator Name
- Marish Oerlemans
- Principal Investigator Email
- m.i.f.oerlemans-4@umcutrecht.nl
- Contact Person Name
- Marish Oerlemans
- Contact Person Email
- m.i.f.oerlemans-4@umcutrecht.nl
Sponsor
Primary sponsor
- Full Name
- Eidos Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Icon Clinical Research Limited
- Responsibilities
- Multiple sponsor duties (codes: 1,12,15,2,4,6,7,8) including Primary/surrogate endpoint test (code 15)
- Name
- PRA Hellas CRO A.E.
- Responsibilities
- CRO (sponsor duties code: 15)
Third parties
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Sponsor duties codes: 1, 12, 15 (Primary/ surrogate endpoint test), 2, 4, 6, 7, 8","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"United Biosource LLC","duties_or_roles":"Sponsor duties codes: 8","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Almac Clinical Services LLC","duties_or_roles":"Sponsor duties codes: 3","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Sponsor duties code: 15 (ECG analysis/ review)","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Almac Clinical Services (Ireland) Limited","duties_or_roles":"Sponsor duties code: 15 (IMP Import)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Duke Clinical And Translational Science Institute","duties_or_roles":"Sponsor duties code: 10","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"Sponsor duties code: 15 (IMP Import)","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"Sponsor duties code: 15 (CRO)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Acoramidis (AG10)
- Active Substance
- Acoramidis hydrochloride
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Orphan Designation
- Yes
- Maximum Dose
- 1424 mg (max daily dose amount as listed)
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