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    Summary
    EudraCT Number:2020-005643-22
    Sponsor's Protocol Code Number:AG10-304
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-03-08
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedIreland - HPRA
    A.2EudraCT number2020-005643-22
    A.3Full title of the trial
    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)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension study and safety monitoring of Acoramidis (AG10) in participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
    A.4.1Sponsor's protocol code numberAG10-304
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorEidos Therapeutics, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportEidos Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEidos Therapeutics, Inc.
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address1800 Owens Street, Suite C-1200
    B.5.3.2Town/ citySan Francisco
    B.5.3.3Post codeCA 94158
    B.5.3.4CountryUnited States
    B.5.4Telephone number0014158871471
    B.5.6E-mailinfo@eidostx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2081 - EMA/OD/096/18
    D.3 Description of the IMP
    D.3.1Product nameAcoramidis (AG10)
    D.3.2Product code AG10
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAcoramidis
    D.3.9.1CAS number 1446711-81-4
    D.3.9.3Other descriptive name3-(3-(3,5-DIMETHYL-1H-PYRAZOL-4-YL)PROPOXY)-4-FLUOROBENZOIC ACID
    D.3.9.4EV Substance CodeSUB193376
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number356
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
    E.1.1.1Medical condition in easily understood language
    Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a rare disease that occurs when a protein, called transthyretin, is accumulated
    in the heart.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002020
    E.1.2Term Amyloid cardiomyopathy
    E.1.2System Organ Class 10007541 - Cardiac disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess safety and tolerability of acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM)
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of acoramidis on: all-cause mortality (ACM) and cardiovascular (CV) mortality; the 6-minute walk test (6MWT); health-related quality of life (QoL) Kansas City Cardiomyopathy Questionnaire (KCCQ); the frequency of CV-related hospitalization (CVH)
    - To assess the pharmacodynamic (PD) effects of acoramidis as assessed by
    •circulating TTR concentration as an in vivo biomarker of stabilization and
    •established ex vivo assays of TTR stabilization
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible to participate in this OLE study, participants must meet all the following criteria:
    1. Completed 30 months of the blinded study treatment in Study AG10-301 and the Study AG10-301 Month 30 visit including assessments and procedures.
    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.
    E.4Principal exclusion criteria
    Participants who meet any of the following criteria at the Day 1 visit will not be eligible to participate in the study:
    1. Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study.
    2. Has had a heart and/or liver transplant within the year prior to Day 1.
    3. Has had implantation of a cardiac mechanical assist device (CMAD).
    4. Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301.
    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.
    6. Known hypersensitivity to acoramidis, its metabolites, or formulation excipients.
    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.
    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 female participants of childbearing potential.
    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.
    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.
    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.
    E.5 End points
    E.5.1Primary end point(s)
    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
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs: Month 0-60; ET, unscheduled visit
    Physical examination, Vital signs, ECG: Day 1, Month 1, Month 6-60 every 6 months, Month 61; ET, unscheduled visit, 30 days post last dose
    E.5.2Secondary end point(s)
    - All-cause mortality and CV mortality
    - Change from Baseline in distance walked during the 6MWT (6MWD)
    - Change from Baseline in KCCQ Overall Summary Score (KCCQ-OS)
    - CV-related hospitalization
    - Change from baseline in TTR (prealbumin) level (an in vivo measure of TTR stabilization)
    - TTR stabilization measured in established ex vivo assays (FPE and Western blot) in the PK-PD substudy
    E.5.2.1Timepoint(s) of evaluation of this end point
    - All-cause mortality and CV mortality: Month 0-61
    - Distance walked during the 6MWT: Day 1, Month 6, 12, 18, 24, 30, 36, 42, 48, 54, 60; ET, unscheduled visit
    - KCCQ will be obtained on Day 1, Month 1, Month 6, and every 6 months up to Month 60 during the study. KCCQ should be completed prior to the EQ-5D-5L and prior to conduction of the 6MWT at those visits during which a 6MWT is performed
    - CV-related hospitalization will be summarized by number of patients who have at least one CV-related hospitalization and the frequency of CV-related hospitalization; cumulative frequency of CV-related hospitalization: Month 0-61
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Information not present in EudraCT
    E.8.4 The trial involves multiple sites in the Member State concerned Information not present in EudraCT
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    New Zealand
    Australia
    Brazil
    Canada
    Israel
    Korea, Republic of
    United Kingdom
    United States
    Belgium
    Czechia
    Denmark
    Greece
    Ireland
    Italy
    Netherlands
    Portugal
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 535
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 223
    F.4.2.2In the whole clinical trial 546
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    The study is planned to continue until participants have access to acoramidis by prescription for treatment of ATTR-CM or up to approximately 60 months treatment period, followed by 1-month follow-up. Total duration of participation in the study is expected to be up to approximately 61 months.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-04-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-08-04
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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