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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04988386




Registration number
NCT04988386
Ethics application status
Date submitted
13/07/2021
Date registered
3/08/2021

Titles & IDs
Public title
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
Scientific 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)
Secondary ID [1] 0 0
AG10-304
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyloid Cardiomyopathy, Transthyretin-Related 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Acoramidis (AG10)

Experimental: AG10 - Open-label study all participants will receive AG10 during this study.


Treatment: Drugs: Acoramidis (AG10)
Acoramidis (AG10) twice daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent Adverse Events [Safety and Tolerability]
Timepoint [1] 0 0
60 months
Secondary outcome [1] 0 0
Evaluate all-cause mortality and cardiovascular mortality
Timepoint [1] 0 0
60 months
Secondary outcome [2] 0 0
Evaluate the effect of acoramidis on the 6-minute walk test (6MWT)
Timepoint [2] 0 0
60 months
Secondary outcome [3] 0 0
Evaluate the effect of acoramidis on health-related quality of life Kansas City Cardiomyopathy Questionnaire
Timepoint [3] 0 0
60 months
Secondary outcome [4] 0 0
Evaluate the effect of acoramidis on the frequency of CV-related hospitalization
Timepoint [4] 0 0
60 months

Eligibility
Key inclusion 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.
Minimum age
18 Years
Maximum age
90 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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.
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/05/2028
Actual
Sample size
Target
Accrual to date
Final
389
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,VIC,WA
Recruitment hospital [1] 0 0
Saint Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [2] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [4] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [5] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [6] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment postcode(s) [4] 0 0
7000 - Hobart
Recruitment postcode(s) [5] 0 0
3128 - Box Hill
Recruitment postcode(s) [6] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
South Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Belgium
State/province [18] 0 0
Limburg
Country [19] 0 0
Belgium
State/province [19] 0 0
West Vlaanderen
Country [20] 0 0
Brazil
State/province [20] 0 0
Rio Grande Do Sul
Country [21] 0 0
Brazil
State/province [21] 0 0
Sao Paulo
Country [22] 0 0
Canada
State/province [22] 0 0
British Columbia
Country [23] 0 0
Canada
State/province [23] 0 0
Manitoba
Country [24] 0 0
Canada
State/province [24] 0 0
Nova Scotia
Country [25] 0 0
Canada
State/province [25] 0 0
Ontario
Country [26] 0 0
Canada
State/province [26] 0 0
Quebec
Country [27] 0 0
Czechia
State/province [27] 0 0
Prague
Country [28] 0 0
Czechia
State/province [28] 0 0
South Moravian
Country [29] 0 0
Denmark
State/province [29] 0 0
Palle Juul-Jensens
Country [30] 0 0
Greece
State/province [30] 0 0
Attica
Country [31] 0 0
Ireland
State/province [31] 0 0
Dublin
Country [32] 0 0
Israel
State/province [32] 0 0
Tel Aviv
Country [33] 0 0
Israel
State/province [33] 0 0
Jerusalem
Country [34] 0 0
Italy
State/province [34] 0 0
Florence
Country [35] 0 0
Italy
State/province [35] 0 0
Arezzo
Country [36] 0 0
Italy
State/province [36] 0 0
Pavia
Country [37] 0 0
Italy
State/province [37] 0 0
Pisa
Country [38] 0 0
Korea, Republic of
State/province [38] 0 0
Gyeonggi-Do
Country [39] 0 0
Netherlands
State/province [39] 0 0
Groningen
Country [40] 0 0
Netherlands
State/province [40] 0 0
Utrecht
Country [41] 0 0
New Zealand
State/province [41] 0 0
Auckland
Country [42] 0 0
New Zealand
State/province [42] 0 0
Waikato
Country [43] 0 0
Portugal
State/province [43] 0 0
Lisboa
Country [44] 0 0
Spain
State/province [44] 0 0
Balearic Islands
Country [45] 0 0
Spain
State/province [45] 0 0
La Coruña
Country [46] 0 0
Spain
State/province [46] 0 0
Madrid
Country [47] 0 0
Spain
State/province [47] 0 0
Navarra
Country [48] 0 0
Spain
State/province [48] 0 0
Valencia
Country [49] 0 0
United Kingdom
State/province [49] 0 0
England

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Eidos Therapeutics, a BridgeBio company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04988386