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Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
EU CTIS ID: 2024-513547-82-00
What this study is testing
To determine the efficacy of acoramidis to prevent ATTR (ATTR-CM or ATTR-PN, whichever occurs first) in asymptomatic carriers with a pathogenic TTR variant who are at risk of developing ATTR but have no clinical evidence of disease at study entry
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Participants must be willing and able to give signed informed consent for study procedures. Informed consent must be obtained prior to initiation of study procedures.
- 2. Male or female ≥ 18 to ≤ 75 years of age inclusive when signing the informed consent. The minimum age requirement will comply with local regulatory requirements.
- 3. Participants must have an established genotype (hetero- or homozygosity) through a medically indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic. Participants with rare pathogenic TTR variants documented to be cardiac radionuclide uptake negative may be included in the trial, provided the participant can be assessed for the primary ATTR-CM endpoint For further details, please refer to the protocol.
- 4. Participant’s age is within 10 years younger than or older than PADO. The participant may be older than PADO. For example, if PADO for a given participant is determined to be 50 years, that participant must be at least 40 years of age (≥ 40) and less than or equal to 75 years of age (≤ 75). Please refer to the Genotype Manual for details on the calculation of PADO. For further details, please refer to the protocol.
- 5. 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 beginning before a radionuclide cardiac amyloid imaging with SPECT is performed during the screening period and continuing for 30 days after the last dose of study drug. Female participants using oral contraceptives must agree to use an additional birth control method, whisch may include a double-barrier method. 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 days after the last dose of study drug. Males must agree to refrain from sperm donation for a minimum of 30 days post the last dose of the study drug.
You likely can't join if
- 1. Myocardial radionuclide uptake of Grade 1 to Grade 3 on planar imaging confirmed to be myocardial by SPECT imaging.
- 10. Clinical evidence of untreated hyperthyroidism or hypothyroidism
- 11. History of type 1 diabetes
- 12. Known active hepatitis B or C (participants with resolved or cured infection are eligible for enrollment).
- 13. Known HIV infection.
- 14. History, within the previous 6 months, of nonreversible cardiomyopathy (eg, reversible cardiomyopathy examples include Takotsubo cardiomyopathy, viral cardiomyopathy, ischemic mitral regurgitation), untreated or uncontrolled cardiac arrhythmia, stroke, MI, or ACS
See the full eligibility criteria
Who can join
- 1. Participants must be willing and able to give signed informed consent for study procedures. Informed consent must be obtained prior to initiation of study procedures.
- 2. Male or female ≥ 18 to ≤ 75 years of age inclusive when signing the informed consent. The minimum age requirement will comply with local regulatory requirements.
- 3. Participants must have an established genotype (hetero- or homozygosity) through a medically indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic. Participants with rare pathogenic TTR variants documented to be cardiac radionuclide uptake negative may be included in the trial, provided the participant can be assessed for the primary ATTR-CM endpoint For further details, please refer to the protocol.
- 4. Participant’s age is within 10 years younger than or older than PADO. The participant may be older than PADO. For example, if PADO for a given participant is determined to be 50 years, that participant must be at least 40 years of age (≥ 40) and less than or equal to 75 years of age (≤ 75). Please refer to the Genotype Manual for details on the calculation of PADO. For further details, please refer to the protocol.
- 5. 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 beginning before a radionuclide cardiac amyloid imaging with SPECT is performed during the screening period and continuing for 30 days after the last dose of study drug. Female participants using oral contraceptives must agree to use an additional birth control method, whisch may include a double-barrier method. 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 days after the last dose of study drug. Males must agree to refrain from sperm donation for a minimum of 30 days post the last dose of the study drug.
What rules you out
- 1. Myocardial radionuclide uptake of Grade 1 to Grade 3 on planar imaging confirmed to be myocardial by SPECT imaging.
- 10. Clinical evidence of untreated hyperthyroidism or hypothyroidism
- 11. History of type 1 diabetes
- 12. Known active hepatitis B or C (participants with resolved or cured infection are eligible for enrollment).
- 13. Known HIV infection.
- 14. History, within the previous 6 months, of nonreversible cardiomyopathy (eg, reversible cardiomyopathy examples include Takotsubo cardiomyopathy, viral cardiomyopathy, ischemic mitral regurgitation), untreated or uncontrolled cardiac arrhythmia, stroke, MI, or ACS
- 15. Chronic kidney disease, defined as eGFR ≤ 45 mL/min/1.73 m2, undergoing renal dialysis, or status post kidney transplant.
- 16. Abnormal liver function tests at screening, defined as ALT or AST > 2 x ULN or total bilirubin > 2 x ULN (or > 3 × ULN if known Gilbert’s Disease).
- 17. Malignancy within 5 years or ongoing malignancy, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- 18. Known hypersensitivity to the study drug (acoramidis or placebo to match) or any of the excipients within.
- 19. Female participants who are pregnant or breastfeeding. Females must agree to discontinue breastfeeding before study drug is administered. At Screening, a negative serum pregnancy test must be confirmed at a maximum of 14 days prior to first dose. NOTE: A negative dipstick urine pregnancy test is also required before any radionuclide cardiac amyloid imaging with SPECT, on Day 1 prior to dosing, and at every In-clinic Visit for WOCBP. A positive urine dipstick pregnancy test will need to be confirmed with a serum test
- 2. Evidence of ATTR-PN (including autonomic neuropathy) by examination or skin biopsy.
- 20. 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
- 21. Participation in another investigational clinical trial within 30 days prior to Screening or within 5 half-lives of any non-ATTR investigational agent (or within the timeframe specified in Exclusion Criterion #7 for ATTR investigational agents) whichever is longer. Participation in observational and/or registry studies must be discussed with the Medical Monitor.
- 22. 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.
- 23. Major surgery as defined by the Investigator within the past 3 months or planned during the next 12 months.
- 3. Known history of AL amyloidosis or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
- 4. History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, MGUS) in which AL has not been ruled out.
- 5. Pre-existing diagnosis of axonal neuropathy from a non-amyloid cause (eg, established diagnosis of diabetic peripheral neuropathy, presence of alcohol-related neuropathy)
- 6. Exclusion Criterion 6 removed in Protocol Amendment 6.0.
- 7. Contraindication to or inability to undergo CMR testing
- 8. Presence of condition known to generate false positive myocardial radionuclide uptake with SPECT imaging (eg, ApoA-1 amyloidosis, chronic hydroxychloroquine use).
- 9. Comorbidity or condition that is likely to result in a life expectancy of < 10 years based on clinical judgment of the Investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Korea, Republic of
- Argentina
- Taiwan
- United Kingdom
- Australia
- New Zealand
- Mexico
- United States
- Canada
- Malaysia
- Japan
- Singapore
- Brazil
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Korea, Republic of; Argentina; Taiwan; United Kingdom; Australia; New Zealand and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.