Transthyretin-type Cardiac AmyloidosisPhase 4ATTR-CMNCT07306949
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
A Phase 4 study testing Acoramidis in people with Transthyretin-type Cardiac Amyloidosis, run by Alexion Pharmaceuticals, Inc. at 16 sites in Japan.
Trial summary
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Transthyretin-type Cardiac Amyloidosis
- What is being tested?
- Acoramidis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 16 sitesJapan
- Study dates
- 1 Oct 2025to 31 Mar 2028
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Treatment history of ATTR-CM is one of the following:
- Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
- Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
- Naive participants must meet the following requirements:
- History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
- Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)
Show all 9Show fewer
- Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods
- Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
- Bone scintigraphy showing strong accumulation ** (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis
Who cannot join
- Have confirmed diagnosis of AL amyloidosis
- Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
- Likelihood of receiving a heart transplant within 1 year from the time screening begins
- Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
- Pregnant or lactating women
- Has a clinically significant medical condition, an abnormal laboratory test result, or a condition that may jeopardize the safety of the study participant, increase the risk of participation in the post-marketing clinical trial, or affect the study
- Participating in an interventional study other than this study, including a clinical trial
- In the opinion of the responsible (sub)physician for the post-marketing clinical trial, has a history of drug abuse, alcoholism, or psychiatric disorder that would preclude compliance with this Post-Marketing Clinical Study Protocol
Trial locations and contacts
16 sites in 1 country. Contact the study team or a site near you to ask about taking part.
16 sites in 1 country
Japan16 sites, 16 recruiting
- Not yet recruiting
Research Site
Bunkyō City, 113-8431
- Not yet recruiting
Research Site
Bunkyō City, 113-8603
- Not yet recruiting
Research Site
Kumamoto, 860-8556
- Recruiting
Research Site
Kurume-shi, 830-0011
- Not yet recruiting
Research Site
Kyoto, 606-8507
- Not yet recruiting
Research Site
Mitaka-shi, 181-8611
- Not yet recruiting
Research Site
Nagoya, 466-8560
- Not yet recruiting
Research Site
Nankoku-shi, 783-8505
- Not yet recruiting
Research Site
Okayama, 700-8558
- Not yet recruiting
Research Site
Ōtsu, 520-2192
- Not yet recruiting
Research Site
Sagamihara-shi, 252-0375
- Not yet recruiting
Research Site
Sapporo, 060-8543
- Not yet recruiting
Research Site
Shinjuku-ku, 160-8582
- Not yet recruiting
Research Site
Suita-shi, 564-8565
- Not yet recruiting
Research Site
Tsu, 514-8507
- Recruiting
Research Site
Yufu-shi, 879-5593
About this study
Official title: A Multicenter, Open-Label, Single Arm, Post-Marketing Clinical Study to Evaluate the Therapeutic Effects of Acoramidis Hydrochlorideon Cardiac Biomarkers and Current Clinical Features in Patients With Transthyretin-type Cardiac Amyloidosis: The CARE ATTR Study
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Conditions
- Transthyretin-type Cardiac Amyloidosis
- Being tested
- Acoramidis
- Keywords
- ATTR-CM, NT-proBNP, ODI, hs-cTnT, Acoramidis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AcoramidisDrug
Also called acoramidis hydrochloride Byontra
Participants will receive acoramidis tablets orally.
| Study group | Role | What they receive |
|---|---|---|
| Acoramidis | Experimental | Participants will receive 800 mg of acoramidis twice daily for 18 months. Receives: Drug: Acoramidis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI)
Measured: Baseline through 12 months
Secondary outcomes (6)
Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT)
Measured: Baseline through 12 months
Change from Baseline in Serum Transthyretin (TTR)
Measured: Baseline, 28 days
Number of Participants with a 30% Change from Baseline in NT-proBNP
Measured: Baseline, 12 months and 18 months
Change from Baseline in hs-CTnT
Measured: Baseline, 12 months and 18 months
Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score
Measured: Baseline, 12 months and 18 months
Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Measured: Baseline, 12 months and 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2025
- Study start1 Oct 2025
- Primary completion31 Mar 2028 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin-type Cardiac AmyloidosisStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07306949
- Sponsor's protocol ID
- D9400L00001
- Other IDs
- 2025-RWE-000214
- Acronym
- ATTR-CM
- Status last verified
- Jan 2026
- First submitted
- 15 Dec 2025
- Last updated
- 7 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label, Single Arm, Post-Marketing Clinical Study to Evaluate the Therapeutic Effects of Acoramidis Hydrochlorideon Cardiac Biomarkers and Current Clinical Features in Patients With Transthyretin-type Cardiac Amyloidosis: The CARE ATTR Study
- ClinicalTrials.gov ID
- NCT07306949
- Acronym / sponsor ID
- ATTR-CM / D9400L00001
- Conditions
- Transthyretin-type Cardiac Amyloidosis
- Keywords
- ATTR-CM, NT-proBNP, ODI, hs-cTnT, Acoramidis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Oct 2025 (actual)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 29 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Acoramidis | Experimental | Drug: AcoramidisParticipants will receive 800 mg of acoramidis twice daily for 18 months. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Treatment history of ATTR-CM is one of the following:
- Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
- Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
- Naive participants must meet the following requirements:
Key exclusion criteria (8 listed)
- Have confirmed diagnosis of AL amyloidosis
- Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
- Likelihood of receiving a heart transplant within 1 year from the time screening begins
- Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
- Pregnant or lactating women
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AcoramidisAlso: acoramidis hydrochloride ByontraParticipants will receive acoramidis tablets orally. | Drug | Acoramidis |
Primary outcome
Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI)
Time frame: Baseline through 12 months
6 secondary outcomes
Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT)
Time frame: Baseline through 12 months
Change from Baseline in Serum Transthyretin (TTR)
Time frame: Baseline, 28 days
Number of Participants with a 30% Change from Baseline in NT-proBNP
Time frame: Baseline, 12 months and 18 months
Change from Baseline in hs-CTnT
Time frame: Baseline, 12 months and 18 months
Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score
Time frame: Baseline, 12 months and 18 months
Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline, 12 months and 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2026.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Acoramidis in people with Transthyretin-type Cardiac Amyloidosis, run by Alexion Pharmaceuticals, Inc. at 16 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alexion Pharmaceuticals, Inc. (Sponsor) (1-855-752-2356, clinicaltrials@alexion.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI) (time frame: Baseline through 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jan 2026.