NCT07306949 Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
| NCT ID | NCT07306949 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Alexion Pharmaceuticals, Inc. |
| Condition | Transthyretin-type Cardiac Amyloidosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 200 participants |
| Start Date | 2025-10-01 |
| Primary Completion | 2028-03-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 200 participants in total. It began in 2025-10-01 with a primary completion date of 2028-03-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * 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: 1. History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics 2. Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm) 3. Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods 1. Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry. 2. Bone scintigraphy showing strong accumulation \*\* (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis Exclusion Criteria: * 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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07306949 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 100 Years, studying Transthyretin-type Cardiac Amyloidosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07306949 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT07306949 currently recruiting?
Yes, NCT07306949 is actively recruiting participants. Contact the research team at clinicaltrials@alexion.com for enrollment information.
Where is the NCT07306949 trial being conducted?
This trial is being conducted at Bunkyō City, Japan, Bunkyō City, Japan, Kumamoto, Japan, Kurume-shi, Japan and 11 additional locations.
Who is sponsoring the NCT07306949 clinical trial?
NCT07306949 is sponsored by Alexion Pharmaceuticals, Inc.. The trial plans to enroll 200 participants.