Acoramidis to slow progression of transthyretin cardiac amyloidosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 4 interventional study gives acoramidis to adults with ATTR-CM who are treatment‑naive or currently treated with tafamidis and considered for switching. Participants will be followed over time using a composite disease progression index and serial cardiac biomarkers including NT-proBNP and high-sensitivity troponin T (hs-cTnT). The primary aim is to confirm that acoramidis prevents deterioration of the progression index and to determine whether these indexes function as surrogate markers of clinical worsening. The trial is being conducted at multiple research sites in Japan and is sponsored by Alexion Pharmaceuticals.

Why it matters

Who can join: the full rules

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

Where it's running

• Research Site
  Kurume-shi, Japan Recruiting
• Research Site
  Yufu-shi, Japan Recruiting
• Research Site
  Bunkyō City, Japan Not yet recruiting
• Research Site
  Bunkyō City, Japan Not yet recruiting
• Research Site
  Kumamoto, Japan Not yet recruiting
• Research Site
  Kyoto, Japan Not yet recruiting

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 68Ga-FAPI-46 PET imaging to stage ATTR-CM and spot progression

  This study tests whether a gallium-68 labeled FAPI PET scan can help stage transthyretin cardiac amyloidosis and identify patients whose disease is progressing.

  Recruiting Phase 2 Nantes University Hospital · Nantes · NCT07549841

• AI-powered ECG tool to detect transthyretin cardiac amyloidosis early.

  This project will test whether the Willem™ AI ECG platform can tell people with transthyretin cardiac amyloidosis from those without using routine 12‑lead digital ECG recordings.

  Recruiting at 3 sites Idoven 1903 S.L. · Evanston, Illinois and 7 other locations · NCT06978660

• Personalized exercise program for patients with transthyretin cardiac amyloidosis

  This study is testing a personalized exercise program to see if it can help people with transthyretin cardiac amyloidosis feel stronger and improve their quality of life.

  Recruiting Brigham and Women's Hospital · Boston, Massachusetts · NCT05797857

• Liver and Coagulation Markers in ATTR Cardiac Amyloidosis

  This study tests liver function and blood-clotting markers in people with ATTR-CA to see if they reflect disease severity and progression.

  Recruiting University of Messina · Messina, Sicily · NCT07766135

• Acoramidis for ATTR-CM: Cardiac Imaging Study

  This study tests whether acoramidis can improve heart structure and function in people with transthyretin amyloid cardiomyopathy.

  Recruiting Phase 4 Eidos Therapeutics, a BridgeBio company · St Louis, Missouri · NCT07695701

• Long-term safety of eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplant or severe liver impairment

  This study will see if eplontersen remains safe over time for adults with transthyretin amyloidosis, including people who have had a liver transplant or have severe liver problems.

  Recruiting at 6 sites AstraZeneca · Los Angeles, California and 5 other locations · NCT07654855

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.