Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers

NCT07306949 | Recruiting Phase 4

• 2 other identifiers: D9400L000012025-RWE-000214
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 16

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.

Conditions

Transthyretin-type Cardiac Amyloidosis

Keywords

ATTR-CM; NT-proBNP; ODI; hs-cTnT; Acoramidis

Outcome Measures

Primary Outcomes (1)

• Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI)

  Baseline through 12 months

Secondary Outcomes (6)

• Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT)

  Baseline through 12 months

• Change from Baseline in Serum Transthyretin (TTR)

  Baseline, 28 days

• Number of Participants with a 30% Change from Baseline in NT-proBNP

  Baseline, 12 months and 18 months

• Change from Baseline in hs-CTnT

  Baseline, 12 months and 18 months

• Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score

  Baseline, 12 months and 18 months

Study Arms (1)

Acoramidis

EXPERIMENTAL

Participants will receive 800 mg of acoramidis twice daily for 18 months.

Drug: Acoramidis

Interventions

Acoramidis DRUG

Participants will receive acoramidis tablets orally.

Also known as: acoramidis hydrochloride Byontra

Acoramidis

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (16)

Research Site

Bunkyō City, 113-8431, Japan

NOT YET RECRUITING

Research Site

Bunkyō City, 113-8603, Japan

NOT YET RECRUITING

Research Site

Kumamoto, 860-8556, Japan

NOT YET RECRUITING

Research Site

Kurume-shi, 830-0011, Japan

RECRUITING

Research Site

Kyoto, 606-8507, Japan

NOT YET RECRUITING

Research Site

Mitaka-shi, 181-8611, Japan

NOT YET RECRUITING

Research Site

Nagoya, 466-8560, Japan

NOT YET RECRUITING

Research Site

Nankoku-shi, 783-8505, Japan

NOT YET RECRUITING

Research Site

Okayama, 700-8558, Japan

NOT YET RECRUITING

Research Site

Ōtsu, 520-2192, Japan

NOT YET RECRUITING

Research Site

Sagamihara-shi, 252-0375, Japan

NOT YET RECRUITING

Research Site

Sapporo, 060-8543, Japan

NOT YET RECRUITING

Research Site

Shinjuku-ku, 160-8582, Japan

NOT YET RECRUITING

Research Site

Suita-shi, 564-8565, Japan

NOT YET RECRUITING

Research Site

Tsu, 514-8507, Japan

NOT YET RECRUITING

Research Site

Yufu-shi, 879-5593, Japan

RECRUITING

MeSH Terms

Interventions

attruby

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2025
• First Posted: December 29, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): March 31, 2028
• Study Completion (Estimated): March 31, 2028
• Last Updated: January 7, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

JP (16)