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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-10-01

Study Completion Date

2028-03-31

Brief Summary

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The purpose of this study is to confirm that the treatment with acolamidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.

Detailed Description

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Conditions

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Transthyretin-type Cardiac Amyloidosis

Keywords

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ATTR-CM NT-proBNP ODI hs-cTnT Acolamidis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Acolamidis

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

Group Type EXPERIMENTAL

Acolamidis

Intervention Type DRUG

Participants will receive acolamidis tablets orally.

Interventions

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Acolamidis

Participants will receive acolamidis tablets orally.

Intervention Type DRUG

Other Intervention Names

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Acolamidis hydrochloride Byontra

Eligibility Criteria

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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: Participantswho 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 acolamidis.
* 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 acolamidis, 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

Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Locations

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