Transthyretin-type Cardiac Amyloidosis
Conditions
Keywords
ATTR-CM, NT-proBNP, ODI, hs-cTnT, Acoramidis
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.
Interventions
DRUGAcoramidis
Participants will receive acoramidis tablets orally.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| 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 Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score | Baseline, 12 months and 18 months |
| Change from Baseline in Estimated Glomerular Filtration Rate (eGFR) | Baseline, 12 months and 18 months |
| Change from Baseline in hs-CTnT | Baseline, 12 months and 18 months |
Countries
Japan
Contacts
Primary ContactAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed