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

ConditionTransthyretin-type Cardiac Amyloidosis
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18-100
SponsorAlexion Pharmaceuticals, Inc.

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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.

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)

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Acoramidis

    Drug

    Participants will receive acoramidis tablets orally.

Treatment groups

200 Participants
are divided into 1 treatment group
Group A: AcoramidisExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
Baseline through 12 months

Secondary outcomes

1

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

Time frame
Baseline through 12 months
2

Change from Baseline in Serum Transthyretin (TTR)

Time frame
Baseline, 28 days
3

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

Time frame
Baseline, 12 months and 18 months
4

Change from Baseline in hs-CTnT

Time frame
Baseline, 12 months and 18 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alexion Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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