Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy (ATTR-ACT)

Pfizer

Status and phase: Completed; Phase 3

Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy

Treatments: Drug: Placebo; Drug: Tafamidis

Study type: Interventional

Funder types: Industry

Identifiers

NCT01994889

2012-002465-35 (EudraCT Number)

B3461028

ATTR-ACT (Other Identifier)

Details and patient eligibility

About

This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

Full description

Phase 3, multicenter, global, three-arm, parallel design, placebo-controlled, double-blind, randomized study to determine efficacy, safety and tolerability of tafamidis on clinical outcomes (all-cause mortality and frequency of cardiovascular-related hospitalizations) in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

Enrollment: 441 patients

Sex: All

Ages: 18 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Medical history of Heart Failure (HF) with at least 1 prior hospitalization for HF or clinical evidence of HF (without hospitalization) manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that required/requires treatment with a diuretic for improvement,
• Evidence of cardiac involvement by echocardiography with an end-diastolic interventricular septal wall thickness > 12 mm,
• Presence of amyloid deposits in biopsy tissue and presence of a variant TTR genotype and/or TTR precursor protein identification by immunohistochemistry, scintigraphy or mass spectrometry

Exclusion criteria

• A New York Heart Association (NYHA) classification of IV.
• Presence of primary (light chain) amyloidosis.
• Prior liver or heart transplantation or implanted cardiac mechanical assist device.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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441 participants in 3 patient groups, including a placebo group

Tafamidis - 20 mg

Experimental group

Description:

Active Treatment-Low dose

Treatment:

Drug: Tafamidis

Drug: Tafamidis

Tafamidis - 80 mg

Experimental group

Description:

Active Treatment-High Dose

Treatment:

Drug: Tafamidis

Drug: Tafamidis

Placebo

Placebo Comparator group

Description:

Placebo control

Treatment:

Drug: Placebo