Phase 3
N=441
Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
Transthyretin (TTR) Amyloid Cardiomyopathy
Bottom Line
View on ClinicalTrials.gov: NCT01994889 ↗Enrolled (actual)
441
Serious AEs
76.9%
Results posted
Apr 2019
Primary outcome: Primary: Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations — 8595; 5071 wins — p=0.0006
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Tafamidis (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Feb 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations |
8595; 5071 | 0.0006 sig |
| SECONDARY All-Cause Mortality |
69; 72; 7; 4; 2; 0 | 0.0259 sig |
| SECONDARY Frequency of Cardiovascular-Related Hospitalizations |
0.999; 0.884 | <0.0001 sig |
| SECONDARY Change From Baseline in the Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 30 |
350.55; 353.26; -30.46; -89.67 | <.0001 sig |
| SECONDARY Change From Baseline in Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) at Month 30 |
67.274; 65.898; -3.855; -14.637 | <.0001 sig |
| SECONDARY Number of Participants With Cardiovascular-Related Mortality |
64; 63 | 0.0383 sig |
| SECONDARY Percentage of Participants With Stabilized Transthyretin (TTR) at Month 1 |
86.1; 3.5 | <0.0001 sig |
Summary
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01994889). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.