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

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ClinicalTrials.gov Identifier: NCT01994889
Recruitment Status : Completed
First Posted : November 26, 2013
Last Update Posted : May 7, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief 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.

Condition or disease Intervention/treatment Phase
Transthyretin (TTR) Amyloid Cardiomyopathy Drug: Tafamidis Drug: Placebo Phase 3

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 441 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multicenter, International, Phase 3, Double-blind, Placebo-controlled, Randomized Study To Evaluate The Efficacy, Safety, And Tolerability Of Daily Oral Dosing Of Tafamidis Meglumine (Pf-06291826) 20 Mg Or 80 Mg In Comparison To Placebo In Subjects Diagnosed With Transthyretin Cardiomyopathy (ttr-cm)
Actual Study Start Date : December 9, 2013
Actual Primary Completion Date : February 7, 2018
Actual Study Completion Date : February 7, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Tafamidis - 20 mg
Active Treatment-Low dose
Drug: Tafamidis
Tafamidis 20 mg in soft gel capsules administered once a day for 30 months

Experimental: Tafamidis - 80 mg
Active Treatment-High Dose
Drug: Tafamidis
Tafamidis 80 mg in soft gel capsules administered once a day for 30 months

Placebo Comparator: Placebo
Placebo control
Drug: Placebo
Placebo in soft gel capsules administered once a day for 30 months




Primary Outcome Measures :
  1. All-cause mortality and frequency of cardiovascular-related hospitalization [ Time Frame: From Baseline to Month 30 ]
    A hierarchical combination of the endpoints for a pooled analysis of the tafamidis treatment groups in comparison to placebo


Secondary Outcome Measures :
  1. 6-Minute Walk Test (6MWT). [ Time Frame: From Baseline to Month 30 ]
    Change from Baseline to Month 30 in the total distance walked in 6 minutes.

  2. Kansas City Cardiomyopathy Questionnaire (KCCQ) [ Time Frame: From Baseline to Month 30 ]
    Change from Baseline to Month 30 in the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS).

  3. Cardiovascular-related mortality [ Time Frame: From Baseline to Month 30 ]
    The total number of deaths adjudicated as being related to cardiovascular causes.

  4. Frequency of cardiovascular-related hospitalization [ Time Frame: From Baseline to Month 30 ]
    The number of times that a subject is hospitalized for cardiovascular-related causes.

  5. All-cause mortality [ Time Frame: From Baseline to Month 30 ]
    The total number of deaths in the study.

  6. TTR stabilization at Month 1 [ Time Frame: From Baseline to Month 1 ]
    Tafamidis stabilizes the transthyretin (TTR) tetramer by binding with very high affinity to the two thyroxine binding sites, preventing the tetramer from dissociating along the weak dimer-dimer interface. TTR stabilization is a measure of the degree of stabilization afforded the TTR molecule by tafamidis, expressed as a percentage change from baseline.



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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01994889


  Show 54 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01994889     History of Changes
Other Study ID Numbers: B3461028
2012-002465-35 ( EudraCT Number )
ATTR-ACT ( Other Identifier: Alias Study Number )
First Posted: November 26, 2013    Key Record Dates
Last Update Posted: May 7, 2018
Last Verified: May 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Pfizer:
amyloidosis
amyloid
transthyretin
TTR

Additional relevant MeSH terms:
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Cardiomyopathies
Amyloidosis
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases