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Trial record 20 of 359 for:    transthyretin

The Effects Of Fx-1006A On Transthyretin Stabilization And Clinical Outcome Measures In Patients With V122I Or Wild-Type TTR Amyloid Cardiomyopathy

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ClinicalTrials.gov Identifier: NCT00694161
Recruitment Status : Completed
First Posted : June 10, 2008
Results First Posted : December 18, 2012
Last Update Posted : January 11, 2013
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cardiomyopathy
Intervention Drug: Fx-1006A
Enrollment 35
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Tafamidis
Hide Arm/Group Description Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Period Title: Part 1 (up to Week 6)
Started 35
Completed 35
Not Completed 0
Period Title: Part 2 (After Week 6 up to Month 12)
Started 35
Completed 32
Not Completed 3
Reason Not Completed
Adverse Event             1
Physician Decision             1
Death             1
Arm/Group Title Tafamidis
Hide Arm/Group Description Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Baseline Participants 35
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 35 participants
76.4  (4.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
Female
3
   8.6%
Male
32
  91.4%
1.Primary Outcome
Title Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Week 6
Hide Description TTR tetramer was assessed using a validated immunoturbidimetric assay. The Fraction of Initial (FOI) is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI.
Time Frame Week 6
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all participants who received at least one dose of study medication.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
97.1
(85.1 to 99.9)
2.Secondary Outcome
Title Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Month 6 and 12
Hide Description TTR tetramer was assessed using a validated immunoturbidimetric assay. The Fraction of Initial (FOI) is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI.
Time Frame Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
Month 6
88.2
(72.5 to 96.7)
Month 12
87.5
(71.0 to 96.5)
3.Other Pre-specified Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (AEs)
Hide Description An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state.
Time Frame Baseline up to 30 days after the last dose
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Measure Type: Number
Unit of Measure: Participants
35
4.Other Pre-specified Outcome
Title Number of Participants With Greater Than or Equal to (>=) Grade 3 Treatment-Emergent AEs
Hide Description An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state. On the basis of intensity, grade 3 was referred as severe, grade 4 as life-threatening and grade 5 as death.
Time Frame Baseline up to 30 days after the last dose
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Measure Type: Number
Unit of Measure: Participants
Grade 3 (severe) 18
Grade 4 (life-threatening) 1
Grade 5 (death) 2
5.Other Pre-specified Outcome
Title Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings
Hide Description ECHO:investigator assessed test to assess cardiac function.ECHO abnormality criteria:any/valvular abnormality,pericardial effusion,abnormal regional wall motion,inferior vena cava respiratory variation,posterior left ventricular wall/septal thickness>=13 millimeter(mm),right ventricular thickness>=7mm,ejection fraction <50%, ratio of early (E) diastolic transmitral flow and atrial(A) contraction velocity (E/A)>=2, ratio of ‘E’to lateral/septal mitral annular velocity (e’) (E/e’prime lateral>15, E/e’prime septal>15), E deceleration time<=150 millisecond(msec),Isovolumic relaxation time<=70msec.
Time Frame Baseline up to Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. The ‘n’ for any post-dose incidence included participants with baseline values that were not abnormal(that is treatment-emergent abnormalities).
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Measure Type: Number
Unit of Measure: Participants
Any ECHO abnormalities (n=34) 31
Pericardial effusion (n=28) 6
Valvular abnormalities- thickening (n=4) 4
Valvular abnormalities- regurgitation (n=7) 6
Abnormal regional wall motion (n=19) 10
Inferior vena cava respiratory variation (n=14) 7
Left ventricular posterior wall thickness (n=0) NA [1] 
Left ventricular septal thickness (n=0) NA [1] 
Right ventricular thickness (n=7) 6
E/A ratio (n=8) 4
E/e' prime lateral (n=13) 5
E/e' prime septal (n=6) 5
Ejection fraction (n=18) 11
E deceleration time (n=16) 8
Isovolumic relaxation time (n=16) 4
[1]
Data not analyzed since no participant were eligible for analysis.
6.Other Pre-specified Outcome
Title Number of Participants Discontinuing From The Study Due to Clinically Significant Clinical or Laboratory Adverse Events (AEs)
Hide Description [Not Specified]
Time Frame Baseline up to Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Measure Type: Number
Unit of Measure: Participants
1
7.Other Pre-specified Outcome
Title Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12
Hide Description Echocardiography was used to measure interventricular septal thickness (IVST), posterior left ventricular wall thickness (PLVWT), right ventricular wall thickness (RVWT), left atrial diameter (LAD): anterior-posterior (ant-post), medio-lateral, superior-inferior (sup-inf) and left ventricular end diastolic diameter (LVEDD).
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: millimeter (mm)
IVST: Baseline (n=34) 20.71  (3.58)
IVST: Change at Month 6 (n=33) -0.64  (2.41)
IVST: Change at Month 12 (n=30) 0.87  (2.52)
PLVWT: Baseline (n=34) 20.20  (3.37)
PLVWT: Change at Month 6 (n=33) -0.50  (3.83)
PLVWT: Change at Month 12 (n=30) -0.00  (2.11)
RVWT: Baseline (n=27) 9.21  (2.34)
RVWT: Change at Month 6 (n=21) -0.47  (2.47)
RVWT: Change at Month 12 (n=20) 0.26  (2.25)
LAD (ant-post): Baseline (n=34) 45.00  (6.14)
LAD (ant-post): Change at Month 6 (n=33) -0.40  (4.33)
LAD (ant-post): Change at Month 12 (n=30) -0.30  (4.19)
LAD (medio-lateral): Baseline (n=34) 44.10  (5.56)
LAD (medio-lateral): Change at Month 6 (n=32) 0.70  (5.17)
LAD (medio-lateral): Change at Month 12 (n=30) -0.90  (6.41)
LAD (sup-inf): Baseline (n=34) 61.50  (8.43)
LAD (sup-inf): Change at Month 6 (n=32) 3.00  (5.90)
LAD (sup-inf): Change at Month 12 (n=30) 0.80  (6.29)
LVEDD: Baseline (n=34) 37.94  (5.19)
LVEDD: Change at Month 6 (n=33) 0.45  (3.49)
LVEDD: Change at Month 12 (n=30) 0.10  (3.41)
8.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricular Mass (LVM) at Month 6 and 12
Hide Description LVM was defined as increase in the mass of left ventricle, estimated by echocardiography. Increased LVM was associated with cardiovascular morbidity and mortality.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: Gram
Baseline (n=34) 372.50  (123.96)
Change at Month 6 (n=33) -19.76  (54.38)
Change at Month 12 (n=30) 13.73  (77.12)
9.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12
Hide Description Left ventricular ejection fraction (LVEF) was the fraction of the end-diastolic volume (EDV) that is ejected out of left ventricle with each contraction, estimated by echocardiography. EDV is the volume of blood within a ventricle immediately before a contraction.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: Percentage of EDV
Baseline (n=34) 46.8  (14.1)
Change at Month 6 (n=33) -0.4  (10.3)
Change at Month 12 (n=30) -3.7  (10.4)
10.Other Pre-specified Outcome
Title Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12
Hide Description Doppler echocardiography was a procedure which used ultrasound technology to examine the heart. Ratio of early (E) diastolic transmitral flow velocity and atrial (A) contraction velocity (E/A) and ratio of the early (E) diastolic transmitral flow velocity to the mitral annular velocity (e’) (E/e') were estimated.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: Ratio
E/A ratio: Baseline (n=19) 2.516  (1.084)
E/A ratio: Change at Month 6 (n=12) 0.425  (1.117)
E/A ratio: Change at Month 12 (n=9) 0.544  (0.575)
E/e’ ratio: Baseline (n=26) 16.240  (9.089)
E/e’ ratio: Change at Month 6 (n=15) 2.217  (3.166)
E/e’ ratio: Change at Month 12 (n=14) -0.717  (5.681)
11.Other Pre-specified Outcome
Title Change From Baseline in Doppler Data: Mitral Deceleration Time at Month 6 and 12
Hide Description Doppler echocardiography was a procedure which used ultrasound technology to examine the heart. The mitral deceleration time was the time taken from the maximum E wave to baseline. E wave arises due to early diastolic filling.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 33
Mean (Standard Deviation)
Unit of Measure: msec
Baseline (n=33) 152.9  (35.3)
Change at Month 6 (n=27) 1.0  (37.5)
Change at Month 12 (n=27) -7.1  (32.6)
12.Other Pre-specified Outcome
Title Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12
Hide Description Tissue Doppler used doppler principles to measure the annular velocities at the lateral and septal areas of the mitral annulus. s’: systolic velocity during ejection, e’: early diastolic mitral annular velocity, a’: late diastolic mitral annular velocity.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific categories.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: centimeter/second (cm/sec)
Septal s’: Baseline (n=23) 3.70  (1.31)
Septal s’: Change at Month 6 (n=16) -0.57  (0.73)
Septal s’: Change at Month 12 (n=14) -0.69  (0.77)
Septal e’: Baseline (n=23) 3.67  (1.60)
Septal e’: Change at Month 6 (n=17) -0.09  (1.12)
Septal e’: Change at Month 12 (n=14) -0.33  (1.32)
Septal a’: Baseline (n=16) 2.51  (1.60)
Septal a’: Change at Month 6 (n=6) -0.40  (0.43)
Septal a’: Change at Month 12 (n=6) -0.42  (0.72)
Lateral s’: Baseline (n=24) 4.53  (1.59)
Lateral s’: Change at Month 6 (n=14) -0.50  (1.16)
Lateral s’: Change at Month 12 (n=13) -0.73  (1.22)
Lateral e’: Baseline (n=26) 5.09  (2.00)
Lateral e’: Change at Month 6 (n=16) -0.76  (0.80)
Lateral e’: Change at Month 12 (n=14) 0.05  (0.88)
Lateral a’: Baseline (n=17) 3.16  (1.94)
Lateral a’: Change at Month 6 (n=7) -0.41  (0.60)
Lateral a’: Change at Month 12 (n=9) 0.06  (1.03)
13.Other Pre-specified Outcome
Title Change From Baseline in Pericardial Effusion at Month 6 and 12
Hide Description Pericardial effusion was the presence of an abnormal amount of fluid in the pericardial cavity, as determined by echocardiography.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
14.Other Pre-specified Outcome
Title Number of Participants With Change From Baseline in Valvular Abnormalities at Month 6 and 12
Hide Description Valvular abnormalities were those abnormalities (thickening or regurgitation) that involved one or more valves of the heart, determined by echocardiography.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
15.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricular Anteroseptal, Left Ventricular Inferolateral Wall Thickness and Right Ventricular End Diastolic Free Wall Thickness at Month 6 and 12
Hide Description Cardiac Magnetic Resonance Imaging (MRI) was done to measure the thickness of left ventricular anteroseptal (LVAS) wall, left ventricular inferolateral (LVIL) wall and right ventricular end diastolic free (RVEDF) wall.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: mm
LVAS wall thickness: Baseline (n=18) 15.808  (3.603)
LVAS wall thickness: Change at Month 6 (n=18) 0.810  (3.447)
LVAS wall thickness: Change at Month 12 (n=15) 0.813  (3.099)
LVIL wall thickness: Baseline (n=18) 15.405  (4.616)
LVIL wall thickness: Change at Month 6 (n=18) -1.472  (4.333)
LVIL wall thickness: Change at Month 12 (n=15) 1.182  (4.782)
RVEDF wall thickness: Baseline (n=17) 10.871  (14.001)
RVEDF wall thickness: Change at Month 6 (n=17) -4.501  (14.734)
RVEDF wall thickness: Change at Month 12 (n=15) -1.577  (4.722)
16.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricular Mass, Mass of Left Ventricular Myocardium With Amyloidosis, Mass of Left Ventricular Myocardium With Fibrosis/Scar and Right Ventricular End Diastolic Mass at Month 6 and 12
Hide Description Cardiac MRI was done to measure LVM, mass of left ventricular (LV) myocardium with amyloidosis, mass of LV myocardium with fibrosis/scar and right ventricular end diastolic mass (RVEDM).
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: Gram
LVM: Baseline (n=18) 221.599  (63.680)
LVM: Change at Month 6 (n=18) 0.557  (24.901)
LVM: Change at Month 12 (n=15) 0.119  (20.027)
LV Amyloidosis: Baseline (n=15) 63.963  (25.614)
LV Amyloidosis : Change at Month 6 (n=14) -13.854  (23.921)
LV Amyloidosis: Change at Month 12 (n=9) -4.346  (38.364)
LV Fibrosis/Scar: Baseline (n=15) 87.746  (60.297)
LV Fibrosis/Scar: Change at Month 6 (n=14) -30.496  (52.176)
LV Fibrosis/Scar: Change at Month 12 (n=9) -20.238  (64.487)
RVEDM: Baseline (n=18) 65.999  (20.296)
RVEDM: Change at Month 6 (n=18) -4.377  (20.648)
RVEDM: Change at Month 12 (n=15) 0.215  (18.038)
17.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricle End Diastolic Volume, Left Ventricle End Systolic Volume, Left Ventricle Stroke Volume, Right Ventricle End Diastolic Volume, Right Ventricle End Systolic Volume, Right Ventricle Stroke Volume at Month 6 and 12.
Hide Description Cardiac MRI was done to measure left ventricle end diastolic volume (LVEDV), left ventricle end systolic volume (LVESV), left ventricle stroke volume (LVSV), right ventricle end diastolic volume (RVEDV), right ventricle end systolic volume (RVESV) and right ventricle stroke volume (RVSV).
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: mL
LVEDV: Baseline (n=18) 166.673  (40.417)
LVEDV: Change at Month 6 (n=18) -0.922  (21.164)
LVEDV: Change at Month 12 (n=15) -1.606  (20.323)
LVESV: Baseline (n=18) 84.651  (29.271)
LVESV: Change at Month 6 (n=18) 3.727  (20.963)
LVESV: Change at Month 12 (n=15) 6.479  (16.810)
LVSV: Baseline (n=18) 82.022  (19.866)
LVSV: Change at Month 6 (n=18) -4.651  (21.391)
LVSV: Change at Month 12 (n=15) -8.083  (15.614)
RVEDV: Baseline (n=18) 175.124  (67.977)
RVEDV: Change at Month 6 (n=18) -4.836  (37.879)
RVEDV: Change at Month 12 (n=15) 19.540  (39.413)
RVESV: Baseline (n=18) 104.662  (48.775)
RVESV: Change at Month 6 (n=18) -1.727  (21.438)
RVESV: Change at Month 12 (n=15) 26.250  (30.858)
RVSV: Baseline (n=18) 70.478  (26.364)
RVSV: Change at Month 6 (n=18) -3.125  (26.125)
RVSV: Change at Month 12 (n=15) -6.730  (33.975)
18.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12
Hide Description Cardiac MRI was done to measure: left ventricular ejection fraction (LVEF) was the fraction of the EDV that is ejected out of left ventricle with each contraction and right ventricular ejection fraction (RVEF) was the fraction of the EDV that is ejected out of right ventricle with each contraction. EDV is the volume of blood within a ventricle immediately before a contraction.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: Percentage of EDV
LVEF: Baseline (n=18) 50.176  (9.183)
LVEF: Change at Month 6 (n=18) -2.713  (13.362)
LVEF: Change at Month 12 (n=14) -3.511  (9.063)
RVEF: Baseline (n=18) 42.323  (11.949)
RVEF: Change at Month 6 (n=18) -1.756  (10.381)
RVEF: Change at Month 12 (n=15) -7.381  (12.040)
19.Other Pre-specified Outcome
Title Change From Baseline in Left Ventricular Cardiac Output and Right Ventricular Cardiac Output at Month 6 and 12
Hide Description Cardiac MRI was done to measure cardiac output, which was the volume of blood being pumped by the heart, in particular by the left or right ventricle in the time interval of one minute.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
MRI data was collected and reported for cardiac output through the measure of stroke volume as given in outcome measure 17.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
20.Other Pre-specified Outcome
Title Change From Baseline in Percentage of Left Ventricular Myocardial Mass With Amyloidosis and Left Ventricular Myocardial Mass With Fibrosis/Scar at Month 6 and 12
Hide Description Cardiac MRI was done to measure percentage of LV myocardial mass with amyloidosis and LV myocardial mass with fibrosis/scar. LV myocardial mass with amyloidosis or fibrosis/scar was calculated from the product of the myocardial volume and specific gravity of heart muscle, in participants with amyloidosis or fibrosis/scar, respectively.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percentage of LVM
LV Amyloidosis: Baseline (n=15) 29.069  (11.111)
LV Amyloidosis: Change at Month 6 (n=14) -8.200  (14.115)
LV Amyloidosis: Change at Month 12 (n=9) -7.017  (13.660)
LV Fibrosis/Scar: Baseline (n=15) 36.513  (17.789)
LV Fibrosis/Scar: Change at Month 6 (n=14) -10.126  (26.397)
LV Fibrosis/Scar: Change at Month 12 (n=9) -8.268  (24.884)
21.Other Pre-specified Outcome
Title Change From Baseline in 4 Chamber Interatrial Septal Thickness at Month 6 and 12
Hide Description Cardiac MRI was done to measure interatrial septal thickness in the 4 chamber view.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Data for this measure was not collected due to a change in the planned analysis.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
22.Other Pre-specified Outcome
Title Change From Baseline in 4 Chamber Left Atrial Dimension and 4 Chamber Right Atrial Dimension at Month 6 and 12
Hide Description Cardiac MRI was done to measure the left and right atrial dimensions which have diagnostic and prognostic significance in cardiology, in the 4 chamber view.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Data for this measure was not collected due to a change in the planned analysis.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
23.Other Pre-specified Outcome
Title Number of Participants With Atrial Fibrillation/Flutter, Atrial Tachycardia, Non-Sustained Ventricular Tachycardia (NSVT) Beats), Sustained Ventricular Tachycardia (SVT), Sinus Pause at Month 6 and 12
Hide Description Holter monitor was a machine that recorded the heart rhythms. Holter monitoring abnormalities of atrial fibrillation/flutter (rapid, irregular heart rhythm), atrial tachycardia (rapid cardiac rate), non-sustained ventricular tachycardia (NSVT)<30 beats, sustained ventricular tachycardia (SVT) >=30 beats and sinus pause (transient interruption in the sinus rhythm) were recorded.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. The ‘n’ for any post-dose incidence included participants with baseline values that were not abnormal(that is treatment-emergent abnormalities).
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Measure Type: Number
Unit of Measure: Participants
Atrial fibrillation/flutter: Baseline (n=33) 6
Atrial fibrillation/flutter: Month 6 (n=27) 9
Atrial fibrillation/flutter: Month 12 (n=22) 9
Atrial tachycardia: Baseline (n=34) 14
Atrial tachycardia: Month 6 (n=20) 0
Atrial tachycardia: Month 12 (n=17) 0
NSVT (<30 beats): Baseline (n=34) 20
NSVT (<30 beats): Month 6 (n=13) 3
NSVT (<30 beats): Month 12 (n=12) 5
SVT (>= 30 beats): Baseline (n=34) 0
SVT (>= 30 beats): Month 6 (n=33) 0
SVT (>= 30 beats): Month 12 (n=30) 1
Sinus pause: Baseline (n=34) 2
Sinus pause: Month 6 (n=31) 4
Sinus pause: Month 12 (n=28) 5
24.Other Pre-specified Outcome
Title 24-Hour Average Heart Rate and Maximium/Minimum Heart Rate
Hide Description Holter monitor was a machine that recorded the heart rhythms. 24-hour average heart rate and maximium/minimum heart rate was recorded using Holter monitoring.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: beats per minute (bpm)
24-hour average heart rate: Baseline (n=34) 74.7  (7.8)
24-hour average heart rate: Month 6 (n=33) 73.0  (7.2)
24-hour average heart rate: Month 12 (n=30) 76.6  (9.0)
Maximum Heart Rate: Baseline (n=34) 111.7  (22.3)
Maximum Heart Rate: Month 6 (n=33) 112.3  (17.0)
Maximum Heart Rate: Month 12 (n=30) 121.9  (20.4)
Minimum Heart Rate: Baseline (n=34) 56.6  (8.0)
Minimum Heart Rate: Month 6: (n=33) 53.1  (11.5)
Minimum Heart Rate Month 12: (n=30) 55.3  (10.1)
25.Other Pre-specified Outcome
Title Number of Participants With Complete Heart Block
Hide Description Complete heart block is the third-degree atrioventricular block in which the impulse generated in the sinoatrial node in the atrium does not propagate to the ventricles.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
No summary was prepared for this data as there were no reports of complete heart block.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
26.Other Pre-specified Outcome
Title Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters
Hide Description Holter monitor was a machine that recorded the heart rhythms. HRV time-domain indices were summarized for root-mean-square of successive differences [RMS SD] of the R-R intervals (R-R is the interval between successive Rs in the ECG wave) between normal beats (NN), magid standard deviation (Magid SD) of normal to normal R-R intervals and Kleiger standard deviation of normal to normal R-R intervals (Kleiger SD). The term ‘NN’ is used in place of ‘R-R’ when the processed beats are normal beats.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 33
Mean (Standard Deviation)
Unit of Measure: msec
HRV- RMS SD: Baseline (n=30) 75.9  (71.0)
HRV- RMS SD: Month 6 (n=26) 88.1  (71.0)
HRV- RMS SD: Month 12 (n=21) 91.7  (72.1)
HRV- Magid SD: Baseline (n=31) 58.3  (42.1)
HRV- Magid SD: Month 6 (n=33) 82.6  (55.5)
HRV- Magid SD: Month 12 (n=30) 78.6  (50.1)
HRV- Kleiger SD: Baseline (n=31) 100.3  (49.1)
HRV- Kleiger SD: Month 6 (n=33) 123.9  (52.8)
HRV- Kleiger SD: Month 12 (n=30) 120.3  (48.7)
27.Other Pre-specified Outcome
Title Heart Rate Variability- Percentage of Successive R-R Intervals With Greater Than 50 Msec Difference Between Normal Beats (pNN50)
Hide Description Holter monitor was a machine that recorded the heart rhythms. The term ‘NN’ was used in place of ‘R-R’ when the processed beats are normal beats. The percentage of successive R-R intervals with greater than 50 msec difference between normal beats was derived by dividing NN50 by the total number of NN intervals (pNN50), where NN50 was the number of interval differences of successive NN intervals greater than 50 msec.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 30
Mean (Standard Deviation)
Unit of Measure: Percentage of intervals
Baseline (n=30) 19.7  (25.5)
Month 6 (n=26) 29.3  (32.7)
Month 12 (n=21) 23.6  (27.3)
28.Other Pre-specified Outcome
Title Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12
Hide Description NYHA: classified as ‘class I’ (participants with cardiac disease but without resulting limitations of physical activity), ‘class II’ (participants with cardiac disease resulting in slight limitation of physical activity), ‘class III’ (participants with cardiac disease resulting in marked limitation of physical activity), ‘class IV’ (participants with cardiac disease resulting in inability to carry on any physical activity without discomfort). Participants with change from baseline were classified as ‘improved' (positive change), ‘no change’ or ‘worsened' (negative change).
Time Frame Baseline, Week 6, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Data at Week 6 was collected but was not summarized due to a change in the planned analysis. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Measure Type: Number
Unit of Measure: Participants
Baseline: Class I (n=35) 5
Baseline: Class II (n=35) 28
Baseline: Class III (n=35) 2
Baseline: Class IV (n=35) 0
Change at Month 3: improved (n=34) 5
Change at Month 3: no change (n=34) 23
Change at Month 3: worsened (n=34) 6
Change at Month 6: improved (n=34) 1
Change at Month 6: no change (n=34) 27
Change at Month 6: worsened (n=34) 6
Change at Month 12: improved (n=32) 4
Change at Month 12: no change (n=32) 20
Change at Month 12: worsened (n=32) 8
29.Other Pre-specified Outcome
Title Cardiothoracic (CT) Ratio
Hide Description Cardiothoracic ratio was defined as the transverse diameter of the heart, compared with that of the thoracic cage, used to help determine enlargement of the heart.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Mean (Standard Deviation)
Unit of Measure: Ratio
Baseline 55.130  (6.075)
Month 6 56.620  (5.904)
Month 12 57.830  (5.366)
30.Other Pre-specified Outcome
Title Number of Participants With Increased Interstitial Markings and Pleural Effusions
Hide Description Chest x-ray was done to record the presence of increased interstitial markings (a large number of interstitial markings was indicative of abnormality in the lung) and pleural effusion, which was defined as accumulation of fluid between the layers of tissue that line the lungs and chest cavity.
Time Frame Baseline, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT) population included all participants who received at least one dose of study medication.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Measure Type: Number
Unit of Measure: Participants
Increased interstitial markings: Baseline 11
Increased interstitial markings: Month 6 4
Increased interstitial markings: Month 12 4
Pleural effusion- right: Baseline 9
Pleural effusion- right: Month 6 8
Pleural effusion- right: Month 12 6
Pleural effusion- left: Baseline 6
Pleural effusion- left: Month 6 5
Pleural effusion- left: Month 12 4
Pleural effusion- bilateral: Baseline 6
Pleural effusion- bilateral: Month 6 4
Pleural effusion- bilateral: Month 12 3
31.Other Pre-specified Outcome
Title Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12
Hide Description Participant’s overall quality of life was measured by the PtGA. At baseline participants answered to question: "in general, how do you feel today?" - on a 5-point scale from '1' (excellent) to '5' (poor). At each follow-up visit, participant’s answered to question: “How do you feel today as compared to when we talked with you at your last clinic visit for this study?” on a 7-point scale- '1' markedly improved, '2' moderately improved, '3' mildly improved, '4' unchanged, '5' mildly worsened, '6' moderately worsened, '7' markedly worsened.
Time Frame Baseline, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Measure Type: Number
Unit of Measure: Participants
Excellent: Baseline (n=33) 5
Very good: Baseline (n=33) 14
Good: Baseline (n=33) 12
Fair: Baseline (n=33) 2
Poor: Baseline (n=33) 0
Markedly improved: Month 3 (n=34) 2
Moderately improved: Month 3 (n=34) 7
Mildly improved: Month 3 (n=34) 6
Unchanged: Month 3 (n=34) 15
Mildly worsened: Month 3 (n=34) 2
Moderately worsened: Month 3 (n=34) 2
Markedly worsened: Month 3 (n=34) 0
Markedly improved: Month 6 (n=34) 3
Moderately improved: Month 6 (n=34) 4
Mildly improved: Month 6 (n=34) 3
Unchanged: Month 6 (n=34) 16
Mildly worsened: Month 6 (n=34) 8
Moderately worsened: Month 6 (n=34) 0
Markedly worsened: Month 6 (n=34) 0
Markedly improved: Month 12 (n=32) 0
Moderately improved: Month 12 (n=32) 5
Mildly improved: Month 12 (n=32) 3
Unchanged: Month 12 (n=32) 16
Mildly worsened: Month 12 (n=32) 4
Moderately worsened: Month 12 (n=32) 3
Markedly worsened: Month 12 (n=32) 1
32.Other Pre-specified Outcome
Title Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12
Hide Description KCCQ was a 23-item heart failure specific questionnaire quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life. Summary scores were scaled to range from 0 to 100, with higher scores representing greater disability.
Time Frame Baseline, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Physical limitations: Baseline (n=35) 72.9  (20.8)
Physical limitations: Change at Month 3 (n=33) -2.7  (15.0)
Physical limitations: Change at Month 6 (n=34) -0.4  (13.6)
Physical limitations: Change at Month 12 (n=31) -8.1  (18.8)
Symptom stability: Baseline (n=35) 55.7  (13.7)
Symptom stability: Change at Month 3 (n=34) 3.7  (27.6)
Symptom stability: Change at Month 6 (n=34) -5.9  (25.4)
Symptom stability: Change at Month 12 (n=31) 0.0  (22.4)
Symptom frequency: Baseline (n=35) 73.8  (22.2)
Symptom frequency: Change at Month 3 (n=34) -1.8  (18.5)
Symptom frequency: Change at Month 6 (n=34) -2.5  (15.9)
Symptom frequency: Change at Month 12 (n=31) -6.0  (18.2)
Symptom burden: Baseline (n=35) 76.9  (18.1)
Symptom burden: Change at Month 3 (n=34) -4.0  (18.3)
Symptom burden: Change at Month 6 (n=34) -4.7  (15.2)
Symptom burden: Change at Month 12 (n=31) -8.6  (19.2)
Total symptom: Baseline (n=35) 75.4  (19.2)
Total symptom: Change at Month 3 (n=34) -2.9  (16.9)
Total symptom: Change at Month 6 (n=34) -3.6  (14.3)
Total symptom: Change at Month 12 (n=31) -7.3  (18.2)
Self-efficacy: Baseline (n=35) 85.7  (19.0)
Self-efficacy: Change at Month 3 (n=33) 1.9  (14.7)
Self-efficacy: Change at Month 6 (n=34) 1.1  (13.5)
Self-efficacy: Change at Month 12 (n=31) 1.6  (16.7)
Quality of life: Baseline (n=35) 66.9  (19.2)
Quality of life: Change at Month 3 (n=34) 1.0  (18.2)
Quality of life: Change at Month 6 (n=34) -0.2  (14.4)
Quality of life: Change at Month 12 (n=31) -4.0  (20.4)
Social limitation: Baseline (n=33) 69.3  (25.4)
Social limitation: Change at Month 3 (n=30) -0.6  (19.6)
Social limitation: Change at Month 6 (n=31) -2.4  (22.6)
Social limitation: Change at Month 12 (n=27) -9.2  (25.2)
Overall summary: Baseline (n=35) 71.4  (18.8)
Overall summary: Change at Month 3 (n=34) -0.8  (14.9)
Overall summary: Change at Month 6 (n=34) -1.5  (12.4)
Overall summary: Change at Month 12 (n=31) -6.9  (18.0)
Clinical summary: Baseline (n=35) 74.1  (18.9)
Clinical summary: Change at Month 3 (n=34) -2.0  (15.9)
Clinical summary: Change at Month 6 (n=34) -2.0  (11.9)
Clinical summary: Change at Month 12 (n=31) -7.7  (17.9)
33.Other Pre-specified Outcome
Title Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12
Hide Description SF-36 was standardized survey evaluating 8 aspects of functional health and well being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. Scores for the 8 domains range from 0-100, where higher scores were better (100=highest level of functioning) and reported as 2 summary scores; Mental Component Score (MCS) and Physical Component Score (PCS). The score for a section was an average of the individual question scores, which were scaled 0-100, where higher scores were better.
Time Frame Baseline, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints. Data was collected at Month 3 but not statistically summarized as planned.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Mean (Standard Deviation)
Unit of Measure: Units on a scale
PCS: Baseline (n=33) 41.0  (9.69)
PCS: Change at Month 6 (n=32) -1.4  (5.87)
PCS: Change at Month 12 (n=29) -2.4  (7.58)
MCS: Baseline (n=33) 52.7  (9.51)
MCS: Change at Month 6 (n=32) 0.3  (8.40)
MCS: Change at Month 12 (n=29) -2.0  (9.56)
Physical functioning: Baseline (n=34) 37.7  (11.28)
Physical functioning: Change at Month 6 (n=33) -0.7  (6.89)
Physical functioning: Change at Month 12 (n=29) -2.0  (7.34)
Role-physical: Baseline (n=35) 42.3  (10.84)
Role-physical: Change at Month 6 (n=34) -1.4  (7.12)
Role-physical: Change at Month 12 (n=30) -3.5  (9.29)
Bodily pain: Baseline (n=35) 53.2  (9.57)
Bodily pain: Change at Month 6 (n=34) -4.0  (8.74)
Bodily pain: Change at Month 12 (n=30) -3.2  (11.81)
General health: Baseline (n=35) 41.5  (9.33)
General health: Change at Month 6 (n=34) 0.7  (9.23)
General health: Change at Month 12 (n=30) -1.6  (8.44)
Vitality: Baseline (n=34) 50.6  (8.11)
Vitality: Change at Month 6 (n=33) -0.1  (6.83)
Vitality: Change at Month 12 (n=30) -2.2  (7.83)
Social functioning: Baseline (n=35) 48.4  (9.84)
Social functioning: Change at Month 6 (n=34) -2.9  (9.23)
Social functioning: Change at Month 12 (n=30) -3.6  (11.40)
Role-emotional: Baseline (n=35) 45.2  (11.71)
Role-emotional: Change at Month 6 (n=34) 0.6  (12.26)
Role-emotional: Change at Month 12 (n=30) -3.0  (12.53)
Mental health: Baseline (n=34) 53.3  (8.50)
Mental health: Change at Month 6 (n=33) 0.3  (5.01)
Mental health: Change at Month 12 (n=30) -1.1  (7.50)
34.Other Pre-specified Outcome
Title Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12
Hide Description Troponin I and troponin T were the cardiac markers. Troponin I and troponin T were part of the troponin complex, where troponin I was bound to actin in thin myofilaments and troponin T was bound to tropomyosin. Higher level of these markers was indicative of heart damage.
Time Frame Baseline, Week 2, Week 6, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Mean (Standard Deviation)
Unit of Measure: nanogram/milliliter (ng/mL)
Troponin I: Baseline (n=33) 0.135  (0.080)
Troponin I: Change at Week 2 (n=35) 0.003  (0.060)
Troponin I: Change at Week 6 (n=35) -0.000  (0.055)
Troponin I: Change at Month 3 (n=34) 0.008  (0.057)
Troponin I: Change at Month 6 (n=34) -0.016  (0.051)
Troponin I: Change at Month 12 (n=32) 0.016  (0.064)
Troponin T: Baseline (n=33) 0.044  (0.037)
Troponin T: Change at Week 2 (n=35) 0.001  (0.025)
Troponin T: Change at Week 6 (n=35) 0.006  (0.020)
Troponin T: Change at Month 3 (n=34) 0.008  (0.021)
Troponin T: Change at Month 6 (n=33) 0.002  (0.020)
Troponin T: Change at Month 12 (n=32) 0.012  (0.023)
35.Other Pre-specified Outcome
Title Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12
Hide Description NT-proBNP was a cardiac marker which had the prognostic value for participants with heart failure or left ventricular dysfunction. Higher level of the marker was indicative of heart damage.
Time Frame Baseline, Week 2, Week 6, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 35
Mean (Standard Deviation)
Unit of Measure: picogram/mL (pg/mL)
Baseline (n=33) 4934.2  (4324.9)
Change at Week 2 (n=35) -1.6  (1349.1)
Change at Week 6 (n=35) 295.0  (1632.0)
Change at Month 3 (n=34) 102.8  (1998.0)
Change at Month 6 (n=33) 111.6  (2032.4)
Change at Month 12 (n=31) 958.2  (3178.0)
36.Other Pre-specified Outcome
Title Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12
Hide Description 6MWT was used to assess the distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed. Continuous pulse oximetry was conducted during the test for safety.. The distance walked in 6 minutes was categorized as: Level 1: <300 meter, Level 2: 300-374.9 meter, Level 3: 375-449.9 meter, Level 4: >=450 meter.
Time Frame Baseline, Month 3, Month 6, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints.
Arm/Group Title Tafamidis
Hide Arm/Group Description:
Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: meters
Distance walked: Baseline (n=34) 354.5  (126.0)
Distance walked: Change at Month 3 (n=31) -4.6  (63.1)
Distance walked: Change at Month 6 (n=33) 3.9  (58.2)
Distance walked: Change at Month 12 (n=28) -11.2  (76.4)
Time Frame [Not Specified]
Adverse Event Reporting Description The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
 
Arm/Group Title Tafamidis
Hide Arm/Group Description Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
All-Cause Mortality
Tafamidis
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Tafamidis
Affected / at Risk (%)
Total   15/35 (42.86%) 
Cardiac disorders   
Cardiac failure congestive * 1  9/35 (25.71%) 
Atrial fibrillation * 1  3/35 (8.57%) 
Cardiac failure * 1  2/35 (5.71%) 
Supraventricular tachycardia * 1  1/35 (2.86%) 
Ventricular tachycardia * 1  1/35 (2.86%) 
Immune system disorders   
Amyloidosis * 1  1/35 (2.86%) 
Infections and infestations   
Cellulitis * 1  1/35 (2.86%) 
Escherichia sepsis * 1  1/35 (2.86%) 
Pneumonia * 1  1/35 (2.86%) 
Injury, poisoning and procedural complications   
Fall * 1  3/35 (8.57%) 
Pelvic fracture * 1  1/35 (2.86%) 
Metabolism and nutrition disorders   
Dehydration * 1  1/35 (2.86%) 
Hypokalaemia * 1  1/35 (2.86%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Glioblastoma multiforme * 1  1/35 (2.86%) 
Nervous system disorders   
Syncope * 1  2/35 (5.71%) 
Cerebrovascular accident * 1  1/35 (2.86%) 
Coordination abnormal * 1  1/35 (2.86%) 
Haemorrhagic stroke * 1  1/35 (2.86%) 
Metabolic encephalopathy * 1  1/35 (2.86%) 
Renal and urinary disorders   
Renal failure acute * 1  1/35 (2.86%) 
Renal impairment * 1  1/35 (2.86%) 
Vascular disorders   
Haemorrhage * 1  1/35 (2.86%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA v 10.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Tafamidis
Affected / at Risk (%)
Total   34/35 (97.14%) 
Blood and lymphatic system disorders   
Anaemia * 1  1/35 (2.86%) 
Cardiac disorders   
Atrial fibrillation * 1  4/35 (11.43%) 
Cardiac failure congestive * 1  3/35 (8.57%) 
Cardiac failure * 1  2/35 (5.71%) 
Atrial tachycardia * 1  1/35 (2.86%) 
Bradycardia * 1  1/35 (2.86%) 
Bundle branch block right * 1  1/35 (2.86%) 
Palpitations * 1  1/35 (2.86%) 
Sinus arrhythmia * 1  1/35 (2.86%) 
Ventricular tachycardia * 1  1/35 (2.86%) 
Bundle branch block * 1  1/35 (2.86%) 
Congenital, familial and genetic disorders   
Colour blindness * 1  1/35 (2.86%) 
Eye disorders   
Corneal disorder * 1  1/35 (2.86%) 
Vitreous floaters * 1  1/35 (2.86%) 
Gastrointestinal disorders   
Diarrhoea * 1  7/35 (20.00%) 
Constipation * 1  5/35 (14.29%) 
Nausea * 1  5/35 (14.29%) 
Abdominal pain upper * 1  3/35 (8.57%) 
Flatulence * 1  3/35 (8.57%) 
Dry mouth * 1  2/35 (5.71%) 
Vomiting * 1  2/35 (5.71%) 
Abdominal pain * 1  1/35 (2.86%) 
Cheilitis * 1  1/35 (2.86%) 
Faecal incontinence * 1  1/35 (2.86%) 
Inguinal hernia * 1  1/35 (2.86%) 
General disorders   
Fatigue * 1  8/35 (22.86%) 
Oedema peripheral * 1  6/35 (17.14%) 
Asthenia * 1  3/35 (8.57%) 
Oedema * 1  3/35 (8.57%) 
Thirst * 1  3/35 (8.57%) 
Catheter related complication * 1  1/35 (2.86%) 
Chest discomfort * 1  1/35 (2.86%) 
Chest pain * 1  1/35 (2.86%) 
Hernia * 1  1/35 (2.86%) 
Hot flush * 1  1/35 (2.86%) 
Localised oedema * 1  1/35 (2.86%) 
Pain * 1  1/35 (2.86%) 
Pyrexia * 1  1/35 (2.86%) 
Hepatobiliary disorders   
Hepatomegaly * 1  4/35 (11.43%) 
Immune system disorders   
Seasonal allergy * 1  3/35 (8.57%) 
Infections and infestations   
Upper respiratory tract infection * 1  6/35 (17.14%) 
Urinary tract infection * 1  3/35 (8.57%) 
Influenza * 1  2/35 (5.71%) 
Nasopharyngitis * 1  2/35 (5.71%) 
Bronchitis * 1  1/35 (2.86%) 
Cellulitis * 1  1/35 (2.86%) 
Furuncle * 1  1/35 (2.86%) 
Infection * 1  1/35 (2.86%) 
Lower respiratory tract infection * 1  1/35 (2.86%) 
Nail infection * 1  1/35 (2.86%) 
Injury, poisoning and procedural complications   
Contusion * 1  2/35 (5.71%) 
Excoriation * 1  2/35 (5.71%) 
Fall * 1  2/35 (5.71%) 
Joint injury * 1  2/35 (5.71%) 
Skin laceration * 1  2/35 (5.71%) 
Joint sprain * 1  1/35 (2.86%) 
Laceration * 1  1/35 (2.86%) 
Post procedural haematuria * 1  1/35 (2.86%) 
Tooth injury * 1  1/35 (2.86%) 
Investigations   
Weight decreased * 1  6/35 (17.14%) 
Work increased * 1  5/35 (14.29%) 
Alanine aminotransferase increased * 1  2/35 (5.71%) 
Aspartate aminotransferase increased * 1  2/35 (5.71%) 
Blood cholesterol increased * 1  2/35 (5.71%) 
Blood thyroid stimulating hormone increased * 1  2/35 (5.71%) 
Aspiration pleural cavity * 1  1/35 (2.86%) 
Blood creatinine increased * 1  1/35 (2.86%) 
Blood pressure decreased * 1  1/35 (2.86%) 
Blood thyroid stimulating hormone decreased * 1  1/35 (2.86%) 
Blood urine present * 1  1/35 (2.86%) 
Blood natriuretic peptide increased * 1  1/35 (2.86%) 
Ejection fraction decreased * 1  1/35 (2.86%) 
Gamma-glutamyltransferase increased * 1  1/35 (2.86%) 
Heart rate increased * 1  1/35 (2.86%) 
International normalised ratio increased * 1  1/35 (2.86%) 
Urine output increased * 1  1/35 (2.86%) 
Venous pressure jugular increased * 1  1/35 (2.86%) 
Metabolism and nutrition disorders   
Anorexia * 1  2/35 (5.71%) 
Decreased appetite * 1  2/35 (5.71%) 
Fluid overload * 1  2/35 (5.71%) 
Gout * 1  2/35 (5.71%) 
Fluid retention * 1  1/35 (2.86%) 
Hyperkalaemia * 1  1/35 (2.86%) 
Hypochloraemia * 1  1/35 (2.86%) 
Hyponatraemia * 1  1/35 (2.86%) 
Musculoskeletal and connective tissue disorders   
Back pain * 1  3/35 (8.57%) 
Pain in extremity * 1  3/35 (8.57%) 
Muscle spasms * 1  2/35 (5.71%) 
Arthralgia * 1  1/35 (2.86%) 
Finger deformity * 1  1/35 (2.86%) 
Joint swelling * 1  1/35 (2.86%) 
Muscle twitching * 1  1/35 (2.86%) 
Musculoskeletal discomfort * 1  1/35 (2.86%) 
Musculoskeletal pain * 1  1/35 (2.86%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Basal cell carcinoma * 1  1/35 (2.86%) 
Benign neoplasm * 1  1/35 (2.86%) 
Lentigo * 1  1/35 (2.86%) 
Prostate cancer * 1  1/35 (2.86%) 
Nervous system disorders   
Dizziness * 1  7/35 (20.00%) 
Dizziness postural * 1  6/35 (17.14%) 
Balance disorder * 1  5/35 (14.29%) 
Aguesia * 1  3/35 (8.57%) 
Tremor * 1  2/35 (5.71%) 
Amnesia * 1  1/35 (2.86%) 
Disturbance in attention * 1  1/35 (2.86%) 
Headache * 1  1/35 (2.86%) 
Loss of consciousness * 1  1/35 (2.86%) 
Memory impairment * 1  1/35 (2.86%) 
Migraine * 1  1/35 (2.86%) 
Neuralgia * 1  1/35 (2.86%) 
Neuropathy * 1  1/35 (2.86%) 
Neuropathy peripheral * 1  1/35 (2.86%) 
Restless legs syndrome * 1  1/35 (2.86%) 
Somnolence * 1  1/35 (2.86%) 
Syncope * 1  1/35 (2.86%) 
Psychiatric disorders   
Insomnia * 1  3/35 (8.57%) 
Confusional state * 1  2/35 (5.71%) 
Depression * 1  2/35 (5.71%) 
Anxiety * 1  1/35 (2.86%) 
Nervousness * 1  1/35 (2.86%) 
Panic attack * 1  1/35 (2.86%) 
Renal and urinary disorders   
Haematuria * 1  2/35 (5.71%) 
Dysuria * 1  1/35 (2.86%) 
Pollakiuria * 1  1/35 (2.86%) 
Urinary incontinence * 1  1/35 (2.86%) 
Reproductive system and breast disorders   
Nipple pain * 1  2/35 (5.71%) 
Gynaecomastia * 1  1/35 (2.86%) 
Scrotal oedema * 1  1/35 (2.86%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnoea * 1  11/35 (31.43%) 
Dyspnoea exertional * 1  6/35 (17.14%) 
Epistaxis * 1  3/35 (8.57%) 
Rhinorrhoea * 1  3/35 (8.57%) 
Cough * 1  2/35 (5.71%) 
Pleural effusion * 1  2/35 (5.71%) 
Sinus congestion * 1  2/35 (5.71%) 
Dysphonia * 1  1/35 (2.86%) 
Postnasal drip * 1  1/35 (2.86%) 
Productive cough * 1  1/35 (2.86%) 
Pulmonary congestion * 1  1/35 (2.86%) 
Rales * 1  1/35 (2.86%) 
Skin and subcutaneous tissue disorders   
Pruritus * 1  3/35 (8.57%) 
Rash * 1  3/35 (8.57%) 
Skin ulcer * 1  3/35 (8.57%) 
Blister * 1  1/35 (2.86%) 
Ingrowing nail * 1  1/35 (2.86%) 
Rash maculo-papular * 1  1/35 (2.86%) 
Skin discolouration * 1  1/35 (2.86%) 
Skin exfoliation * 1  1/35 (2.86%) 
Skin lesion * 1  1/35 (2.86%) 
Surgical and medical procedures   
Foot operation * 1  1/35 (2.86%) 
Implantable defibrillator insertion * 1  1/35 (2.86%) 
Mole excision * 1  1/35 (2.86%) 
Tooth extraction * 1  1/35 (2.86%) 
Vascular disorders   
Hypotension * 1  1/35 (2.86%) 
Varicose vein * 1  1/35 (2.86%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA v 10.0
Results for Holter monitoring parameters, increased interstitial markings, pleural effusions, PtGA and cardiothoracic ratio are presented as absolute values at specified time points and not as change from baseline as planned.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00694161     History of Changes
Other Study ID Numbers: FX1B-201
B3461025
First Submitted: June 6, 2008
First Posted: June 10, 2008
Results First Submitted: November 16, 2012
Results First Posted: December 18, 2012
Last Update Posted: January 11, 2013