Trial Outcomes & Findings for The Effects Of Fx-1006A On Transthyretin Stabilization And Clinical Outcome Measures In Patients With V122I Or Wild-Type TTR Amyloid Cardiomyopathy (NCT NCT00694161)

Last Updated: 2013-01-11

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

35 participants

Primary outcome timeframe

Week 6

Results posted on

2013-01-11

Participant Flow

Participant milestones

Participant milestones
Measure	Tafamidis 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.
Part 1 (up to Week 6) STARTED	35
Part 1 (up to Week 6) COMPLETED	35
Part 1 (up to Week 6) NOT COMPLETED	0
Part 2 (After Week 6 up to Month 12) STARTED	35
Part 2 (After Week 6 up to Month 12) COMPLETED	32
Part 2 (After Week 6 up to Month 12) NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Tafamidis 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.
Part 2 (After Week 6 up to Month 12) Adverse Event	1
Part 2 (After Week 6 up to Month 12) Physician Decision	1
Part 2 (After Week 6 up to Month 12) Death	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tafamidis n=35 Participants 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.
Age Continuous	76.4 Years STANDARD_DEVIATION 4.7 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	32 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 6

Population: Intent-to-Treat (ITT) population included all participants who received at least one dose of study medication.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Week 6	97.1 Percentage of participants Interval 85.1 to 99.9

SECONDARY outcome

Timeframe: Month 6, Month 12

Population: 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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Month 6 and 12 Month 6	88.2 Percentage of participants Interval 72.5 to 96.7
Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Month 6 and 12 Month 12	87.5 Percentage of participants Interval 71.0 to 96.5

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 30 days after the last dose

Population: ITT population included all participants who received at least one dose of study medication.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Number of Participants With Treatment-Emergent Adverse Events (AEs)	35 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 30 days after the last dose

Population: ITT population included all participants who received at least one dose of study medication.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Number of Participants With Greater Than or Equal to (>=) Grade 3 Treatment-Emergent AEs Grade 3 (severe)	18 Participants
Number of Participants With Greater Than or Equal to (>=) Grade 3 Treatment-Emergent AEs Grade 4 (life-threatening)	1 Participants
Number of Participants With Greater Than or Equal to (>=) Grade 3 Treatment-Emergent AEs Grade 5 (death)	2 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Month 12

Population: 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).

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Any ECHO abnormalities (n=34)	31 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Pericardial effusion (n=28)	6 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Valvular abnormalities- thickening (n=4)	4 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Valvular abnormalities- regurgitation (n=7)	6 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Abnormal regional wall motion (n=19)	10 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Inferior vena cava respiratory variation (n=14)	7 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Left ventricular posterior wall thickness (n=0)	NA Participants Data not analyzed since no participant were eligible for analysis.
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Left ventricular septal thickness (n=0)	NA Participants Data not analyzed since no participant were eligible for analysis.
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Right ventricular thickness (n=7)	6 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E/A ratio (n=8)	4 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E/e' prime lateral (n=13)	5 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E/e' prime septal (n=6)	5 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Ejection fraction (n=18)	11 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E deceleration time (n=16)	8 Participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Isovolumic relaxation time (n=16)	4 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Month 12

Population: ITT population included all participants who received at least one dose of study medication.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Number of Participants Discontinuing From The Study Due to Clinically Significant Clinical or Laboratory Adverse Events (AEs)	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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).

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 IVST: Baseline (n=34)	20.71 millimeter (mm) Standard Deviation 3.58
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 IVST: Change at Month 6 (n=33)	-0.64 millimeter (mm) Standard Deviation 2.41
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 IVST: Change at Month 12 (n=30)	0.87 millimeter (mm) Standard Deviation 2.52
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 PLVWT: Baseline (n=34)	20.20 millimeter (mm) Standard Deviation 3.37
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 PLVWT: Change at Month 6 (n=33)	-0.50 millimeter (mm) Standard Deviation 3.83
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 PLVWT: Change at Month 12 (n=30)	-0.00 millimeter (mm) Standard Deviation 2.11
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 RVWT: Baseline (n=27)	9.21 millimeter (mm) Standard Deviation 2.34
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 RVWT: Change at Month 6 (n=21)	-0.47 millimeter (mm) Standard Deviation 2.47
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 RVWT: Change at Month 12 (n=20)	0.26 millimeter (mm) Standard Deviation 2.25
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (ant-post): Baseline (n=34)	45.00 millimeter (mm) Standard Deviation 6.14
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (ant-post): Change at Month 6 (n=33)	-0.40 millimeter (mm) Standard Deviation 4.33
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (ant-post): Change at Month 12 (n=30)	-0.30 millimeter (mm) Standard Deviation 4.19
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (medio-lateral): Baseline (n=34)	44.10 millimeter (mm) Standard Deviation 5.56
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (medio-lateral): Change at Month 6 (n=32)	0.70 millimeter (mm) Standard Deviation 5.17
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (medio-lateral): Change at Month 12 (n=30)	-0.90 millimeter (mm) Standard Deviation 6.41
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (sup-inf): Baseline (n=34)	61.50 millimeter (mm) Standard Deviation 8.43
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (sup-inf): Change at Month 6 (n=32)	3.00 millimeter (mm) Standard Deviation 5.90
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LAD (sup-inf): Change at Month 12 (n=30)	0.80 millimeter (mm) Standard Deviation 6.29
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LVEDD: Baseline (n=34)	37.94 millimeter (mm) Standard Deviation 5.19
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LVEDD: Change at Month 6 (n=33)	0.45 millimeter (mm) Standard Deviation 3.49
Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 LVEDD: Change at Month 12 (n=30)	0.10 millimeter (mm) Standard Deviation 3.41

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

LVM was defined as increase in the mass of left ventricle, estimated by echocardiography. Increased LVM was associated with cardiovascular morbidity and mortality.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Change From Baseline in Left Ventricular Mass (LVM) at Month 6 and 12 Baseline (n=34)	372.50 Gram Standard Deviation 123.96
Change From Baseline in Left Ventricular Mass (LVM) at Month 6 and 12 Change at Month 6 (n=33)	-19.76 Gram Standard Deviation 54.38
Change From Baseline in Left Ventricular Mass (LVM) at Month 6 and 12 Change at Month 12 (n=30)	13.73 Gram Standard Deviation 77.12

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12 Baseline (n=34)	46.8 Percentage of EDV Standard Deviation 14.1
Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12 Change at Month 6 (n=33)	-0.4 Percentage of EDV Standard Deviation 10.3
Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12 Change at Month 12 (n=30)	-3.7 Percentage of EDV Standard Deviation 10.4

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=26 Participants 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.
Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 E/A ratio: Baseline (n=19)	2.516 Ratio Standard Deviation 1.084
Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 E/A ratio: Change at Month 6 (n=12)	0.425 Ratio Standard Deviation 1.117
Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 E/A ratio: Change at Month 12 (n=9)	0.544 Ratio Standard Deviation 0.575
Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 E/e' ratio: Baseline (n=26)	16.240 Ratio Standard Deviation 9.089
Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 E/e' ratio: Change at Month 6 (n=15)	2.217 Ratio Standard Deviation 3.166
Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 E/e' ratio: Change at Month 12 (n=14)	-0.717 Ratio Standard Deviation 5.681

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=33 Participants 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.
Change From Baseline in Doppler Data: Mitral Deceleration Time at Month 6 and 12 Baseline (n=33)	152.9 msec Standard Deviation 35.3
Change From Baseline in Doppler Data: Mitral Deceleration Time at Month 6 and 12 Change at Month 6 (n=27)	1.0 msec Standard Deviation 37.5
Change From Baseline in Doppler Data: Mitral Deceleration Time at Month 6 and 12 Change at Month 12 (n=27)	-7.1 msec Standard Deviation 32.6

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=26 Participants 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.
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal s': Baseline (n=23)	3.70 centimeter/second (cm/sec) Standard Deviation 1.31
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal s': Change at Month 6 (n=16)	-0.57 centimeter/second (cm/sec) Standard Deviation 0.73
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal s': Change at Month 12 (n=14)	-0.69 centimeter/second (cm/sec) Standard Deviation 0.77
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal e': Baseline (n=23)	3.67 centimeter/second (cm/sec) Standard Deviation 1.60
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal e': Change at Month 6 (n=17)	-0.09 centimeter/second (cm/sec) Standard Deviation 1.12
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal e': Change at Month 12 (n=14)	-0.33 centimeter/second (cm/sec) Standard Deviation 1.32
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal a': Baseline (n=16)	2.51 centimeter/second (cm/sec) Standard Deviation 1.60
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal a': Change at Month 6 (n=6)	-0.40 centimeter/second (cm/sec) Standard Deviation 0.43
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Septal a': Change at Month 12 (n=6)	-0.42 centimeter/second (cm/sec) Standard Deviation 0.72
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral s': Baseline (n=24)	4.53 centimeter/second (cm/sec) Standard Deviation 1.59
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral s': Change at Month 6 (n=14)	-0.50 centimeter/second (cm/sec) Standard Deviation 1.16
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral s': Change at Month 12 (n=13)	-0.73 centimeter/second (cm/sec) Standard Deviation 1.22
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral e': Baseline (n=26)	5.09 centimeter/second (cm/sec) Standard Deviation 2.00
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral e': Change at Month 6 (n=16)	-0.76 centimeter/second (cm/sec) Standard Deviation 0.80
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral e': Change at Month 12 (n=14)	0.05 centimeter/second (cm/sec) Standard Deviation 0.88
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral a': Baseline (n=17)	3.16 centimeter/second (cm/sec) Standard Deviation 1.94
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral a': Change at Month 6 (n=7)	-0.41 centimeter/second (cm/sec) Standard Deviation 0.60
Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 Lateral a': Change at Month 12 (n=9)	0.06 centimeter/second (cm/sec) Standard Deviation 1.03

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis.

Pericardial effusion was the presence of an abnormal amount of fluid in the pericardial cavity, as determined by echocardiography.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis.

Valvular abnormalities were those abnormalities (thickening or regurgitation) that involved one or more valves of the heart, determined by echocardiography.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=18 Participants 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.
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 LVAS wall thickness: Baseline (n=18)	15.808 mm Standard Deviation 3.603
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 LVAS wall thickness: Change at Month 6 (n=18)	0.810 mm Standard Deviation 3.447
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 LVAS wall thickness: Change at Month 12 (n=15)	0.813 mm Standard Deviation 3.099
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 LVIL wall thickness: Baseline (n=18)	15.405 mm Standard Deviation 4.616
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 LVIL wall thickness: Change at Month 6 (n=18)	-1.472 mm Standard Deviation 4.333
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 LVIL wall thickness: Change at Month 12 (n=15)	1.182 mm Standard Deviation 4.782
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 RVEDF wall thickness: Baseline (n=17)	10.871 mm Standard Deviation 14.001
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 RVEDF wall thickness: Change at Month 6 (n=17)	-4.501 mm Standard Deviation 14.734
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 RVEDF wall thickness: Change at Month 12 (n=15)	-1.577 mm Standard Deviation 4.722

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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).

Outcome measures

Outcome measures
Measure	Tafamidis n=18 Participants 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.
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 LVM: Change at Month 6 (n=18)	0.557 Gram Standard Deviation 24.901
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 LVM: Change at Month 12 (n=15)	0.119 Gram Standard Deviation 20.027
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 LV Amyloidosis: Baseline (n=15)	63.963 Gram Standard Deviation 25.614
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 LV Amyloidosis : Change at Month 6 (n=14)	-13.854 Gram Standard Deviation 23.921
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 LV Amyloidosis: Change at Month 12 (n=9)	-4.346 Gram Standard Deviation 38.364
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 LV Fibrosis/Scar: Baseline (n=15)	87.746 Gram Standard Deviation 60.297
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 LV Fibrosis/Scar: Change at Month 6 (n=14)	-30.496 Gram Standard Deviation 52.176
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 LV Fibrosis/Scar: Change at Month 12 (n=9)	-20.238 Gram Standard Deviation 64.487
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 RVEDM: Baseline (n=18)	65.999 Gram Standard Deviation 20.296
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 RVEDM: Change at Month 6 (n=18)	-4.377 Gram Standard Deviation 20.648
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 RVEDM: Change at Month 12 (n=15)	0.215 Gram Standard Deviation 18.038
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 LVM: Baseline (n=18)	221.599 Gram Standard Deviation 63.680

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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).

Outcome measures

Outcome measures
Measure	Tafamidis n=18 Participants 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.
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. LVEDV: Baseline (n=18)	166.673 mL Standard Deviation 40.417
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. LVEDV: Change at Month 6 (n=18)	-0.922 mL Standard Deviation 21.164
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. LVEDV: Change at Month 12 (n=15)	-1.606 mL Standard Deviation 20.323
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. LVESV: Baseline (n=18)	84.651 mL Standard Deviation 29.271
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. LVESV: Change at Month 6 (n=18)	3.727 mL Standard Deviation 20.963
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. LVESV: Change at Month 12 (n=15)	6.479 mL Standard Deviation 16.810
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. LVSV: Baseline (n=18)	82.022 mL Standard Deviation 19.866
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. LVSV: Change at Month 6 (n=18)	-4.651 mL Standard Deviation 21.391
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. LVSV: Change at Month 12 (n=15)	-8.083 mL Standard Deviation 15.614
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. RVEDV: Baseline (n=18)	175.124 mL Standard Deviation 67.977
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. RVEDV: Change at Month 6 (n=18)	-4.836 mL Standard Deviation 37.879
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. RVEDV: Change at Month 12 (n=15)	19.540 mL Standard Deviation 39.413
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. RVESV: Baseline (n=18)	104.662 mL Standard Deviation 48.775
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. RVESV: Change at Month 6 (n=18)	-1.727 mL Standard Deviation 21.438
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. RVESV: Change at Month 12 (n=15)	26.250 mL Standard Deviation 30.858
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. RVSV: Baseline (n=18)	70.478 mL Standard Deviation 26.364
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. RVSV: Change at Month 6 (n=18)	-3.125 mL Standard Deviation 26.125
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. RVSV: Change at Month 12 (n=15)	-6.730 mL Standard Deviation 33.975

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=18 Participants 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.
Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 LVEF: Baseline (n=18)	50.176 Percentage of EDV Standard Deviation 9.183
Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 LVEF: Change at Month 6 (n=18)	-2.713 Percentage of EDV Standard Deviation 13.362
Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 LVEF: Change at Month 12 (n=14)	-3.511 Percentage of EDV Standard Deviation 9.063
Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 RVEF: Baseline (n=18)	42.323 Percentage of EDV Standard Deviation 11.949
Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 RVEF: Change at Month 6 (n=18)	-1.756 Percentage of EDV Standard Deviation 10.381
Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 RVEF: Change at Month 12 (n=15)	-7.381 Percentage of EDV Standard Deviation 12.040

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: MRI data was collected and reported for cardiac output through the measure of stroke volume as given in outcome measure 17.

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.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=15 Participants 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.
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 LV Amyloidosis: Baseline (n=15)	29.069 Percentage of LVM Standard Deviation 11.111
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 LV Amyloidosis: Change at Month 6 (n=14)	-8.200 Percentage of LVM Standard Deviation 14.115
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 LV Amyloidosis: Change at Month 12 (n=9)	-7.017 Percentage of LVM Standard Deviation 13.660
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 LV Fibrosis/Scar: Baseline (n=15)	36.513 Percentage of LVM Standard Deviation 17.789
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 LV Fibrosis/Scar: Change at Month 6 (n=14)	-10.126 Percentage of LVM Standard Deviation 26.397
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 LV Fibrosis/Scar: Change at Month 12 (n=9)	-8.268 Percentage of LVM Standard Deviation 24.884

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: Data for this measure was not collected due to a change in the planned analysis.

Cardiac MRI was done to measure interatrial septal thickness in the 4 chamber view.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: Data for this measure was not collected due to a change in the planned analysis.

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.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
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 Atrial fibrillation/flutter: Baseline (n=33)	6 Participants
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 Atrial fibrillation/flutter: Month 6 (n=27)	9 Participants
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 Atrial fibrillation/flutter: Month 12 (n=22)	9 Participants
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 Atrial tachycardia: Baseline (n=34)	14 Participants
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 Atrial tachycardia: Month 6 (n=20)	0 Participants
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 Atrial tachycardia: Month 12 (n=17)	0 Participants
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 NSVT (<30 beats): Baseline (n=34)	20 Participants
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 NSVT (<30 beats): Month 6 (n=13)	3 Participants
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 NSVT (<30 beats): Month 12 (n=12)	5 Participants
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 SVT (>= 30 beats): Baseline (n=34)	0 Participants
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 SVT (>= 30 beats): Month 6 (n=33)	0 Participants
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 SVT (>= 30 beats): Month 12 (n=30)	1 Participants
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 Sinus pause: Baseline (n=34)	2 Participants
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 Sinus pause: Month 6 (n=31)	4 Participants
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 Sinus pause: Month 12 (n=28)	5 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate 24-hour average heart rate: Baseline (n=34)	74.7 beats per minute (bpm) Standard Deviation 7.8
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate 24-hour average heart rate: Month 6 (n=33)	73.0 beats per minute (bpm) Standard Deviation 7.2
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate 24-hour average heart rate: Month 12 (n=30)	76.6 beats per minute (bpm) Standard Deviation 9.0
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate Maximum Heart Rate: Baseline (n=34)	111.7 beats per minute (bpm) Standard Deviation 22.3
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate Maximum Heart Rate: Month 6 (n=33)	112.3 beats per minute (bpm) Standard Deviation 17.0
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate Maximum Heart Rate: Month 12 (n=30)	121.9 beats per minute (bpm) Standard Deviation 20.4
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate Minimum Heart Rate: Baseline (n=34)	56.6 beats per minute (bpm) Standard Deviation 8.0
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate Minimum Heart Rate: Month 6: (n=33)	53.1 beats per minute (bpm) Standard Deviation 11.5
24-Hour Average Heart Rate and Maximium/Minimum Heart Rate Minimum Heart Rate Month 12: (n=30)	55.3 beats per minute (bpm) Standard Deviation 10.1

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: No summary was prepared for this data as there were no reports of complete heart block.

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.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=33 Participants 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.
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- RMS SD: Baseline (n=30)	75.9 msec Standard Deviation 71.0
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- RMS SD: Month 6 (n=26)	88.1 msec Standard Deviation 71.0
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- RMS SD: Month 12 (n=21)	91.7 msec Standard Deviation 72.1
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- Magid SD: Baseline (n=31)	58.3 msec Standard Deviation 42.1
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- Magid SD: Month 6 (n=33)	82.6 msec Standard Deviation 55.5
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- Magid SD: Month 12 (n=30)	78.6 msec Standard Deviation 50.1
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- Kleiger SD: Baseline (n=31)	100.3 msec Standard Deviation 49.1
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- Kleiger SD: Month 6 (n=33)	123.9 msec Standard Deviation 52.8
Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters HRV- Kleiger SD: Month 12 (n=30)	120.3 msec Standard Deviation 48.7

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=30 Participants 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.
Heart Rate Variability- Percentage of Successive R-R Intervals With Greater Than 50 Msec Difference Between Normal Beats (pNN50) Baseline (n=30)	19.7 Percentage of intervals Standard Deviation 25.5
Heart Rate Variability- Percentage of Successive R-R Intervals With Greater Than 50 Msec Difference Between Normal Beats (pNN50) Month 6 (n=26)	29.3 Percentage of intervals Standard Deviation 32.7
Heart Rate Variability- Percentage of Successive R-R Intervals With Greater Than 50 Msec Difference Between Normal Beats (pNN50) Month 12 (n=21)	23.6 Percentage of intervals Standard Deviation 27.3

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 6, Month 3, Month 6, Month 12

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Baseline: Class I (n=35)	5 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Baseline: Class II (n=35)	28 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Baseline: Class III (n=35)	2 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Baseline: Class IV (n=35)	0 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 3: improved (n=34)	5 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 3: no change (n=34)	23 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 3: worsened (n=34)	6 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 6: improved (n=34)	1 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 6: no change (n=34)	27 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 6: worsened (n=34)	6 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 12: improved (n=32)	4 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 12: no change (n=32)	20 Participants
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 Change at Month 12: worsened (n=32)	8 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: ITT population included all participants who received at least one dose of study medication.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Cardiothoracic (CT) Ratio Month 6	56.620 Ratio Standard Deviation 5.904
Cardiothoracic (CT) Ratio Baseline	55.130 Ratio Standard Deviation 6.075
Cardiothoracic (CT) Ratio Month 12	57.830 Ratio Standard Deviation 5.366

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: Intent to Treat (ITT) population included all participants who received at least one dose of study medication.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Number of Participants With Increased Interstitial Markings and Pleural Effusions Increased interstitial markings: Baseline	11 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Increased interstitial markings: Month 6	4 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Increased interstitial markings: Month 12	4 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- right: Baseline	9 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- right: Month 6	8 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- right: Month 12	6 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- left: Baseline	6 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- left: Month 6	5 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- left: Month 12	4 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- bilateral: Baseline	6 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- bilateral: Month 6	4 Participants
Number of Participants With Increased Interstitial Markings and Pleural Effusions Pleural effusion- bilateral: Month 12	3 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 3, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Excellent: Baseline (n=33)	5 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Very good: Baseline (n=33)	14 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Good: Baseline (n=33)	12 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Fair: Baseline (n=33)	2 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Poor: Baseline (n=33)	0 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Markedly improved: Month 3 (n=34)	2 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Moderately improved: Month 3 (n=34)	7 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Mildly improved: Month 3 (n=34)	6 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Unchanged: Month 3 (n=34)	15 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Mildly worsened: Month 3 (n=34)	2 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Moderately worsened: Month 3 (n=34)	2 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Markedly worsened: Month 3 (n=34)	0 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Markedly improved: Month 6 (n=34)	3 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Moderately improved: Month 6 (n=34)	4 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Mildly improved: Month 6 (n=34)	3 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Unchanged: Month 6 (n=34)	16 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Mildly worsened: Month 6 (n=34)	8 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Moderately worsened: Month 6 (n=34)	0 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Markedly worsened: Month 6 (n=34)	0 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Markedly improved: Month 12 (n=32)	0 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Moderately improved: Month 12 (n=32)	5 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Mildly improved: Month 12 (n=32)	3 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Unchanged: Month 12 (n=32)	16 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Mildly worsened: Month 12 (n=32)	4 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Moderately worsened: Month 12 (n=32)	3 Participants
Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 Markedly worsened: Month 12 (n=32)	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 3, Month 6, Month 12

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Physical limitations: Baseline (n=35)	72.9 Units on a scale Standard Deviation 20.8
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Physical limitations: Change at Month 3 (n=33)	-2.7 Units on a scale Standard Deviation 15.0
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Physical limitations: Change at Month 6 (n=34)	-0.4 Units on a scale Standard Deviation 13.6
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Physical limitations: Change at Month 12 (n=31)	-8.1 Units on a scale Standard Deviation 18.8
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom stability: Baseline (n=35)	55.7 Units on a scale Standard Deviation 13.7
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom stability: Change at Month 3 (n=34)	3.7 Units on a scale Standard Deviation 27.6
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom stability: Change at Month 6 (n=34)	-5.9 Units on a scale Standard Deviation 25.4
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom stability: Change at Month 12 (n=31)	0.0 Units on a scale Standard Deviation 22.4
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom frequency: Baseline (n=35)	73.8 Units on a scale Standard Deviation 22.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom frequency: Change at Month 3 (n=34)	-1.8 Units on a scale Standard Deviation 18.5
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom frequency: Change at Month 6 (n=34)	-2.5 Units on a scale Standard Deviation 15.9
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom frequency: Change at Month 12 (n=31)	-6.0 Units on a scale Standard Deviation 18.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom burden: Baseline (n=35)	76.9 Units on a scale Standard Deviation 18.1
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom burden: Change at Month 3 (n=34)	-4.0 Units on a scale Standard Deviation 18.3
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom burden: Change at Month 6 (n=34)	-4.7 Units on a scale Standard Deviation 15.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Symptom burden: Change at Month 12 (n=31)	-8.6 Units on a scale Standard Deviation 19.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Total symptom: Baseline (n=35)	75.4 Units on a scale Standard Deviation 19.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Total symptom: Change at Month 3 (n=34)	-2.9 Units on a scale Standard Deviation 16.9
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Total symptom: Change at Month 6 (n=34)	-3.6 Units on a scale Standard Deviation 14.3
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Total symptom: Change at Month 12 (n=31)	-7.3 Units on a scale Standard Deviation 18.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Self-efficacy: Baseline (n=35)	85.7 Units on a scale Standard Deviation 19.0
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Self-efficacy: Change at Month 3 (n=33)	1.9 Units on a scale Standard Deviation 14.7
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Self-efficacy: Change at Month 6 (n=34)	1.1 Units on a scale Standard Deviation 13.5
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Self-efficacy: Change at Month 12 (n=31)	1.6 Units on a scale Standard Deviation 16.7
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Quality of life: Baseline (n=35)	66.9 Units on a scale Standard Deviation 19.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Quality of life: Change at Month 3 (n=34)	1.0 Units on a scale Standard Deviation 18.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Quality of life: Change at Month 6 (n=34)	-0.2 Units on a scale Standard Deviation 14.4
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Quality of life: Change at Month 12 (n=31)	-4.0 Units on a scale Standard Deviation 20.4
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Social limitation: Baseline (n=33)	69.3 Units on a scale Standard Deviation 25.4
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Social limitation: Change at Month 3 (n=30)	-0.6 Units on a scale Standard Deviation 19.6
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Social limitation: Change at Month 6 (n=31)	-2.4 Units on a scale Standard Deviation 22.6
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Social limitation: Change at Month 12 (n=27)	-9.2 Units on a scale Standard Deviation 25.2
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Overall summary: Baseline (n=35)	71.4 Units on a scale Standard Deviation 18.8
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Overall summary: Change at Month 3 (n=34)	-0.8 Units on a scale Standard Deviation 14.9
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Overall summary: Change at Month 6 (n=34)	-1.5 Units on a scale Standard Deviation 12.4
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Overall summary: Change at Month 12 (n=31)	-6.9 Units on a scale Standard Deviation 18.0
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Clinical summary: Baseline (n=35)	74.1 Units on a scale Standard Deviation 18.9
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Clinical summary: Change at Month 3 (n=34)	-2.0 Units on a scale Standard Deviation 15.9
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Clinical summary: Change at Month 6 (n=34)	-2.0 Units on a scale Standard Deviation 11.9
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 Clinical summary: Change at Month 12 (n=31)	-7.7 Units on a scale Standard Deviation 17.9

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 3, Month 6, Month 12

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 PCS: Baseline (n=33)	41.0 Units on a scale Standard Deviation 9.69
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 PCS: Change at Month 6 (n=32)	-1.4 Units on a scale Standard Deviation 5.87
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 PCS: Change at Month 12 (n=29)	-2.4 Units on a scale Standard Deviation 7.58
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 MCS: Baseline (n=33)	52.7 Units on a scale Standard Deviation 9.51
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 MCS: Change at Month 6 (n=32)	0.3 Units on a scale Standard Deviation 8.40
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 MCS: Change at Month 12 (n=29)	-2.0 Units on a scale Standard Deviation 9.56
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Physical functioning: Baseline (n=34)	37.7 Units on a scale Standard Deviation 11.28
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Physical functioning: Change at Month 6 (n=33)	-0.7 Units on a scale Standard Deviation 6.89
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Physical functioning: Change at Month 12 (n=29)	-2.0 Units on a scale Standard Deviation 7.34
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Role-physical: Baseline (n=35)	42.3 Units on a scale Standard Deviation 10.84
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Role-physical: Change at Month 6 (n=34)	-1.4 Units on a scale Standard Deviation 7.12
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Role-physical: Change at Month 12 (n=30)	-3.5 Units on a scale Standard Deviation 9.29
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Bodily pain: Baseline (n=35)	53.2 Units on a scale Standard Deviation 9.57
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Bodily pain: Change at Month 6 (n=34)	-4.0 Units on a scale Standard Deviation 8.74
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Bodily pain: Change at Month 12 (n=30)	-3.2 Units on a scale Standard Deviation 11.81
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 General health: Baseline (n=35)	41.5 Units on a scale Standard Deviation 9.33
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 General health: Change at Month 6 (n=34)	0.7 Units on a scale Standard Deviation 9.23
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 General health: Change at Month 12 (n=30)	-1.6 Units on a scale Standard Deviation 8.44
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Vitality: Baseline (n=34)	50.6 Units on a scale Standard Deviation 8.11
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Vitality: Change at Month 6 (n=33)	-0.1 Units on a scale Standard Deviation 6.83
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Vitality: Change at Month 12 (n=30)	-2.2 Units on a scale Standard Deviation 7.83
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Social functioning: Baseline (n=35)	48.4 Units on a scale Standard Deviation 9.84
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Social functioning: Change at Month 6 (n=34)	-2.9 Units on a scale Standard Deviation 9.23
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Social functioning: Change at Month 12 (n=30)	-3.6 Units on a scale Standard Deviation 11.40
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Role-emotional: Baseline (n=35)	45.2 Units on a scale Standard Deviation 11.71
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Role-emotional: Change at Month 6 (n=34)	0.6 Units on a scale Standard Deviation 12.26
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Role-emotional: Change at Month 12 (n=30)	-3.0 Units on a scale Standard Deviation 12.53
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Mental health: Baseline (n=34)	53.3 Units on a scale Standard Deviation 8.50
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Mental health: Change at Month 6 (n=33)	0.3 Units on a scale Standard Deviation 5.01
Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 Mental health: Change at Month 12 (n=30)	-1.1 Units on a scale Standard Deviation 7.50

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 2, Week 6, Month 3, Month 6, Month 12

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin I: Baseline (n=33)	0.135 nanogram/milliliter (ng/mL) Standard Deviation 0.080
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin I: Change at Week 2 (n=35)	0.003 nanogram/milliliter (ng/mL) Standard Deviation 0.060
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin I: Change at Week 6 (n=35)	-0.000 nanogram/milliliter (ng/mL) Standard Deviation 0.055
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin I: Change at Month 3 (n=34)	0.008 nanogram/milliliter (ng/mL) Standard Deviation 0.057
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin I: Change at Month 6 (n=34)	-0.016 nanogram/milliliter (ng/mL) Standard Deviation 0.051
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin I: Change at Month 12 (n=32)	0.016 nanogram/milliliter (ng/mL) Standard Deviation 0.064
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin T: Baseline (n=33)	0.044 nanogram/milliliter (ng/mL) Standard Deviation 0.037
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin T: Change at Week 2 (n=35)	0.001 nanogram/milliliter (ng/mL) Standard Deviation 0.025
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin T: Change at Week 6 (n=35)	0.006 nanogram/milliliter (ng/mL) Standard Deviation 0.020
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin T: Change at Month 3 (n=34)	0.008 nanogram/milliliter (ng/mL) Standard Deviation 0.021
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin T: Change at Month 6 (n=33)	0.002 nanogram/milliliter (ng/mL) Standard Deviation 0.020
Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 Troponin T: Change at Month 12 (n=32)	0.012 nanogram/milliliter (ng/mL) Standard Deviation 0.023

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 2, Week 6, Month 3, Month 6, Month 12

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=35 Participants 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.
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 Baseline (n=33)	4934.2 picogram/mL (pg/mL) Standard Deviation 4324.9
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 Change at Week 2 (n=35)	-1.6 picogram/mL (pg/mL) Standard Deviation 1349.1
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 Change at Week 6 (n=35)	295.0 picogram/mL (pg/mL) Standard Deviation 1632.0
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 Change at Month 3 (n=34)	102.8 picogram/mL (pg/mL) Standard Deviation 1998.0
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 Change at Month 6 (n=33)	111.6 picogram/mL (pg/mL) Standard Deviation 2032.4
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 Change at Month 12 (n=31)	958.2 picogram/mL (pg/mL) Standard Deviation 3178.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 3, Month 6, Month 12

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.

Outcome measures

Outcome measures
Measure	Tafamidis n=34 Participants 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.
Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12 Distance walked: Baseline (n=34)	354.5 meters Standard Deviation 126.0
Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12 Distance walked: Change at Month 3 (n=31)	-4.6 meters Standard Deviation 63.1
Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12 Distance walked: Change at Month 6 (n=33)	3.9 meters Standard Deviation 58.2
Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12 Distance walked: Change at Month 12 (n=28)	-11.2 meters Standard Deviation 76.4

Adverse Events

Tafamidis

Serious events: 15 serious events

Other events: 34 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tafamidis n=35 participants at risk 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.
Cardiac disorders Cardiac failure congestive	25.7% 9/35 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.
Cardiac disorders Atrial fibrillation	8.6% 3/35 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.
Cardiac disorders Cardiac failure	5.7% 2/35 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.
Cardiac disorders Supraventricular tachycardia	2.9% 1/35 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.
Cardiac disorders Ventricular tachycardia	2.9% 1/35 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.
Immune system disorders Amyloidosis	2.9% 1/35 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.
Infections and infestations Cellulitis	2.9% 1/35 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.
Infections and infestations Escherichia sepsis	2.9% 1/35 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.
Infections and infestations Pneumonia	2.9% 1/35 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.
Injury, poisoning and procedural complications Fall	8.6% 3/35 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.
Injury, poisoning and procedural complications Pelvic fracture	2.9% 1/35 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.
Metabolism and nutrition disorders Dehydration	2.9% 1/35 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.
Metabolism and nutrition disorders Hypokalaemia	2.9% 1/35 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Glioblastoma multiforme	2.9% 1/35 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.
Nervous system disorders Syncope	5.7% 2/35 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.
Nervous system disorders Cerebrovascular accident	2.9% 1/35 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.
Nervous system disorders Coordination abnormal	2.9% 1/35 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.
Nervous system disorders Haemorrhagic stroke	2.9% 1/35 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.
Nervous system disorders Metabolic encephalopathy	2.9% 1/35 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.
Renal and urinary disorders Renal failure acute	2.9% 1/35 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.
Renal and urinary disorders Renal impairment	2.9% 1/35 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.
Vascular disorders Haemorrhage	2.9% 1/35 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.

Other adverse events

Other adverse events
Measure	Tafamidis n=35 participants at risk 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.
Investigations Blood cholesterol increased	5.7% 2/35 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.
Investigations Blood thyroid stimulating hormone increased	5.7% 2/35 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.
Investigations Aspiration pleural cavity	2.9% 1/35 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.
Investigations Blood creatinine increased	2.9% 1/35 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.
Investigations Blood pressure decreased	2.9% 1/35 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.
Investigations Blood thyroid stimulating hormone decreased	2.9% 1/35 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.
Investigations Blood urine present	2.9% 1/35 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.
Investigations Blood natriuretic peptide increased	2.9% 1/35 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.
Blood and lymphatic system disorders Anaemia	2.9% 1/35 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.
Cardiac disorders Atrial fibrillation	11.4% 4/35 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.
Cardiac disorders Cardiac failure congestive	8.6% 3/35 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.
Cardiac disorders Cardiac failure	5.7% 2/35 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.
Cardiac disorders Atrial tachycardia	2.9% 1/35 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.
Cardiac disorders Bradycardia	2.9% 1/35 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.
Cardiac disorders Bundle branch block right	2.9% 1/35 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.
Cardiac disorders Palpitations	2.9% 1/35 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.
Cardiac disorders Sinus arrhythmia	2.9% 1/35 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.
Cardiac disorders Ventricular tachycardia	2.9% 1/35 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.
Congenital, familial and genetic disorders Colour blindness	2.9% 1/35 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.
Eye disorders Corneal disorder	2.9% 1/35 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.
Eye disorders Vitreous floaters	2.9% 1/35 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.
Gastrointestinal disorders Diarrhoea	20.0% 7/35 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.
Gastrointestinal disorders Constipation	14.3% 5/35 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.
Gastrointestinal disorders Nausea	14.3% 5/35 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.
Gastrointestinal disorders Abdominal pain upper	8.6% 3/35 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.
Gastrointestinal disorders Flatulence	8.6% 3/35 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.
Gastrointestinal disorders Dry mouth	5.7% 2/35 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.
Gastrointestinal disorders Vomiting	5.7% 2/35 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.
Gastrointestinal disorders Abdominal pain	2.9% 1/35 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.
Gastrointestinal disorders Cheilitis	2.9% 1/35 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.
Gastrointestinal disorders Faecal incontinence	2.9% 1/35 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.
Gastrointestinal disorders Inguinal hernia	2.9% 1/35 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.
General disorders Fatigue	22.9% 8/35 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.
General disorders Oedema peripheral	17.1% 6/35 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.
General disorders Asthenia	8.6% 3/35 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.
General disorders Oedema	8.6% 3/35 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.
General disorders Thirst	8.6% 3/35 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.
General disorders Catheter related complication	2.9% 1/35 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.
General disorders Chest discomfort	2.9% 1/35 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.
General disorders Chest pain	2.9% 1/35 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.
General disorders Hernia	2.9% 1/35 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.
General disorders Hot flush	2.9% 1/35 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.
General disorders Localised oedema	2.9% 1/35 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.
General disorders Pain	2.9% 1/35 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.
General disorders Pyrexia	2.9% 1/35 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.
Hepatobiliary disorders Hepatomegaly	11.4% 4/35 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.
Immune system disorders Seasonal allergy	8.6% 3/35 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.
Infections and infestations Upper respiratory tract infection	17.1% 6/35 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.
Infections and infestations Urinary tract infection	8.6% 3/35 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.
Infections and infestations Influenza	5.7% 2/35 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.
Infections and infestations Nasopharyngitis	5.7% 2/35 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.
Infections and infestations Bronchitis	2.9% 1/35 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.
Infections and infestations Cellulitis	2.9% 1/35 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.
Infections and infestations Furuncle	2.9% 1/35 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.
Infections and infestations Infection	2.9% 1/35 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.
Infections and infestations Lower respiratory tract infection	2.9% 1/35 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.
Infections and infestations Nail infection	2.9% 1/35 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.
Injury, poisoning and procedural complications Contusion	5.7% 2/35 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.
Injury, poisoning and procedural complications Excoriation	5.7% 2/35 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.
Injury, poisoning and procedural complications Fall	5.7% 2/35 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.
Injury, poisoning and procedural complications Joint injury	5.7% 2/35 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.
Injury, poisoning and procedural complications Skin laceration	5.7% 2/35 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.
Injury, poisoning and procedural complications Joint sprain	2.9% 1/35 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.
Injury, poisoning and procedural complications Laceration	2.9% 1/35 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.
Injury, poisoning and procedural complications Post procedural haematuria	2.9% 1/35 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.
Injury, poisoning and procedural complications Tooth injury	2.9% 1/35 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.
Investigations Weight decreased	17.1% 6/35 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.
Investigations Work increased	14.3% 5/35 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.
Investigations Alanine aminotransferase increased	5.7% 2/35 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.
Investigations Aspartate aminotransferase increased	5.7% 2/35 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.
Investigations Ejection fraction decreased	2.9% 1/35 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.
Investigations Gamma-glutamyltransferase increased	2.9% 1/35 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.
Investigations Heart rate increased	2.9% 1/35 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.
Investigations International normalised ratio increased	2.9% 1/35 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.
Investigations Urine output increased	2.9% 1/35 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.
Investigations Venous pressure jugular increased	2.9% 1/35 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.
Metabolism and nutrition disorders Anorexia	5.7% 2/35 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.
Metabolism and nutrition disorders Decreased appetite	5.7% 2/35 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.
Metabolism and nutrition disorders Fluid overload	5.7% 2/35 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.
Metabolism and nutrition disorders Gout	5.7% 2/35 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.
Metabolism and nutrition disorders Fluid retention	2.9% 1/35 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.
Metabolism and nutrition disorders Hyperkalaemia	2.9% 1/35 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.
Metabolism and nutrition disorders Hypochloraemia	2.9% 1/35 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.
Metabolism and nutrition disorders Hyponatraemia	2.9% 1/35 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.
Musculoskeletal and connective tissue disorders Back pain	8.6% 3/35 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.
Musculoskeletal and connective tissue disorders Pain in extremity	8.6% 3/35 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.
Musculoskeletal and connective tissue disorders Muscle spasms	5.7% 2/35 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.
Musculoskeletal and connective tissue disorders Arthralgia	2.9% 1/35 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.
Musculoskeletal and connective tissue disorders Finger deformity	2.9% 1/35 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.
Musculoskeletal and connective tissue disorders Joint swelling	2.9% 1/35 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.
Musculoskeletal and connective tissue disorders Muscle twitching	2.9% 1/35 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.
Musculoskeletal and connective tissue disorders Musculoskeletal discomfort	2.9% 1/35 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.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	2.9% 1/35 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal cell carcinoma	2.9% 1/35 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Benign neoplasm	2.9% 1/35 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lentigo	2.9% 1/35 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Prostate cancer	2.9% 1/35 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.
Nervous system disorders Dizziness	20.0% 7/35 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.
Nervous system disorders Dizziness postural	17.1% 6/35 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.
Nervous system disorders Balance disorder	14.3% 5/35 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.
Nervous system disorders Aguesia	8.6% 3/35 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.
Nervous system disorders Tremor	5.7% 2/35 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.
Nervous system disorders Amnesia	2.9% 1/35 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.
Nervous system disorders Disturbance in attention	2.9% 1/35 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.
Nervous system disorders Headache	2.9% 1/35 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.
Nervous system disorders Loss of consciousness	2.9% 1/35 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.
Nervous system disorders Memory impairment	2.9% 1/35 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.
Nervous system disorders Migraine	2.9% 1/35 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.
Nervous system disorders Neuralgia	2.9% 1/35 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.
Nervous system disorders Neuropathy	2.9% 1/35 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.
Nervous system disorders Neuropathy peripheral	2.9% 1/35 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.
Nervous system disorders Restless legs syndrome	2.9% 1/35 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.
Nervous system disorders Somnolence	2.9% 1/35 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.
Nervous system disorders Syncope	2.9% 1/35 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.
Psychiatric disorders Insomnia	8.6% 3/35 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.
Psychiatric disorders Confusional state	5.7% 2/35 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.
Psychiatric disorders Depression	5.7% 2/35 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.
Psychiatric disorders Anxiety	2.9% 1/35 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.
Psychiatric disorders Nervousness	2.9% 1/35 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.
Psychiatric disorders Panic attack	2.9% 1/35 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.
Renal and urinary disorders Haematuria	5.7% 2/35 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.
Renal and urinary disorders Dysuria	2.9% 1/35 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.
Renal and urinary disorders Pollakiuria	2.9% 1/35 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.
Renal and urinary disorders Urinary incontinence	2.9% 1/35 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.
Reproductive system and breast disorders Nipple pain	5.7% 2/35 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.
Reproductive system and breast disorders Gynaecomastia	2.9% 1/35 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.
Reproductive system and breast disorders Scrotal oedema	2.9% 1/35 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.
Respiratory, thoracic and mediastinal disorders Dyspnoea	31.4% 11/35 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.
Respiratory, thoracic and mediastinal disorders Dyspnoea exertional	17.1% 6/35 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.
Respiratory, thoracic and mediastinal disorders Epistaxis	8.6% 3/35 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.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	8.6% 3/35 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.
Respiratory, thoracic and mediastinal disorders Cough	5.7% 2/35 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.
Respiratory, thoracic and mediastinal disorders Pleural effusion	5.7% 2/35 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.
Respiratory, thoracic and mediastinal disorders Sinus congestion	5.7% 2/35 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.
Respiratory, thoracic and mediastinal disorders Dysphonia	2.9% 1/35 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.
Respiratory, thoracic and mediastinal disorders Postnasal drip	2.9% 1/35 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.
Respiratory, thoracic and mediastinal disorders Productive cough	2.9% 1/35 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.
Respiratory, thoracic and mediastinal disorders Pulmonary congestion	2.9% 1/35 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.
Respiratory, thoracic and mediastinal disorders Rales	2.9% 1/35 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.
Skin and subcutaneous tissue disorders Pruritus	8.6% 3/35 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.
Skin and subcutaneous tissue disorders Rash	8.6% 3/35 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.
Skin and subcutaneous tissue disorders Skin ulcer	8.6% 3/35 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.
Skin and subcutaneous tissue disorders Blister	2.9% 1/35 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.
Skin and subcutaneous tissue disorders Ingrowing nail	2.9% 1/35 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.
Skin and subcutaneous tissue disorders Rash maculo-papular	2.9% 1/35 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.
Skin and subcutaneous tissue disorders Skin discolouration	2.9% 1/35 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.
Skin and subcutaneous tissue disorders Skin exfoliation	2.9% 1/35 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.
Skin and subcutaneous tissue disorders Skin lesion	2.9% 1/35 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.
Surgical and medical procedures Foot operation	2.9% 1/35 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.
Surgical and medical procedures Implantable defibrillator insertion	2.9% 1/35 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.
Surgical and medical procedures Mole excision	2.9% 1/35 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.
Surgical and medical procedures Tooth extraction	2.9% 1/35 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.
Vascular disorders Hypotension	2.9% 1/35 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.
Vascular disorders Varicose vein	2.9% 1/35 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.
Cardiac disorders Bundle branch block	2.9% 1/35 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.

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer, Inc.

Phone: 1-800-718-1021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER