Trial Outcomes & Findings for An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy (NCT NCT00791492)

NCT ID: NCT00791492

Last Updated: 2012-12-17

Results Overview

Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 86 participants
• Primary outcome timeframe: Month 6
• Results posted on: 2012-12-17

Participant Flow

Participants who completed study FX-005 (NCT00409175), were eligible for the current study FX-006 (NCT00791492).

Participant milestones

Measure	Tafamidis-Tafamidis Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Overall Study STARTED	45	41
Overall Study TREATED	44	41
Overall Study COMPLETED	39	38
Overall Study NOT COMPLETED	6	3

Reasons for withdrawal

Measure	Tafamidis-Tafamidis Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Overall Study Withdrawal by Subject	1	2
Overall Study Liver transplantation	5	1

Baseline Characteristics

An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

Baseline characteristics by cohort

Measure	Tafamidis-Tafamidis n=44 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Total n=85 Participants Total of all reporting groups
Age Continuous	41.3 years STANDARD_DEVIATION 13.4 • n=5 Participants	39.6 years STANDARD_DEVIATION 13.2 • n=7 Participants	40.4 years STANDARD_DEVIATION 13.3 • n=5 Participants
Sex: Female, Male Female	24 Participants n=5 Participants	23 Participants n=7 Participants	47 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants	18 Participants n=7 Participants	38 Participants n=5 Participants

PRIMARY outcome

Timeframe: Month 6

Population: Intent-to-treat (ITT) population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure.

Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than[<] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.

Outcome measures

Measure	Tafamidis-Tafamidis n=37 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=32 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6	62.2 percentage of participants Interval 44.8 to 77.5	68.8 percentage of participants Interval 50.0 to 83.9

PRIMARY outcome

Timeframe: Month 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.

Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than[<] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.

Outcome measures

Measure	Tafamidis-Tafamidis n=33 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=30 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 12	54.5 percentage of participants Interval 36.4 to 71.9	60.0 percentage of participants Interval 40.6 to 77.3

PRIMARY outcome

Timeframe: Baseline, Month 6

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure.

Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=33 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 Baseline	21.05 units on a scale Standard Deviation 21.88	38.09 units on a scale Standard Deviation 31.89
Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 Change at Month 6	0.03 units on a scale Standard Deviation 8.83	-4.88 units on a scale Standard Deviation 15.29

PRIMARY outcome

Timeframe: Baseline, Month 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure.

Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.

Outcome measures

Measure	Tafamidis-Tafamidis n=32 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=30 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 12	2.25 units on a scale Standard Deviation 8.91	-2.33 units on a scale Standard Deviation 15.66

SECONDARY outcome

Timeframe: Baseline, Month 6, 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group.

NIS-LL: assessed muscle weakness, reflexes and sensation; scored separately for left and right limbs. Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) are scored on scale 0 (normal) to 4 (paralysis), higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) were scored 0 = normal, 1= decreased, or 2 = absent. Total possible NIS-LL score range 0-88, higher score=greater impairment.

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=33 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Neuropathy Impairment Score - Lower Limb (NIS-LL) Score at Month 6 and 12 Baseline (n=38,33)	8.38 units on a scale Standard Deviation 13.23	17.50 units on a scale Standard Deviation 20.82
Change From Baseline in Neuropathy Impairment Score - Lower Limb (NIS-LL) Score at Month 6 and 12 Change at Month 6 (n=37,32)	0.93 units on a scale Standard Deviation 3.65	1.89 units on a scale Standard Deviation 4.55
Change From Baseline in Neuropathy Impairment Score - Lower Limb (NIS-LL) Score at Month 6 and 12 Change at Month 12 (n=33,30)	1.36 units on a scale Standard Deviation 4.77	1.60 units on a scale Standard Deviation 8.20

SECONDARY outcome

Timeframe: Baseline, Month 6, 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group.

Norfolk QOL-DN: 35-item participant-rated questionnaire to assess impact of DN on QOL; Item 1-7:scored as 1=symptoms present, 0=symptoms absent. Item 8-35:scored on 5-point Likert scale: 0=no problem,4=severe problem (except item 32: -2=much better, 0=about same, 2=much worse). Norfolk QOL-DN summarized in 5 domains(score range):physical functioning/large fiber neuropathy(-2 to 58), activities of daily living(ADLs) (0 to 20), symptoms(0 to 32), small fiber neuropathy(0 to 16), autonomic neuropathy(0 to 12); higher score=greater impairment,for each. Total score=-2 to138(higher score=worse QOL).

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=33 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Baseline: ADLs (n=38,33)	2.21 units on a scale Standard Deviation 4.38	3.48 units on a scale Standard Deviation 5.13
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Baseline: small fiber neuropathy (n=38,33)	2.50 units on a scale Standard Deviation 3.85	4.42 units on a scale Standard Deviation 4.23
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 6: physical functioning (n=37,32)	0.14 units on a scale Standard Deviation 4.08	-3.00 units on a scale Standard Deviation 8.08
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 6: ADLs (n=37,32)	-0.30 units on a scale Standard Deviation 1.45	-0.59 units on a scale Standard Deviation 3.02
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 12: ADLs (n=32,30)	0.13 units on a scale Standard Deviation 1.01	-0.50 units on a scale Standard Deviation 3.35
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 12: symptoms (n=32,30)	-0.66 units on a scale Standard Deviation 2.54	-1.10 units on a scale Standard Deviation 3.32
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 12:small fiber neuropathy(n=32,30)	0.41 units on a scale Standard Deviation 2.87	0.40 units on a scale Standard Deviation 2.61
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 12: autonomic neuropathy (n=32,30)	0.53 units on a scale Standard Deviation 1.46	-0.30 units on a scale Standard Deviation 1.66
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Baseline: physical functioning (n=38,33)	9.45 units on a scale Standard Deviation 11.43	19.88 units on a scale Standard Deviation 17.44
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Baseline: symptoms (n=38,33)	5.34 units on a scale Standard Deviation 4.43	7.48 units on a scale Standard Deviation 5.48
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Baseline: autonomic neuropathy (n=38,33)	1.55 units on a scale Standard Deviation 2.37	2.82 units on a scale Standard Deviation 3.12
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 6: symptoms (n=34,32)	0.00 units on a scale Standard Deviation 3.18	-0.75 units on a scale Standard Deviation 4.27
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 6: small fiber neuropathy(n=37,32)	-0.14 units on a scale Standard Deviation 2.64	-0.09 units on a scale Standard Deviation 2.32
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 6: autonomic neuropathy (n=37,32)	0.19 units on a scale Standard Deviation 1.33	-0.44 units on a scale Standard Deviation 2.00
Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 Change at Month 12: physical functioning(n=32,30)	1.84 units on a scale Standard Deviation 6.00	-0.83 units on a scale Standard Deviation 9.62

SECONDARY outcome

Timeframe: Baseline, Month 6, 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group.

Summated 7 score: composite score included five Nerve Conduction Studies (NCS) attributes (peroneal nerve distal motor latency, peroneal nerve compound muscle action potential, peroneal nerve motor conduction velocity, tibial nerve distal motor latency, and sural nerve sensory nerve action potential amplitude) along with Vibration Detection Threshold (VDT) obtained in great toes, and Heart Rate Response to Deep Breathing (HRDB) value. Score was determined through reference to normal values for age, sex and height. Total score range= -26 to 26, where higher score=worse nerve function.

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=33 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6 and 12 Baseline (n=38,33)	6.68 units on a scale Standard Deviation 8.51	10.06 units on a scale Standard Deviation 10.68
Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6 and 12 Change at Month 6 (n=37,32)	-0.01 units on a scale Standard Deviation 2.99	1.38 units on a scale Standard Deviation 3.58
Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6 and 12 Change at Month 12 (n=33,30)	0.64 units on a scale Standard Deviation 3.23	1.48 units on a scale Standard Deviation 4.02

SECONDARY outcome

Timeframe: Baseline, Month 6, 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group.

Summated 3 Nerve Tests Small Fiber Normal Deviates Score (NTSFnds) included cooling threshold for the lower limbs, heat pain threshold for the lower limbs and HRDB. Total score range= -11.2 to 11.2, where higher score=worse nerve function.

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=33 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6 and 12 Baseline (n=38,33)	4.78 units on a scale Standard Deviation 4.33	7.08 units on a scale Standard Deviation 4.39
Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6 and 12 Change at Month 6 (n=36,32)	0.43 units on a scale Standard Deviation 1.51	0.38 units on a scale Standard Deviation 1.08
Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6 and 12 Change at Month 12 (n=32,30)	0.59 units on a scale Standard Deviation 1.28	0.50 units on a scale Standard Deviation 1.48

SECONDARY outcome

Timeframe: Baseline, Month 6, 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group.

BMI was calculated by weight divided by height squared. mBMI was calculated by multiplying BMI by serum albumin levels to compensate for edema formation. A progressive decline in mBMI indicated worsening of disease severity.

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=33 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Modified Body Mass Index (mBMI) at Month 6 and 12 Baseline (n=38,33)	23.94 (kilogram/square meter)*(gram/liter) Standard Deviation 2.97	22.71 (kilogram/square meter)*(gram/liter) Standard Deviation 5.15
Change From Baseline in Modified Body Mass Index (mBMI) at Month 6 and 12 Change at Month 6 (n=37,32)	-0.13 (kilogram/square meter)*(gram/liter) Standard Deviation 0.81	0.42 (kilogram/square meter)*(gram/liter) Standard Deviation 1.14
Change From Baseline in Modified Body Mass Index (mBMI) at Month 6 and 12 Change at Month 12 (n=33,30)	-0.12 (kilogram/square meter)*(gram/liter) Standard Deviation 1.16	0.73 (kilogram/square meter)*(gram/liter) Standard Deviation 1.45

SECONDARY outcome

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

Population: Safety population included participants who received at least one dose of study medication. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants evaluable for this measure at the specified time point for each arm group.

Troponin I was biomarker of cardiac stress (myocardial necrosis and increased filling pressures/ left ventricular [LV] wall stress).

Outcome measures

Measure	Tafamidis-Tafamidis n=29 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=30 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 Change at Month 3 (n=29,30)	0.0 nanogram/milliliter (ng/mL) Interval -0.1 to 0.3	0.0 nanogram/milliliter (ng/mL) Interval 0.0 to 0.7
Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 Change at Month 6 (n=29,30)	0.0 nanogram/milliliter (ng/mL) Interval 0.0 to 0.6	0.0 nanogram/milliliter (ng/mL) Interval 0.0 to 0.1
Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 Change at Month 12 (n=26,28)	0.0 nanogram/milliliter (ng/mL) Interval -0.3 to 0.4	0.0 nanogram/milliliter (ng/mL) Interval 0.0 to 0.0
Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 Baseline (n=29,30)	0.2 nanogram/milliliter (ng/mL) Interval 0.2 to 0.5	0.2 nanogram/milliliter (ng/mL) Interval 0.2 to 0.2
Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 Change at Week 6 (n=28,29)	0.0 nanogram/milliliter (ng/mL) Interval 0.0 to 0.1	0.0 nanogram/milliliter (ng/mL) Interval 0.0 to 0.0

SECONDARY outcome

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

Population: Safety population included participants who received at least one dose of study medication. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants evaluable for this measure at the specified time point for each arm group.

NT-proBNP was biomarker of cardiac stress (myocardial necrosis and increased filling pressures/ LV wall stress).

Outcome measures

Measure	Tafamidis-Tafamidis n=33 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=34 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Week 6, Month 3, 6 and 12 Baseline (n=33,34)	41.0 picogram/milliliter (pg/mL) Interval 19.0 to 2879.0	75.0 picogram/milliliter (pg/mL) Interval 20.0 to 1127.0
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Week 6, Month 3, 6 and 12 Change at Week 6 (n=33,33)	6.0 picogram/milliliter (pg/mL) Interval -650.0 to 525.0	-1.0 picogram/milliliter (pg/mL) Interval -292.0 to 97.0
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Week 6, Month 3, 6 and 12 Change at Month 3 (n=33,34)	11.0 picogram/milliliter (pg/mL) Interval -52.0 to 1355.0	1.0 picogram/milliliter (pg/mL) Interval -514.0 to 229.0
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Week 6, Month 3, 6 and 12 Change at Month 6 (n=33,33)	9.0 picogram/milliliter (pg/mL) Interval -210.0 to 3878.0	0.0 picogram/milliliter (pg/mL) Interval -473.0 to 170.0
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Week 6, Month 3, 6 and 12 Change at Month 12 (n=30,29)	15.0 picogram/milliliter (pg/mL) Interval -104.0 to 3840.0	-6.0 picogram/milliliter (pg/mL) Interval -342.0 to 410.0

SECONDARY outcome

Timeframe: Baseline

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure.

IENF density was quantified in 3 millimeter (mm) immunostained skin punch biopsies containing epidermis and superficial dermis to evaluate the amount and morphological appearance of small diameter nerve fibers, both somatic and autonomic, in sensory neuropathies. It is used in diagnosing various neuropathic conditions.

Outcome measures

Measure	Tafamidis-Tafamidis n=11 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=5 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Intraepidermal Nerve Fiber (IENF) Density Left distal leg	8.2 fibers/mm Standard Deviation 9.8	8.0 fibers/mm Standard Deviation 9.2
Intraepidermal Nerve Fiber (IENF) Density Left proximal thigh	16.1 fibers/mm Standard Deviation 12.6	15.3 fibers/mm Standard Deviation 11.9

SECONDARY outcome

Timeframe: Month 6, 12

Population: ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure.

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.

Outcome measures

Measure	Tafamidis-Tafamidis n=36 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=32 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer Month 6	94 percentage of participants Interval 81.0 to 99.0	97 percentage of participants Interval 83.0 to 99.0
Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer Month 12	94 percentage of participants Interval 80.0 to 99.0	93 percentage of participants Interval 77.0 to 99.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 30 days after last dose of study medication

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

An Adverse Event (AE) was any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. SAE: AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent/significant disability/incapacity; congenital anomaly. Treatment-emergent were events between first dose of study drug, up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state.

Outcome measures

Measure	Tafamidis-Tafamidis n=44 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)	5 participants	4 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 30 days after last dose of study medication

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

AE=any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. Grade 3 (Severe) events=unacceptable or intolerable events, significantly interrupting usual daily activity, require systemic drug therapy/other treatment. Grade 4 (Life-threatening) events caused participant to be in imminent danger of death. Grade 5 (Death) events=death related to an AE. Treatment-emergent 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

Measure	Tafamidis-Tafamidis n=44 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Number of Participants With Treatment-emergent Adverse Events Greater Than or Equal to Grade 3	6 participants	4 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Day 1 up to Month 12 (anytime on-treatment)

Population: Safety population included participants who received at least one dose of study medication. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants evaluable for this measure at the specified time point for each arm group.

Clinically significant ECHO findings included: LV posterior wall thickness greater than or equal to (>=)13 mm, LV septal thickness >= 13 mm, right ventricular thickness >= 7 mm, ratio of peak mitral early diastolic and atrial contraction velocity (E/A ratio) >= 2, prime septal (E/E) >15, ejection fraction < 50 percent (%), E deceleration time <= 150 millisecond (ms), isovolumic relaxation time (IVRT) <= 70 ms, any valve thickening (> trace regurgitation in mitral, aortic, pulmonary, or tricuspid valves), abnormal respiratory variation of inferior vena cava, pericardial effusion.

Outcome measures

Measure	Tafamidis-Tafamidis n=38 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=34 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Number of Participants With Clinically Significant Treatment-emergent Echocardiography (ECHO) Findings Baseline (n=38,34)	23 participants	18 participants
Number of Participants With Clinically Significant Treatment-emergent Echocardiography (ECHO) Findings Anytime on-treatment (n=21,23)	16 participants	13 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Day 1 up to Month 12 (anytime on-treatment)

Population: Safety population included participants who received at least one dose of study medication. 'n' signifies participants for this measure at specified time point for each arm group.

Clinically significant ECG findings included: corrected QT (QTc) > 450 ms, QTc >500 ms, change in QTc between 30 and 60 ms, change in QTc greater than or equal to 60 ms.

Outcome measures

Measure	Tafamidis-Tafamidis n=44 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Number of Participants With Clinically Significant Treatment-emergent Electrocardiogram (ECG) Findings Baseline (n=44,41)	16 participants	17 participants
Number of Participants With Clinically Significant Treatment-emergent Electrocardiogram (ECG) Findings Anytime on-treatment (n=28,24)	6 participants	6 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Day 1 up to Month 12 (anytime on-treatment)

Population: Safety population included participants who received at least one dose of study medication. 'n' signifies participants for this measure at specified time point for each arm group.

Clinically significant Holter monitor findings included: atrial fibrillation/flutter, atrial tachycardia, non-sustained ventricular tachycardia (<30 beats), sustained ventricular tachycardia (>= 30 beats), sinus pause (RR >2.0 second, where RR=60/heart rate), ventricular premature contractions.

Outcome measures

Measure	Tafamidis-Tafamidis n=44 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Number of Participants With Clinically Significant Treatment-emergent Holter Monitor Findings Baseline (n=44,41)	20 participants	21 participants
Number of Participants With Clinically Significant Treatment-emergent Holter Monitor Findings Anytime on-treatment (n=24,20)	8 participants	7 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Month 12

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

An AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.

Outcome measures

Measure	Tafamidis-Tafamidis n=44 Participants Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 Participants Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Number of Participants Who Discontinued Due to Clinical or Laboratory Adverse Events	0 participants	0 participants

Adverse Events

Tafamidis-Tafamidis

Serious events: 5 serious events

Other events: 24 other events

Deaths: 0 deaths

Placebo-Tafamidis

Serious events: 4 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Measure	Tafamidis-Tafamidis n=44 participants at risk Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 participants at risk Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Blood and lymphatic system disorders Anaemia	0.00% 0/44 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.	2.4% 1/41 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	4.5% 2/44 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.	2.4% 1/41 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	2.3% 1/44 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.	2.4% 1/41 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 Hypersensitivity	2.3% 1/44 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.	0.00% 0/41 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 Catheter sepsis	2.3% 1/44 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.	0.00% 0/41 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 Catheter site infection	2.3% 1/44 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.	0.00% 0/41 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	0.00% 0/44 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.	2.4% 1/41 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 Meningitis	0.00% 0/44 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.	2.4% 1/41 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 Osteomyelitis	0.00% 0/44 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.	2.4% 1/41 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	0.00% 0/44 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.	2.4% 1/41 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 electrolytes decreased	2.3% 1/44 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.	0.00% 0/41 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	0.00% 0/44 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.	2.4% 1/41 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) Lymphoma	2.3% 1/44 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.	0.00% 0/41 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	2.3% 1/44 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.	0.00% 0/41 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

Measure	Tafamidis-Tafamidis n=44 participants at risk Participants who received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.	Placebo-Tafamidis n=41 participants at risk Participants who received placebo matched to tafamidis (Fx-1006A) 20 mg capsule orally once daily for 18 months during previous study FX-005 (NCT00409175), received tafamidis (Fx-1006A) 20 mg capsule orally once daily for 12 months.
Eye disorders Punctate keratitis	6.8% 3/44 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.	7.3% 3/41 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 Dry eye	4.5% 2/44 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.	7.3% 3/41 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	9.1% 4/44 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.	7.3% 3/41 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	4.5% 2/44 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.	9.8% 4/41 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	11.4% 5/44 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.	14.6% 6/41 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	6.8% 3/44 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.	17.1% 7/41 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	11.4% 5/44 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.	7.3% 3/41 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	4.5% 2/44 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.	7.3% 3/41 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 Thermal burn	9.1% 4/44 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.	9.8% 4/41 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	4.5% 2/44 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.	14.6% 6/41 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	2.3% 1/44 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.	12.2% 5/41 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

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Phone: 1-800-718-1021

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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