Trial Outcomes & Findings for The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis (NCT NCT00630864)

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

21 participants

Primary outcome timeframe

Week 6

Results posted on

2013-01-18

Participant Flow

Participant milestones

Participant milestones
Measure	Tafamidis Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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	21
Part 1 (up to Week 6) COMPLETED	20
Part 1 (up to Week 6) NOT COMPLETED	1
Part 2 (After Week 6 up to Month 12) STARTED	20
Part 2 (After Week 6 up to Month 12) COMPLETED	18
Part 2 (After Week 6 up to Month 12) NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Tafamidis Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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) Adverse Event	1
Part 2 (After Week 6 up to Month 12) Liver transplant	2

Baseline Characteristics

The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Age Continuous	63.10 years STANDARD_DEVIATION 9.86 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 6

Population: Intent-to-Treat (ITT) population included all participants who received at least 1 dose of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were 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

Outcome measures
Measure	Tafamidis n=19 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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	94.7 percentage of participants Interval 74.0 to 99.9

SECONDARY outcome

Timeframe: Month 6, Month 12

Population: ITT population included all participants who received at least 1 dose of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure.

TTR tetramer was assessed using a validated immunoturbidimetric assay. The 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

Outcome measures
Measure	Tafamidis n=18 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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 (n= 18)	100.0 percentage of participants Interval 81.5 to 100.0
Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Month 6 and 12 Month 12 (n= 17)	100.0 percentage of participants Interval 80.5 to 100.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 30 days after the last dose

Population: ITT population included all participants who received at least 1 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=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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)	17 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 1 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. On the basis of intensity, grade 3 was referred as severe, grade 4 as life-threatening and grade 5 as death.

Outcome measures

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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 Adverse Events	3 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 1 up to Month 12

Population: ITT population. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal (that is treatment-emergent abnormalities). Abnormalities at early termination were excluded from the analysis.

ECHO: investigator assessed test to assess cardiac function. ECHO abnormality criteria: any abnormality, valvular abnormality, pericardial effusion, abnormal regional wall motion, inferior vena cava respiratory variation, posterior (P) left ventricular (LV) wall/septal (S) thickness, right ventricular thickness, ejection fraction, ratio of early (E) diastolic transmitral flow and atrial(A) contraction velocity (E/A), ratio of 'E'to lateral/septal mitral annular velocity (e') (E/e'prime lateral, E/e'prime septal), E deceleration time (DT), isovolumic relaxation time (IVRT).

Outcome measures

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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= 19)	12 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Pericardial effusion (n= 19)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Valvular abnormalities- thickening (n= 5)	3 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Valvular abnormalities - regurgitation (n= 9)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Abnormal regional wall motion (n= 11)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Inferior vena cava respiratory variation (n= 13)	0 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings LV P wall thickness >=13 millimeter (mm) (n= 3)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings LV S thickness >=13 mm (n= 3)	1 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Right ventricular thickness >=7 mm (n= 13)	6 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E/A ratio >=2 (n= 16)	3 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E/e'prime Lateral >15 (n= 15)	3 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings E/e'prime Septal >15 (n= 10)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings Ejection fraction < 50 percent (%) (n= 17)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings EDT <= 150 milliseconds (msec) (n= 14)	1 participants
Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings IVRT <=70 msec (n= 13)	2 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 1 up to Month 12

Population: ITT population. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal (that is treatment-emergent abnormalities). Abnormalities at early termination were excluded from the analysis.

ECG: investigator assessed test to assess cardiac function. ECG abnormality criteria: any abnormality, arrhythmia, rhythm, conduction, morphology, myocardial infarction, ST segment, T waves and abnormal U waves.

Outcome measures

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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 Electrocardiogram (ECG) Findings Any ECG abnormalities (n= 21)	9 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings Arrhythmia (n= 18)	8 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings Rhythm (n= 18)	1 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings Conduction (n= 8)	2 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings Morphology (n= 20)	0 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings Myocardial infarction (n= 18)	0 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings ST segment (n= 20)	1 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings T waves (n=18)	1 participants
Number of Participants With Clinically Significant Treatment-Emergent Electrocardiogram (ECG) Findings Abnormal U waves (n= 21)	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 1 up to Month 12

Population: ITT population. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal (that is treatment-emergent abnormalities). Abnormalities at early termination were excluded from the analysis.

Holter monitoring recorded heart rhythm. Holter monitoring abnormality criteria: any abnormality, atrial fibrillation/flutter, atrial tachycardia, non-sustained ventricular tachycardia (VT), sustained VT and sinus pause.

Outcome measures

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion 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 Holter Monitoring Findings Atrial tachycardia (n= 9)	4 participants
Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings Any Holter monitoring abnormalities (n= 19)	9 participants
Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings Atrial fibrillation/flutter (n= 18)	1 participants
Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings Non-sustained VT <30 beats (n= 11)	4 participants
Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings Sustained VT >=30 beats (n= 19)	0 participants
Number of Participants With Clinically Significant Treatment-Emergent Holter Monitoring Findings Sinus pause (n= 18)	1 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Month 12

Population: ITT population included all participants who received at least 1 dose of study medication.

Outcome measures

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Number of Participants Who Discontinued Due to Clinical or Laboratory Adverse Events	1 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: ITT population included all participants who received at least 1 dose of study medication. Here, 'n' signifies those participants who were analyzed for the specific category.

NIS assessed cranial nerves(nerve 3,6; facial, palate and tongue weakness),muscle weakness (respiratory; neck, elbow(E), wrist(W), finger(F), hip, knee(K) flexion; shoulder, thumb abduction; brachioradialis; E, W, hip, K extension; F spread; toe, dorsal and plantar ankle flexors; toe extensors); score: 0-4, higher score=more weakness, reflexes(biceps and triceps brachii; brachioradialis; quadriceps femoris; triceps surae), index F and great toe sensation(touch pressure, pin-prick, vibration, joint position)score:0=normal,1=decreased or 2=absent. Total score=0-244, higher score=more impairment.

Outcome measures

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 6, Month 12 Baseline (n= 21)	48.70 units on a scale Standard Deviation 44.31
Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 6, Month 12 Change at Month 6 (n= 17)	2.00 units on a scale Standard Deviation 9.52
Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 6, Month 12 Change at Month 12 (n= 18)	5.30 units on a scale Standard Deviation 12.62

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Month 6, Month 12

Population: ITT population included all participants who received at least 1 dose of study medication. Here, 'n' signifies those participants who were analyzed for the specific category.

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 0 to 4 scale, 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

Outcome measures
Measure	Tafamidis n=21 Participants Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Change From Baseline in the Neuropathy Impairment Score-Lower Limb (NIS-LL) at Month 6, Month 12 Baseline (n= 21)	27.60 units on a scale Standard Deviation 24.67
Change From Baseline in the Neuropathy Impairment Score-Lower Limb (NIS-LL) at Month 6, Month 12 Change at Month 6 (n= 19)	-0.50 units on a scale Standard Deviation 5.73
Change From Baseline in the Neuropathy Impairment Score-Lower Limb (NIS-LL) at Month 6, Month 12 Change at Month 12 (n= 18)	2.70 units on a scale Standard Deviation 6.21

Adverse Events

Tafamidis

Serious events: 8 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tafamidis n=21 participants at risk Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Cardiac disorders Atrioventricular block	4.8% 1/21 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 Coronary artery stenosis	4.8% 1/21 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 Faecaloma	4.8% 1/21 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 Subileus	4.8% 1/21 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 Malaise	4.8% 1/21 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	9.5% 2/21 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 Ankle fracture	4.8% 1/21 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 Avulsion fracture	4.8% 1/21 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 Arthritis	4.8% 1/21 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 Transient ischaemic attack	4.8% 1/21 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 retention	4.8% 1/21 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 Carpal tunnel decompression	4.8% 1/21 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=21 participants at risk Participants with transthyretin (TTR) variants other than valine replaced by methionine at position 30 (V30M) received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1, participants who achieved TTR stabilization at Week 6 or as per investigator's discretion continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2.
Gastrointestinal disorders Diarrhoea	23.8% 5/21 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	14.3% 3/21 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% 3/21 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	9.5% 2/21 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	9.5% 2/21 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	9.5% 2/21 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 Laryngitis	9.5% 2/21 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 Sinusitis	9.5% 2/21 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	19.0% 4/21 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	19.0% 4/21 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	9.5% 2/21 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	9.5% 2/21 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 Paraesthesia	9.5% 2/21 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	9.5% 2/21 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	14.3% 3/21 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	9.5% 2/21 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	9.5% 2/21 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	14.3% 3/21 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	9.5% 2/21 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 Orthostatic hypotension	9.5% 2/21 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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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.
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Restriction type: OTHER