Trial Outcomes & Findings for A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) (NCT NCT04814186)
NCT ID: NCT04814186
Last Updated: 2024-12-18
Results Overview
An adverse event (AE) was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. All AEs that started after the first dosing but before the last dose plus the lag time (28 days) were TEAEs. A serious adverse event (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; persistent disability/incapacity; congenital anomaly/birth defect. A severe TEAE was an event that prevented normal everyday activities. Severe TEAEs were assessed by the investigator.
COMPLETED
PHASE4
53 participants
From first dose of study intervention on Day 1 (baseline) up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
2024-12-18
Participant Flow
A total of 53 participants were enrolled in this study.
Participant milestones
| Measure |
All Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Treatment Phase
STARTED
|
53
|
|
Treatment Phase
COMPLETED
|
43
|
|
Treatment Phase
NOT COMPLETED
|
10
|
|
Follow-Up Phase
STARTED
|
53
|
|
Follow-Up Phase
COMPLETED
|
44
|
|
Follow-Up Phase
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
All Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Treatment Phase
Adverse Event
|
1
|
|
Treatment Phase
Death
|
6
|
|
Treatment Phase
Withdrawal by Subject
|
2
|
|
Treatment Phase
Other
|
1
|
|
Follow-Up Phase
Adverse Event
|
1
|
|
Follow-Up Phase
Death
|
6
|
|
Follow-Up Phase
Withdrawal by Subject
|
1
|
|
Follow-Up Phase
Other
|
1
|
Baseline Characteristics
A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Baseline characteristics by cohort
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
36 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
47 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
53 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose of study intervention on Day 1 (baseline) up to 28 days post the last dose of study intervention (up to a maximum of 393 days)Population: Safety analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule.
An adverse event (AE) was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. All AEs that started after the first dosing but before the last dose plus the lag time (28 days) were TEAEs. A serious adverse event (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; persistent disability/incapacity; congenital anomaly/birth defect. A severe TEAE was an event that prevented normal everyday activities. Severe TEAEs were assessed by the investigator.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
TEAEs
|
45 Participants
|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
Treatment-Emergent SAEs
|
21 Participants
|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
Severe TEAEs
|
17 Participants
|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
TEAEs Leading to Death
|
6 Participants
|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
Discontinuations From Study Due to TEAEs
|
1 Participants
|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
Permanent Discontinuations From Study Intervention Due to TEAEs
|
1 Participants
|
|
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
Dosing Interruptions of the Study Intervention due to TEAEs
|
3 Participants
|
PRIMARY outcome
Timeframe: From first dose of study intervention on Day 1 (baseline) up to 28 days post the last dose of study intervention (up to a maximum of 393 days)Population: Safety analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule.
An AE was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. All AEs that started after the first dosing but before the last dose plus the lag time (28 days) were TEAEs. 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; persistent disability/incapacity; congenital anomaly/birth defect. A severe TEAE was an event that prevented normal everyday activities. Severe TEAEs and treatment-related TEAEs were assessed by the investigator.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Number of Participants With Treatment-Related TEAEs
TEAEs
|
3 Participants
|
|
Number of Participants With Treatment-Related TEAEs
Treatment-Emergent SAEs
|
0 Participants
|
|
Number of Participants With Treatment-Related TEAEs
Severe TEAEs
|
0 Participants
|
|
Number of Participants With Treatment-Related TEAEs
TEAEs Leading to Death
|
0 Participants
|
|
Number of Participants With Treatment-Related TEAEs
Discontinuations From Study Due to TEAEs
|
0 Participants
|
|
Number of Participants With Treatment-Related TEAEs
Permanent Discontinuations From Study Intervention Due to TEAEs
|
0 Participants
|
|
Number of Participants With Treatment-Related TEAEs
Dosing Interruptions of the Study Intervention due to TEAEs
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, months 6 and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule. Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of participants who had a reportable measurement of 6MWT at baseline, at Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
As a measure of functional capacity, the 6MWT measured the distance a participant could walk on a straight course in 6 minutes. 6MWT was conducted in accordance with guidelines established by the American Thoracic Society at Month 6 and Month 12 visits. Baseline was defined as the last measurement prior to first dose of study intervention.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
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|---|---|
|
Change From Baseline (CFB) for Distance Walked During 6 Minute Walk Test (6MWT) at Months 6 and 12
Month 6
|
-19.3 Meters
Standard Deviation 73.36
|
|
Change From Baseline (CFB) for Distance Walked During 6 Minute Walk Test (6MWT) at Months 6 and 12
Month 12
|
1.5 Meters
Standard Deviation 91.29
|
SECONDARY outcome
Timeframe: Baseline, months 6 and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule. Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of participants who had a reportable measurement of NT-proBNP at baseline, at Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
Plasma NT-proBNP is a guideline-mandated biomarker in heart failure. Baseline was defined as the last measurement prior to the first dose of study treatment.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
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|---|---|
|
Change From Baseline for N Terminal Prohormone B Type Natriuretic Peptide (NT-proBNP) at Months 6 and 12
Month 6
|
452.9 Nanograms per liter (ng/L)
Standard Deviation 2625.04
|
|
Change From Baseline for N Terminal Prohormone B Type Natriuretic Peptide (NT-proBNP) at Months 6 and 12
Month 12
|
-422.2 Nanograms per liter (ng/L)
Standard Deviation 1581.82
|
SECONDARY outcome
Timeframe: Predose on Day 1 (baseline), predose and 3 hours post dose at Month 1 visit, 7 hours post dose at Month 6 visit, and 1 hour post dose at Month 12 visitPopulation: Pharmacodynamic (PD) analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule and who had at least 1 TTR stabilization value. Number of Participants Analyzed represents the number of participants who had PD samples collected, and both the baseline and post dose TTR and TTR tetramer concentrations \>= the lower limit of quantification (LLOQ). Number Analyzed for each row represents the number of evaluable participants at each timepoint.
Blood sample of approximate 10 mL was collected at baseline, Months 1, 6, and 12 visits for measurement of TTR and TTR tetramer concentrations. TTR concentration was obtained prior to urea denaturation and TTR tetramer concentration was obtained after urea denaturation. Month 1 visit occurred 1 month post Day 1 +/- 1 week, Month 6 visit occurred 6 months post Day 1 +/- 2 weeks, and Month 12 visit occurred 12 months post Day 1 +/- 2 weeks or within 2 weeks after end of treatment. Responder was the participant who achieved TTR stabilization (ie, who had been TTR stabilized). Declaring a participant to have been (TTR) 'stabilized' was defined as the participant whose percent stabilization was equal to or greater than 32%. Percent stabilization (%) was calculated as (\[fraction of initial {FOI} dosed - FOI baseline\] / FOI baseline)×100, and FOI was calculated as average TTR tetramer concentration (post-denaturation) / average TTR concentration (pre-denaturation).
Outcome measures
| Measure |
All Participants
n=30 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
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|---|---|
|
Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12
Predose at Month 1
|
96.4 Percentages of participants
Interval 81.7 to 99.9
|
|
Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12
3 hours post dose at Month 1
|
96.4 Percentages of participants
Interval 81.7 to 99.9
|
|
Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12
7 hours post dose at Month 6
|
96.2 Percentages of participants
Interval 80.4 to 99.9
|
|
Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12
1 hour post dose at Month 12
|
94.7 Percentages of participants
Interval 74.0 to 99.9
|
SECONDARY outcome
Timeframe: Predose on Day 1 (baseline), predose and 3 hours post dose at Month 1 visit, 7 hours post dose at Month 6 visit, and 1 hour post dose at Month 12 visitPopulation: PD analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule and who had at least 1 TTR concentration value. Number of Participants Analyzed represents the total number of participants in the PD analysis set. Number Analyzed for each row represents the number of participants with evaluable values at the specific timepoints.
One K2EDTA blood sample of approximate 10 mL was collected on Day 1 (baseline), and at Months 1, 6, and 12 visits for measurement of TTR concentration. Month 1 visit occurred 1 month post Day 1 +/- 1 week, Month 6 visit occurred 6 months post Day 1 +/- 2 weeks, and Month 12 visit occurred 12 months post Day 1 +/- 2 weeks or within 2 weeks after end of treatment.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
TTR Concentration at Baseline, Months 1, 6, and 12
Predose on Day 1 (baseline)
|
12.70 Milligrams per deciliter (mg/dL)
Standard Deviation 5.670
|
|
TTR Concentration at Baseline, Months 1, 6, and 12
Predose at Month 1
|
19.73 Milligrams per deciliter (mg/dL)
Standard Deviation 7.019
|
|
TTR Concentration at Baseline, Months 1, 6, and 12
3 hours post dose at Month 1
|
19.58 Milligrams per deciliter (mg/dL)
Standard Deviation 6.547
|
|
TTR Concentration at Baseline, Months 1, 6, and 12
7 hours post dose at Month 6
|
21.48 Milligrams per deciliter (mg/dL)
Standard Deviation 7.717
|
|
TTR Concentration at Baseline, Months 1, 6, and 12
1 hour post dose at Month 12
|
21.79 Milligrams per deciliter (mg/dL)
Standard Deviation 7.003
|
SECONDARY outcome
Timeframe: Day 1 (baseline), Months 6, and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule. Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of evaluable participants who had reportable KCCQ-OS scores at baseline, at Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
The KCCQ is a self-administered, 23-item questionnaire. KCCQ-OS scores are transformed to a 0 to 100 range, with lower scores denoting poorer quality of life.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
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|---|---|
|
Change From Baseline for Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Scores at Months 6 and 12
Month 6
|
-0.4 Score on a scale of 100
Standard Deviation 18.97
|
|
Change From Baseline for Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Scores at Months 6 and 12
Month 12
|
-0.3 Score on a scale of 100
Standard Deviation 19.89
|
SECONDARY outcome
Timeframe: Day 1 (baseline), Months 6, and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule. Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of evaluable participants who had reportable EQ-5D-5L scores at baseline, Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
The EQ-5D-5L is a brief, self-administered generic health status instrument. The instrument consists of 2 parts. In the first part, respondents are asked to rate their current health state on 5 dimensions (mobility, self-care, usual activities, pain or discomfort, and anxiety or depression) with each dimension having 5 levels of function (no problems, slight problems, moderate problems, severe problems, and extreme problems). The second part is a participant's self-rating of current health state on a Visual Analog Scale (EQ-5D VAS) with endpoints labeled 'best imaginable health state' (score of 100) and 'worst imaginable health state' (score of 0). The index values were calculated using the scoring algorithm based on preferences solicited from the Chinese population with 0 representing death and 1 representing full health. Higher EQ-5D-5L index value indicated a better quality of life. Change from baseline for EQ-5D-5L index values are reported for this outcome measure.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
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|---|---|
|
Change From Baseline for EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Index Values at Months 6 and 12
Month 6
|
0.0 Index value on a scale of 0-1
Standard Deviation 0.24
|
|
Change From Baseline for EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Index Values at Months 6 and 12
Month 12
|
0.0 Index value on a scale of 0-1
Standard Deviation 0.21
|
SECONDARY outcome
Timeframe: Day 1 (baseline), Months 6, and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule . Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of evaluable participants who had reportable EuroQol VAS scores at baseline, Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
The EQ-5D-5L (5 levels version) is a brief, self-administered generic health status instrument. The instrument consists of 2 parts. In the first part, respondents are asked to rate their current health state on 5 dimensions (mobility, self-care, usual activities, pain or discomfort, and anxiety or depression) with each dimension having 3 levels of function (no problems, slight problems, moderate problems, severe problems and extreme problems). The second part is a participant's self-rating of current health state on a EQ-5D VAS with endpoints labeled 'best imaginable health state' (score of 100) and 'worst imaginable health state' (score of 0). The scores from the 5 dimensions may be used to calculate a single index value, also known as a utility score. EQ-5D VAS scores are reported for this outcome measure.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Change From Baseline for EuroQol VAS at Months 6 and 12
Month 6
|
-0.6 Score on a scale of 100
Standard Deviation 20.23
|
|
Change From Baseline for EuroQol VAS at Months 6 and 12
Month 12
|
3.4 Score on a scale of 100
Standard Deviation 15.65
|
SECONDARY outcome
Timeframe: Day 1 (baseline), Months 6, and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule. Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of evaluable participants who had reportable PCS scores at baseline, Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
SF-12 is developed as a shorter alternative to the SF-36 for use in large-scale studies. It is an instrument with different weights for scoring physical and mental health, and measures health-related quality of life with 12 items categorized in eight areas: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. This questionnaire was completed by the participant after the KCCQ and EQ-5D-5L, and prior to other required visit assessments. PCS score ranges from 0 to 100, and higher PCS score indicates a better quality of life. Change from baseline for PCS for SF-12 is reported for this outcome measure.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Change From Baseline for Physical Component Summary (PCS) for Short-Form Survey 12 (SF-12) at Months 6 and 12
Month 6
|
0.1 Score on a scale of 100
Standard Deviation 8.24
|
|
Change From Baseline for Physical Component Summary (PCS) for Short-Form Survey 12 (SF-12) at Months 6 and 12
Month 12
|
-0.0 Score on a scale of 100
Standard Deviation 9.14
|
SECONDARY outcome
Timeframe: Day 1 (baseline), Months 6, and 12Population: Efficacy analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule. Number of Participants Analyzed represents the total number of participants in the efficacy analysis set regardless of the type of outcome measures. Number Analyzed for each row represents the number of evaluable participants who had reportable MCS scores at baseline, Months 6 (for reporting CFB at Month 6) and 12 (for reporting CFB at Month 12).
SF-12 is developed as a shorter alternative to the SF-36 for use in large-scale studies. It is an instrument with different weights for scoring physical and mental health, and measures health-related quality of life with 12 items categorized in eight areas: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. This questionnaire was completed by the participant after the KCCQ and EQ-5D-5L, and prior to other required visit assessments. MCS score ranges from 0 to 100, and higher MCS score indicates a better quality of life. Change from baseline for MCS for SF-12 is reported for this outcome measure.
Outcome measures
| Measure |
All Participants
n=53 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Change From Baseline for Mental Component Summary (MCS) for SF-12 at Months 6 and 12
Month 6
|
1.4 Score on a scale of 100
Standard Deviation 10.60
|
|
Change From Baseline for Mental Component Summary (MCS) for SF-12 at Months 6 and 12
Month 12
|
1.5 Score on a scale of 100
Standard Deviation 12.54
|
SECONDARY outcome
Timeframe: Predose and 3 hours post dose at Month 1 visit, 7 hours post dose at Month 6 visit, and 1 hour post at Month 12 visitPopulation: Pharmacokinetic (PK) analysis set included all enrolled participants who took at least 1 dose of tafamidis free acid 61 mg soft capsule and who had at least 1 quantifiable plasma tafamidis concentration. Number of Participants Analyzed represents the number of participants who had at least 1 tafamidis concentration \>=LLOQ. Number Analyzed for each row represents the number of participants with non-missing concentrations at each timepoint.
Blood samples of approximately 3 mL, to provide an approximately 1 mL plasma, were collected for measurement of plasma concentrations of tafamidis at Months 1, 6, and 12 visits. Month 1 visit occurred 1 month post Day 1 +/- 1 week, Month 6 visit occurred 6 months post Day 1 +/- 2 weeks, and Month 12 visit occurred 12 months post Day 1 +/- 2 weeks or within 2 weeks after end of treatment.
Outcome measures
| Measure |
All Participants
n=47 Participants
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Plasma Concentrations of Tafamidis at Months 1, 6, and 12
Predose at Month 1
|
10410 nanograms per milliliter (ng/mL)
Standard Deviation 5521.1
|
|
Plasma Concentrations of Tafamidis at Months 1, 6, and 12
3 hours post dose at Month 1
|
12040 nanograms per milliliter (ng/mL)
Standard Deviation 4950.1
|
|
Plasma Concentrations of Tafamidis at Months 1, 6, and 12
7 hours post dose at Month 6
|
14100 nanograms per milliliter (ng/mL)
Standard Deviation 5950.5
|
|
Plasma Concentrations of Tafamidis at Months 1, 6, and 12
1 hour post dose at Month 12
|
11950 nanograms per milliliter (ng/mL)
Standard Deviation 5475.8
|
Adverse Events
All Participants
Serious adverse events
| Measure |
All Participants
n=53 participants at risk
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Cardiac disorders
Atrial thrombosis
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Cardiac disorders
Cardiac failure
|
11.3%
6/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Cardiac disorders
Cardiac failure acute
|
5.7%
3/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Cardiac disorders
Cardiorenal syndrome
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Congenital, familial and genetic disorders
Hereditary neuropathic amyloidosis
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Gastrointestinal disorders
Vomiting
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
General disorders
Disease progression
|
3.8%
2/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
General disorders
Oedema peripheral
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
COVID-19
|
5.7%
3/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
Erysipelas
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
Gastroenteritis
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
Pneumonia
|
3.8%
2/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
Respiratory tract infection
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Metabolism and nutrition disorders
Electrolyte imbalance
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Nervous system disorders
Dizziness
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Nervous system disorders
Haemorrhagic cerebral infarction
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Nervous system disorders
Syncope
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Renal and urinary disorders
Chronic kidney disease
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.9%
1/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
Other adverse events
| Measure |
All Participants
n=53 participants at risk
Participants received tafamidis free acid 61 milligrams (mg) once daily (QD). Participants were instructed to take the study intervention on a daily basis (up to a maximum of 1 year).
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
11.3%
6/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Gastrointestinal disorders
Vomiting
|
7.5%
4/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Hepatobiliary disorders
Hepatic function abnormal
|
7.5%
4/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
COVID-19
|
22.6%
12/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
Nasopharyngitis
|
7.5%
4/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Infections and infestations
Pneumonia
|
5.7%
3/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
9.4%
5/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Renal and urinary disorders
Haematuria
|
5.7%
3/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.7%
3/53 • From the first dose of study intervention up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for AEs.
|
Additional Information
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