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| ID | Type | Description | Link |
|---|---|---|---|
| B3461025 |
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Open-label, multicenter, international, single-treatment study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients with V122I or wild-type TTR amyloid cardiomyopathy.
The study will be conducted in two parts. Part 1 will include a six-week dosing period during which all enrolled patients will self-administer oral Fx-1006A 20 mg soft gelatin capsules once daily for six weeks. At Week 6, blood samples will be collected from each patient to determine TTR stabilization. Patients who complete the Week 6 visit will continue taking daily oral Fx 1006A 20 mg for up to a total of 12 months during Part 2 of this study. If it is determined that a patient is not stabilized at Week 6 (based on TTR stabilization data), the patient will be discontinued from the study. Safety and clinical outcomes will be evaluated during Part 2 of this study.
Two whole blood samples for pharmacodynamic assessments (TTR stabilization) and pharmacokinetic assessments (Fx-1006A concentrations as well as calculated steady-state parameters) will be collected at Baseline and Week 6. At Months 6 and 12, two whole blood samples will be collected for pharmacodynamic assessments, and four whole blood samples (two samples per time point) will be collected for pharmacokinetic assessments to be utilized in population pharmacokinetic modeling.
Echocardiography, chest x-ray, cardiac MRI, and 24-hour Holter monitoring will be conducted at Baseline, and Months 6 and 12. Six-minute walk test and quality of life utilizing the Patient Global Assessment, KCCQ, and SF-36 will be assessed at Baseline, and Months 3, 6, and 12. NYHA Classification will be assessed at Baseline, Week 6, and Months 3, 6, and 12. Serum markers of troponin I and T, and NT-pro-BNP levels will be assessed at each study visit.
Safety and tolerability will be assessed throughout the study. Vital signs, 12-lead ECG, blood and urine samples for clinical laboratory tests (serum chemistry, hematology, coagulation panel, and urinalysis), AEs, and concomitant medications (including diuretic usage) will be assessed at each study visit. Abbreviated physical examinations will be conducted at Baseline, Weeks 2 and 6, and Months 3 and 6, and a complete physical examination will be conducted at Month 12.
Clinic visits will be conducted during Screening (Days -30 to -1) and Baseline (Day 0); procedures scheduled for the Baseline visit may be conducted over a period of one week to accommodate patient scheduling. All Baseline procedures must be completed prior to the first self-administered dose on Day 1. Day 1 will be defined as administration of the first dose of study medication, which patients will self-administer at home. During treatment, clinic visits will be conducted at Week 2 (± 2 days), Week 6 (± 1 week), Month 3 (± 1 week), Month 6 (± 2 weeks), and Month 12 (± 2 weeks). Procedures scheduled for the Month 6 and 12 visits may occur over one week during the visit window to accommodate patient scheduling. Monthly telephone contacts (± 1 week of the scheduled date) will be made during months in which no clinical site visits are scheduled (Months 4, 5, 7, 8, 9, 10, and 11) for assessment of AEs and concomitant medications. A final telephone contact to assess AEs and concomitant medication usage will be made 30 days after the last dose of study medication for each patient.
Patients who discontinue from the study at any time will have a final visit performed, including all safety assessments, at the time of discontinuation. Any patient discontinuing after the Month 6 visit will also have all exploratory assessments performed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fx-1006A | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fx-1006A | Drug | Fx-1006A 20mg soft gelatin capsules once daily (at the same time each day) for 12 months |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Week 6 | 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. | Week 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Month 6 and 12 | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | 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. | Baseline up to 30 days after the last dose |
Inclusion Criteria:
Patient is > 40 years-old.
Patient participated in FoldRx Study Fx-001 (TRACS) OR Patient has documented TTR amyloid cardiomyopathy and NYHA Classification of I or II.
TTR amyloid cardiomyopathy is defined as:
Patient's symptoms of congestive heart failure (CHF) have been optimally managed prior to baseline, as assessed by the Principal Investigator. Optimal CHF management includes stable drug regimen for ≥ 4 weeks prior to enrollment and stable dose of beta blocker for ≥ 3 months prior to enrollment.
If female, patient is post-menopausal. If male with a female partner of childbearing potential, willing to use an acceptable method of birth control for the duration of the study and for at least 3 months after the last dose of study medication.
Patient is, in the opinion of the Investigator, willing and able to comply with the study medication regimen and all other study requirements
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jeff Packman, MBA | FoldRx Pharmaceuticals, Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Emory University School of Medicine | Atlanta | Georgia | 30322 | United States | ||
| University of Chicago |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28951660 | Derived | Sultan MB, Gundapaneni B, Schumacher J, Schwartz JH. Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin Cardiomyopathy. Clin Med Insights Cardiol. 2017 Sep 18;11:1179546817730322. doi: 10.1177/1179546817730322. eCollection 2017. | |
| 25872787 | Derived | Maurer MS, Grogan DR, Judge DP, Mundayat R, Packman J, Lombardo I, Quyyumi AA, Aarts J, Falk RH. Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail. 2015 May;8(3):519-26. doi: 10.1161/CIRCHEARTFAILURE.113.000890. Epub 2015 Apr 14. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Tafamidis | Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1 (up to Week 6) |
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| Month 6, Month 12 |
| Number of Participants With Greater Than or Equal to (>=) Grade 3 Treatment-Emergent AEs | 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. | Baseline up to 30 days after the last dose |
| Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | ECHO:investigator assessed test to assess cardiac function.ECHO abnormality criteria:any/valvular abnormality,pericardial effusion,abnormal regional wall motion,inferior vena cava respiratory variation,posterior left ventricular wall/septal thickness>=13 millimeter(mm),right ventricular thickness>=7mm,ejection fraction <50%, ratio of early (E) diastolic transmitral flow and atrial(A) contraction velocity (E/A)>=2, ratio of 'E'to lateral/septal mitral annular velocity (e') (E/e'prime lateral>15, E/e'prime septal>15), E deceleration time<=150 millisecond(msec),Isovolumic relaxation time<=70msec. | Baseline up to Month 12 |
| Number of Participants Discontinuing From The Study Due to Clinically Significant Clinical or Laboratory Adverse Events (AEs) | Baseline up to Month 12 |
| Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 | Echocardiography was used to measure interventricular septal thickness (IVST), posterior left ventricular wall thickness (PLVWT), right ventricular wall thickness (RVWT), left atrial diameter (LAD): anterior-posterior (ant-post), medio-lateral, superior-inferior (sup-inf) and left ventricular end diastolic diameter (LVEDD). | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricular Mass (LVM) at Month 6 and 12 | LVM was defined as increase in the mass of left ventricle, estimated by echocardiography. Increased LVM was associated with cardiovascular morbidity and mortality. | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12 | Left ventricular ejection fraction (LVEF) was the fraction of the end-diastolic volume (EDV) that is ejected out of left ventricle with each contraction, estimated by echocardiography. EDV is the volume of blood within a ventricle immediately before a contraction. | Baseline, Month 6, Month 12 |
| Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 | Doppler echocardiography was a procedure which used ultrasound technology to examine the heart. Ratio of early (E) diastolic transmitral flow velocity and atrial (A) contraction velocity (E/A) and ratio of the early (E) diastolic transmitral flow velocity to the mitral annular velocity (e') (E/e') were estimated. | Baseline, Month 6, Month 12 |
| Change From Baseline in Doppler Data: Mitral Deceleration Time at Month 6 and 12 | Doppler echocardiography was a procedure which used ultrasound technology to examine the heart. The mitral deceleration time was the time taken from the maximum E wave to baseline. E wave arises due to early diastolic filling. | Baseline, Month 6, Month 12 |
| Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 | Tissue Doppler used doppler principles to measure the annular velocities at the lateral and septal areas of the mitral annulus. s': systolic velocity during ejection, e': early diastolic mitral annular velocity, a': late diastolic mitral annular velocity. | Baseline, Month 6, Month 12 |
| Change From Baseline in Pericardial Effusion at Month 6 and 12 | Pericardial effusion was the presence of an abnormal amount of fluid in the pericardial cavity, as determined by echocardiography. | Baseline, Month 6, Month 12 |
| Number of Participants With Change From Baseline in Valvular Abnormalities at Month 6 and 12 | Valvular abnormalities were those abnormalities (thickening or regurgitation) that involved one or more valves of the heart, determined by echocardiography. | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricular Anteroseptal, Left Ventricular Inferolateral Wall Thickness and Right Ventricular End Diastolic Free Wall Thickness at Month 6 and 12 | Cardiac Magnetic Resonance Imaging (MRI) was done to measure the thickness of left ventricular anteroseptal (LVAS) wall, left ventricular inferolateral (LVIL) wall and right ventricular end diastolic free (RVEDF) wall. | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricular Mass, Mass of Left Ventricular Myocardium With Amyloidosis, Mass of Left Ventricular Myocardium With Fibrosis/Scar and Right Ventricular End Diastolic Mass at Month 6 and 12 | Cardiac MRI was done to measure LVM, mass of left ventricular (LV) myocardium with amyloidosis, mass of LV myocardium with fibrosis/scar and right ventricular end diastolic mass (RVEDM). | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricle End Diastolic Volume, Left Ventricle End Systolic Volume, Left Ventricle Stroke Volume, Right Ventricle End Diastolic Volume, Right Ventricle End Systolic Volume, Right Ventricle Stroke Volume at Month 6 and 12. | Cardiac MRI was done to measure left ventricle end diastolic volume (LVEDV), left ventricle end systolic volume (LVESV), left ventricle stroke volume (LVSV), right ventricle end diastolic volume (RVEDV), right ventricle end systolic volume (RVESV) and right ventricle stroke volume (RVSV). | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 | Cardiac MRI was done to measure: left ventricular ejection fraction (LVEF) was the fraction of the EDV that is ejected out of left ventricle with each contraction and right ventricular ejection fraction (RVEF) was the fraction of the EDV that is ejected out of right ventricle with each contraction. EDV is the volume of blood within a ventricle immediately before a contraction. | Baseline, Month 6, Month 12 |
| Change From Baseline in Left Ventricular Cardiac Output and Right Ventricular Cardiac Output at Month 6 and 12 | Cardiac MRI was done to measure cardiac output, which was the volume of blood being pumped by the heart, in particular by the left or right ventricle in the time interval of one minute. | Baseline, Month 6, Month 12 |
| Change From Baseline in Percentage of Left Ventricular Myocardial Mass With Amyloidosis and Left Ventricular Myocardial Mass With Fibrosis/Scar at Month 6 and 12 | Cardiac MRI was done to measure percentage of LV myocardial mass with amyloidosis and LV myocardial mass with fibrosis/scar. LV myocardial mass with amyloidosis or fibrosis/scar was calculated from the product of the myocardial volume and specific gravity of heart muscle, in participants with amyloidosis or fibrosis/scar, respectively. | Baseline, Month 6, Month 12 |
| Change From Baseline in 4 Chamber Interatrial Septal Thickness at Month 6 and 12 | Cardiac MRI was done to measure interatrial septal thickness in the 4 chamber view. | Baseline, Month 6, Month 12 |
| Change From Baseline in 4 Chamber Left Atrial Dimension and 4 Chamber Right Atrial Dimension at Month 6 and 12 | Cardiac MRI was done to measure the left and right atrial dimensions which have diagnostic and prognostic significance in cardiology, in the 4 chamber view. | Baseline, Month 6, Month 12 |
| Number of Participants With Atrial Fibrillation/Flutter, Atrial Tachycardia, Non-Sustained Ventricular Tachycardia (NSVT) Beats), Sustained Ventricular Tachycardia (SVT), Sinus Pause at Month 6 and 12 | Holter monitor was a machine that recorded the heart rhythms. Holter monitoring abnormalities of atrial fibrillation/flutter (rapid, irregular heart rhythm), atrial tachycardia (rapid cardiac rate), non-sustained ventricular tachycardia (NSVT)<30 beats, sustained ventricular tachycardia (SVT) >=30 beats and sinus pause (transient interruption in the sinus rhythm) were recorded. | Baseline, Month 6, Month 12 |
| 24-Hour Average Heart Rate and Maximium/Minimum Heart Rate | Holter monitor was a machine that recorded the heart rhythms. 24-hour average heart rate and maximium/minimum heart rate was recorded using Holter monitoring. | Baseline, Month 6, Month 12 |
| Number of Participants With Complete Heart Block | Complete heart block is the third-degree atrioventricular block in which the impulse generated in the sinoatrial node in the atrium does not propagate to the ventricles. | Baseline, Month 6, Month 12 |
| Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters | Holter monitor was a machine that recorded the heart rhythms. HRV time-domain indices were summarized for root-mean-square of successive differences [RMS SD] of the R-R intervals (R-R is the interval between successive Rs in the ECG wave) between normal beats (NN), magid standard deviation (Magid SD) of normal to normal R-R intervals and Kleiger standard deviation of normal to normal R-R intervals (Kleiger SD). The term 'NN' is used in place of 'R-R' when the processed beats are normal beats. | Baseline, Month 6, Month 12 |
| Heart Rate Variability- Percentage of Successive R-R Intervals With Greater Than 50 Msec Difference Between Normal Beats (pNN50) | Holter monitor was a machine that recorded the heart rhythms. The term 'NN' was used in place of 'R-R' when the processed beats are normal beats. The percentage of successive R-R intervals with greater than 50 msec difference between normal beats was derived by dividing NN50 by the total number of NN intervals (pNN50), where NN50 was the number of interval differences of successive NN intervals greater than 50 msec. | Baseline, Month 6, Month 12 |
| Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 | NYHA: classified as 'class I' (participants with cardiac disease but without resulting limitations of physical activity), 'class II' (participants with cardiac disease resulting in slight limitation of physical activity), 'class III' (participants with cardiac disease resulting in marked limitation of physical activity), 'class IV' (participants with cardiac disease resulting in inability to carry on any physical activity without discomfort). Participants with change from baseline were classified as 'improved' (positive change), 'no change' or 'worsened' (negative change). | Baseline, Week 6, Month 3, Month 6, Month 12 |
| Cardiothoracic (CT) Ratio | Cardiothoracic ratio was defined as the transverse diameter of the heart, compared with that of the thoracic cage, used to help determine enlargement of the heart. | Baseline, Month 6, Month 12 |
| Number of Participants With Increased Interstitial Markings and Pleural Effusions | Chest x-ray was done to record the presence of increased interstitial markings (a large number of interstitial markings was indicative of abnormality in the lung) and pleural effusion, which was defined as accumulation of fluid between the layers of tissue that line the lungs and chest cavity. | Baseline, Month 6, Month 12 |
| Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 | Participant's overall quality of life was measured by the PtGA. At baseline participants answered to question: "in general, how do you feel today?" - on a 5-point scale from '1' (excellent) to '5' (poor). At each follow-up visit, participant's answered to question: "How do you feel today as compared to when we talked with you at your last clinic visit for this study?" on a 7-point scale- '1' markedly improved, '2' moderately improved, '3' mildly improved, '4' unchanged, '5' mildly worsened, '6' moderately worsened, '7' markedly worsened. | Baseline, Month 3, Month 6, Month 12 |
| Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 | KCCQ was a 23-item heart failure specific questionnaire quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life. Summary scores were scaled to range from 0 to 100, with higher scores representing greater disability. | Baseline, Month 3, Month 6, Month 12 |
| Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 | SF-36 was standardized survey evaluating 8 aspects of functional health and well being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. Scores for the 8 domains range from 0-100, where higher scores were better (100=highest level of functioning) and reported as 2 summary scores; Mental Component Score (MCS) and Physical Component Score (PCS). The score for a section was an average of the individual question scores, which were scaled 0-100, where higher scores were better. | Baseline, Month 3, Month 6, Month 12 |
| Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 | Troponin I and troponin T were the cardiac markers. Troponin I and troponin T were part of the troponin complex, where troponin I was bound to actin in thin myofilaments and troponin T was bound to tropomyosin. Higher level of these markers was indicative of heart damage. | Baseline, Week 2, Week 6, Month 3, Month 6, Month 12 |
| Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 | NT-proBNP was a cardiac marker which had the prognostic value for participants with heart failure or left ventricular dysfunction. Higher level of the marker was indicative of heart damage. | Baseline, Week 2, Week 6, Month 3, Month 6, Month 12 |
| Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12 | 6MWT was used to assess the distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed. Continuous pulse oximetry was conducted during the test for safety.. The distance walked in 6 minutes was categorized as: Level 1: <300 meter, Level 2: 300-374.9 meter, Level 3: 375-449.9 meter, Level 4: >=450 meter. | Baseline, Month 3, Month 6, Month 12 |
| Chicago |
| Illinois |
| 60637 |
| United States |
| Johns Hopkins Hospital | Baltimore | Maryland | 21205 | United States |
| Harvard Vanguard Medical Associates | Boston | Massachusetts | 02215 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Columbia University | New York | New York | 10032 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| Part 2 (After Week 6 up to Month 12) |
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| ID | Title | Description |
|---|---|---|
| BG000 | Tafamidis | Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2. |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
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| Age Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
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| Primary | Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Week 6 | 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. | Intent-to-Treat (ITT) population included all participants who received at least one dose of study medication. | Posted | Number | 95% Confidence Interval | Percentage of participants | Week 6 |
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| Secondary | Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Month 6 and 12 | 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. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. | Posted | Number | 95% Confidence Interval | Percentage of participants | Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Treatment-Emergent Adverse Events (AEs) | 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. | ITT population included all participants who received at least one dose of study medication. | Posted | Number | Participants | Baseline up to 30 days after the last dose |
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| Other Pre-specified | Number of Participants With Greater Than or Equal to (>=) Grade 3 Treatment-Emergent AEs | 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. | ITT population included all participants who received at least one dose of study medication. | Posted | Number | Participants | Baseline up to 30 days after the last dose |
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| Other Pre-specified | Number of Participants With Clinically Significant Treatment-Emergent Echocardiography (ECHO) Findings | ECHO:investigator assessed test to assess cardiac function.ECHO abnormality criteria:any/valvular abnormality,pericardial effusion,abnormal regional wall motion,inferior vena cava respiratory variation,posterior left ventricular wall/septal thickness>=13 millimeter(mm),right ventricular thickness>=7mm,ejection fraction <50%, ratio of early (E) diastolic transmitral flow and atrial(A) contraction velocity (E/A)>=2, ratio of 'E'to lateral/septal mitral annular velocity (e') (E/e'prime lateral>15, E/e'prime septal>15), E deceleration time<=150 millisecond(msec),Isovolumic relaxation time<=70msec. | ITT population. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal(that is treatment-emergent abnormalities). | Posted | Number | Participants | Baseline up to Month 12 |
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| Other Pre-specified | Number of Participants Discontinuing From The Study Due to Clinically Significant Clinical or Laboratory Adverse Events (AEs) | ITT population included all participants who received at least one dose of study medication. | Posted | Number | Participants | Baseline up to Month 12 |
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| Other Pre-specified | Change From Baseline in Echocardiographic (ECHO) Parameters at Month 6 and 12 | Echocardiography was used to measure interventricular septal thickness (IVST), posterior left ventricular wall thickness (PLVWT), right ventricular wall thickness (RVWT), left atrial diameter (LAD): anterior-posterior (ant-post), medio-lateral, superior-inferior (sup-inf) and left ventricular end diastolic diameter (LVEDD). | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | millimeter (mm) | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricular Mass (LVM) at Month 6 and 12 | LVM was defined as increase in the mass of left ventricle, estimated by echocardiography. Increased LVM was associated with cardiovascular morbidity and mortality. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Gram | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12 | Left ventricular ejection fraction (LVEF) was the fraction of the end-diastolic volume (EDV) that is ejected out of left ventricle with each contraction, estimated by echocardiography. EDV is the volume of blood within a ventricle immediately before a contraction. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Percentage of EDV | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Doppler Data: E/A and E/e' Ratio at Month 6 and 12 | Doppler echocardiography was a procedure which used ultrasound technology to examine the heart. Ratio of early (E) diastolic transmitral flow velocity and atrial (A) contraction velocity (E/A) and ratio of the early (E) diastolic transmitral flow velocity to the mitral annular velocity (e') (E/e') were estimated. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Ratio | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Doppler Data: Mitral Deceleration Time at Month 6 and 12 | Doppler echocardiography was a procedure which used ultrasound technology to examine the heart. The mitral deceleration time was the time taken from the maximum E wave to baseline. E wave arises due to early diastolic filling. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | msec | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Tissue Doppler- Septal and Lateral Velocity at Month 6 and 12 | Tissue Doppler used doppler principles to measure the annular velocities at the lateral and septal areas of the mitral annulus. s': systolic velocity during ejection, e': early diastolic mitral annular velocity, a': late diastolic mitral annular velocity. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific categories. | Posted | Mean | Standard Deviation | centimeter/second (cm/sec) | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Pericardial Effusion at Month 6 and 12 | Pericardial effusion was the presence of an abnormal amount of fluid in the pericardial cavity, as determined by echocardiography. | Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis. | Posted | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Change From Baseline in Valvular Abnormalities at Month 6 and 12 | Valvular abnormalities were those abnormalities (thickening or regurgitation) that involved one or more valves of the heart, determined by echocardiography. | Data for this pre-specified outcome measure was collected and reported in individual participant listings but not statistically summarized for analysis. | Posted | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricular Anteroseptal, Left Ventricular Inferolateral Wall Thickness and Right Ventricular End Diastolic Free Wall Thickness at Month 6 and 12 | Cardiac Magnetic Resonance Imaging (MRI) was done to measure the thickness of left ventricular anteroseptal (LVAS) wall, left ventricular inferolateral (LVIL) wall and right ventricular end diastolic free (RVEDF) wall. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | mm | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricular Mass, Mass of Left Ventricular Myocardium With Amyloidosis, Mass of Left Ventricular Myocardium With Fibrosis/Scar and Right Ventricular End Diastolic Mass at Month 6 and 12 | Cardiac MRI was done to measure LVM, mass of left ventricular (LV) myocardium with amyloidosis, mass of LV myocardium with fibrosis/scar and right ventricular end diastolic mass (RVEDM). | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Gram | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricle End Diastolic Volume, Left Ventricle End Systolic Volume, Left Ventricle Stroke Volume, Right Ventricle End Diastolic Volume, Right Ventricle End Systolic Volume, Right Ventricle Stroke Volume at Month 6 and 12. | Cardiac MRI was done to measure left ventricle end diastolic volume (LVEDV), left ventricle end systolic volume (LVESV), left ventricle stroke volume (LVSV), right ventricle end diastolic volume (RVEDV), right ventricle end systolic volume (RVESV) and right ventricle stroke volume (RVSV). | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | mL | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricular Ejection Fraction and Right Ventricular Ejection Fraction at Month 6 and 12 | Cardiac MRI was done to measure: left ventricular ejection fraction (LVEF) was the fraction of the EDV that is ejected out of left ventricle with each contraction and right ventricular ejection fraction (RVEF) was the fraction of the EDV that is ejected out of right ventricle with each contraction. EDV is the volume of blood within a ventricle immediately before a contraction. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Percentage of EDV | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Left Ventricular Cardiac Output and Right Ventricular Cardiac Output at Month 6 and 12 | Cardiac MRI was done to measure cardiac output, which was the volume of blood being pumped by the heart, in particular by the left or right ventricle in the time interval of one minute. | MRI data was collected and reported for cardiac output through the measure of stroke volume as given in outcome measure 17. | Posted | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Percentage of Left Ventricular Myocardial Mass With Amyloidosis and Left Ventricular Myocardial Mass With Fibrosis/Scar at Month 6 and 12 | Cardiac MRI was done to measure percentage of LV myocardial mass with amyloidosis and LV myocardial mass with fibrosis/scar. LV myocardial mass with amyloidosis or fibrosis/scar was calculated from the product of the myocardial volume and specific gravity of heart muscle, in participants with amyloidosis or fibrosis/scar, respectively. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Percentage of LVM | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in 4 Chamber Interatrial Septal Thickness at Month 6 and 12 | Cardiac MRI was done to measure interatrial septal thickness in the 4 chamber view. | Data for this measure was not collected due to a change in the planned analysis. | Posted | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in 4 Chamber Left Atrial Dimension and 4 Chamber Right Atrial Dimension at Month 6 and 12 | Cardiac MRI was done to measure the left and right atrial dimensions which have diagnostic and prognostic significance in cardiology, in the 4 chamber view. | Data for this measure was not collected due to a change in the planned analysis. | Posted | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Atrial Fibrillation/Flutter, Atrial Tachycardia, Non-Sustained Ventricular Tachycardia (NSVT) Beats), Sustained Ventricular Tachycardia (SVT), Sinus Pause at Month 6 and 12 | Holter monitor was a machine that recorded the heart rhythms. Holter monitoring abnormalities of atrial fibrillation/flutter (rapid, irregular heart rhythm), atrial tachycardia (rapid cardiac rate), non-sustained ventricular tachycardia (NSVT)<30 beats, sustained ventricular tachycardia (SVT) >=30 beats and sinus pause (transient interruption in the sinus rhythm) were recorded. | ITT population. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. The 'n' for any post-dose incidence included participants with baseline values that were not abnormal(that is treatment-emergent abnormalities). | Posted | Number | Participants | Baseline, Month 6, Month 12 |
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| Other Pre-specified | 24-Hour Average Heart Rate and Maximium/Minimum Heart Rate | Holter monitor was a machine that recorded the heart rhythms. 24-hour average heart rate and maximium/minimum heart rate was recorded using Holter monitoring. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | beats per minute (bpm) | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Complete Heart Block | Complete heart block is the third-degree atrioventricular block in which the impulse generated in the sinoatrial node in the atrium does not propagate to the ventricles. | No summary was prepared for this data as there were no reports of complete heart block. | Posted | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Heart Rate Variability (HRV)- Standard Deviation (SD) Parameters | Holter monitor was a machine that recorded the heart rhythms. HRV time-domain indices were summarized for root-mean-square of successive differences [RMS SD] of the R-R intervals (R-R is the interval between successive Rs in the ECG wave) between normal beats (NN), magid standard deviation (Magid SD) of normal to normal R-R intervals and Kleiger standard deviation of normal to normal R-R intervals (Kleiger SD). The term 'NN' is used in place of 'R-R' when the processed beats are normal beats. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | msec | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Heart Rate Variability- Percentage of Successive R-R Intervals With Greater Than 50 Msec Difference Between Normal Beats (pNN50) | Holter monitor was a machine that recorded the heart rhythms. The term 'NN' was used in place of 'R-R' when the processed beats are normal beats. The percentage of successive R-R intervals with greater than 50 msec difference between normal beats was derived by dividing NN50 by the total number of NN intervals (pNN50), where NN50 was the number of interval differences of successive NN intervals greater than 50 msec. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Percentage of intervals | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Week 6, Month 3, 6 and 12 | NYHA: classified as 'class I' (participants with cardiac disease but without resulting limitations of physical activity), 'class II' (participants with cardiac disease resulting in slight limitation of physical activity), 'class III' (participants with cardiac disease resulting in marked limitation of physical activity), 'class IV' (participants with cardiac disease resulting in inability to carry on any physical activity without discomfort). Participants with change from baseline were classified as 'improved' (positive change), 'no change' or 'worsened' (negative change). | ITT population included all participants who received at least one dose of study medication. Data at Week 6 was collected but was not summarized due to a change in the planned analysis. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Number | Participants | Baseline, Week 6, Month 3, Month 6, Month 12 |
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| Other Pre-specified | Cardiothoracic (CT) Ratio | Cardiothoracic ratio was defined as the transverse diameter of the heart, compared with that of the thoracic cage, used to help determine enlargement of the heart. | ITT population included all participants who received at least one dose of study medication. | Posted | Mean | Standard Deviation | Ratio | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Increased Interstitial Markings and Pleural Effusions | Chest x-ray was done to record the presence of increased interstitial markings (a large number of interstitial markings was indicative of abnormality in the lung) and pleural effusion, which was defined as accumulation of fluid between the layers of tissue that line the lungs and chest cavity. | Intent to Treat (ITT) population included all participants who received at least one dose of study medication. | Posted | Number | Participants | Baseline, Month 6, Month 12 |
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| Other Pre-specified | Number of Participants With Change in Patient Global Assessment (PtGA) at Month 3, 6 and 12 | Participant's overall quality of life was measured by the PtGA. At baseline participants answered to question: "in general, how do you feel today?" - on a 5-point scale from '1' (excellent) to '5' (poor). At each follow-up visit, participant's answered to question: "How do you feel today as compared to when we talked with you at your last clinic visit for this study?" on a 7-point scale- '1' markedly improved, '2' moderately improved, '3' mildly improved, '4' unchanged, '5' mildly worsened, '6' moderately worsened, '7' markedly worsened. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Number | Participants | Baseline, Month 3, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score at Month 3, 6 and 12 | KCCQ was a 23-item heart failure specific questionnaire quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life. Summary scores were scaled to range from 0 to 100, with higher scores representing greater disability. | ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Month 3, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in the Short Form 36 (SF-36) at Month 3, 6 and 12 | SF-36 was standardized survey evaluating 8 aspects of functional health and well being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. Scores for the 8 domains range from 0-100, where higher scores were better (100=highest level of functioning) and reported as 2 summary scores; Mental Component Score (MCS) and Physical Component Score (PCS). The score for a section was an average of the individual question scores, which were scaled 0-100, where higher scores were better. | ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints. Data was collected at Month 3 but not statistically summarized as planned. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Month 3, Month 6, Month 12 |
|
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| Other Pre-specified | Change From Baseline in Troponin I and Troponin T at Week 2, 6, Month 3, 6 and 12 | Troponin I and troponin T were the cardiac markers. Troponin I and troponin T were part of the troponin complex, where troponin I was bound to actin in thin myofilaments and troponin T was bound to tropomyosin. Higher level of these markers was indicative of heart damage. | ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | nanogram/milliliter (ng/mL) | Baseline, Week 2, Week 6, Month 3, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide(NT-proBNP) Levels at Week 2, 6, Month 3, 6 and 12 | NT-proBNP was a cardiac marker which had the prognostic value for participants with heart failure or left ventricular dysfunction. Higher level of the marker was indicative of heart damage. | ITT population included all participants who received at least one dose of study medication. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | picogram/mL (pg/mL) | Baseline, Week 2, Week 6, Month 3, Month 6, Month 12 |
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| Other Pre-specified | Change From Baseline in 6-Minute Walk Test (6MWT) at Month 3, 6 and 12 | 6MWT was used to assess the distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed. Continuous pulse oximetry was conducted during the test for safety.. The distance walked in 6 minutes was categorized as: Level 1: <300 meter, Level 2: 300-374.9 meter, Level 3: 375-449.9 meter, Level 4: >=450 meter. | ITT population included all participants who received at least one dose of study medication. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. Here 'n' signifies those participants who were evaluable for specific timepoints. | Posted | Mean | Standard Deviation | meters | Baseline, Month 3, Month 6, Month 12 |
|
|
Not provided
The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Tafamidis | Participants with variant transthyretin (TTR) genotype (valine replaced at position 122 by isoleucine or V122I) and wild-type TTR genotype received tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily up to Week 6 in Part 1 and participants who achieved TTR stabilization at Week 6 continued to receive tafamidis (Fx-1006A) 20 mg capsule orally once daily up to Month 12 in Part 2. | 15 | 35 | 34 | 35 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac failure congestive | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Amyloidosis | Immune system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Glioblastoma multiforme | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Coordination abnormal | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Haemorrhagic stroke | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Metabolic encephalopathy | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA v 10.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Atrial tachycardia | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Bundle branch block right | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Sinus arrhythmia | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Colour blindness | Congenital, familial and genetic disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Corneal disorder | Eye disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Vitreous floaters | Eye disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cheilitis | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Faecal incontinence | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Oedema | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Thirst | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Catheter related complication | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hernia | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hot flush | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Localised oedema | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Furuncle | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Nail infection | Infections and infestations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Joint sprain | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Post procedural haematuria | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Tooth injury | Injury, poisoning and procedural complications | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Work increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood cholesterol increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood thyroid stimulating hormone increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Aspiration pleural cavity | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood pressure decreased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood thyroid stimulating hormone decreased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood urine present | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blood natriuretic peptide increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Ejection fraction decreased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Urine output increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Venous pressure jugular increased | Investigations | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Anorexia | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Fluid retention | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hypochloraemia | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Finger deformity | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Benign neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Lentigo | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dizziness postural | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Aguesia | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Disturbance in attention | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Memory impairment | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Neuropathy | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Restless legs syndrome | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Nervousness | Psychiatric disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Nipple pain | Reproductive system and breast disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Gynaecomastia | Reproductive system and breast disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Scrotal oedema | Reproductive system and breast disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Postnasal drip | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Foot operation | Surgical and medical procedures | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Implantable defibrillator insertion | Surgical and medical procedures | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Mole excision | Surgical and medical procedures | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Tooth extraction | Surgical and medical procedures | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Varicose vein | Vascular disorders | MedDRA v 10.0 | Non-systematic Assessment |
| |
| Bundle branch block | Cardiac disorders | MedDRA v 10.0 | Non-systematic Assessment |
|
Results for Holter monitoring parameters, increased interstitial markings, pleural effusions, PtGA and cardiothoracic ratio are presented as absolute values at specified time points and not as change from baseline as planned.
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C547076 | tafamidis |
Not provided
Not provided
Not provided
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