Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 6,718
- 试验地点
- 112
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate. The principal aims of this outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogenous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.
详细描述
n/a NA
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must meet all of the following inclusion criteria to be eligible for inclusion into THAOS:
- •Evidence of a personally signed and dated informed consent document indicating that the participant (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
- •Males and females greater than or equal to 18 years of age.
- •Confirmed genotyped TTR mutation with or without a diagnosis of hereditary or wild-type ATTR amyloidosis. Confirmation of ATTRwt amyloidosis will be determined by genotyped confirmation that patient does not possess a known mutation in TTR gene (ie, is a carrier of wild-type allele only) via genetic testing and one of the following set of criteria (a, b, or c):
- •Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
- •Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of >12 mm, and presence of amyloid in non-cardiac tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
- •Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of >12 mm, and presence of amyloid in cardiac tissue indirectly confirmed by scintigraphy with a "bone seeking tracer" eg, 99mTC-DPD [99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid], 99mTC- PYP [Pyrophosphate], and 99mTC-HMDP [hydroxymethylene diphosphonate] with Perugini grade greater than or equal to
- •Exclusion Criteria
- •Patients meeting any of the following will not be included in the study:
- •Patient has evidence of primary (light chain) or secondary amyloidosis.
排除标准
- 未提供
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)
An AE was any untoward medical occurrence in a participant who administered a medicinal product without regard to possibility of causal relationship with the study treatment. SAE was defined as one of the following: resulted in death; was life-threatening (immediate risk of death); requires inpatient hospitalization or prolongation of hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); constituted a congenital anomaly/birth defect. Treatment-emergent AE was defined as an AE with onset date occurring during the on-treatment period. AEs included all SAEs and non-SAEs.
Number of Participants With Treatment Emergent Treatment Related AEs and SAEs
时间窗: From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)
A treatment-related AE was any untoward medical occurrence attributed to the administered medicinal product in a participant who received study drug. Treatment emergent AEs included both SAEs and all non-SAEs. A treatment-related SAE was a treatment-related AE and was defined as any untoward medical occurrence at any dose that: resulted in death; was life-threatening (immediate risk of death); requires inpatient hospitalization or prolongation of hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); constituted a congenital anomaly/birth defect. Causality was assessed by the investigator.
Number of All-Cause Deaths
时间窗: From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)
Number of deaths due to any cause was analyzed as time from enrollment or first treatment of tafamidis.
次要结局
- Number of Participants With Modified Polyneuropathy Disability (mPND) Scores at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Coutinho Disease Stages at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Karnofsky Performance Index at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Heart Failure at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With New York Heart Association (NYHA) Classifications at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants Diagnosed With ATTR at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Prior Misdiagnosis at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With ATTR Genotypes at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Past or Current Clinical Trial Participation at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Past or Current Tafamidis Compassionate Use/Early Access or Other Non-commercial Program at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Known Family History of Symptomatic ATTR at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Affected Generations at Baseline(At the start of data collection at Baseline (Day 1))
- Derived Neuropathy Impairment Score-Lower Limb (NIS-LL) at Baseline(At the start of data collection at Baseline (Day 1))
- Body Mass Index (BMI) at Baseline(At the start of data collection at Baseline (Day 1))
- Modified Body Mass Index (mBMI) at Baseline(At the start of data collection at Baseline (Day 1))
- Sitting Systolic and Diastolic Blood Pressures (SBP and DBP) at Baseline(At the start of data collection at Baseline (Day 1))
- Left Ventricular (LV) Septum Thickness at Baseline(At the start of data collection at Baseline (Day 1))
- Left Ventricular (LV) Ejection Fraction at Baseline(At the start of data collection at Baseline (Day 1))
- Euro Quality of Life-5 Dimensions-3 Level (EQ-5D-3L): Visual Analog Scale (VAS) Overall Health Score at Baseline(At the start of data collection at Baseline (Day 1))
- EQ-5D-3L: VAS Derived Index at Baseline(At the start of data collection at Baseline (Day 1))
- Norfolk Total Quality of Life (QoL) Score at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Abnormal Electrocardiogram (ECG) at Baseline(At the start of data collection at Baseline (Day 1))
- Number of Participants With Atrial Fibrillation/Flutter, Pacemaker Implanted, and Implantable Cardioverter/Defibrillator (ICD) at Baseline(At the start of data collection at Baseline (Day 1))