Clinical Trial
Study Details
Outcomes
Selected events of interest are defined as hepatic events, cardiovascular events, renal events, ocular events and infusion-related reactions, hypersensitivity, and other events in patients diagnosed with hATTR amyloidosis.
PND Scores: Stage 0=No symptoms; Stage I=Sensory disturbances but preserved walking capabilities; Stage II=Impaired walking capacity, but ability to walk without a stick or crutches; Stage IIIA=Walking with help of 1 stick or crutch; Stage IIIB=Walking with the help of 2 sticks or crutches; Stage IV=confined to wheel chair or bedridden.
FAP Scores: Stage 0=No symptoms; Stage I=Unimpaired ambulation; mostly mild sensory, motor and autonomic neuropathy in the lower limbs; Stage II=Assistance with ambulation required, mostly moderate impairment progression to the lower limbs, upper limbs, and trunk; Stage III=Wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs.
NIS : 74 items, assess muscle weakness, reflexes and sensation; scored separately for left, right limbs (37 items for each side). Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) scored on scale 0 (normal) to 4 (paralysis), higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) scored 0 = normal, 1= decreased, or 2 = absent. Total possible NIS score range 0-244, higher score=greater impairment.
Cardiomyopathy will be assessed using New York Heart Association (NYHA) Class: I=No symptoms; II=Symptoms with ordinary physical activity; III=Symptoms with less than ordinary physical activity; IV=Symptoms at rest.
Norfolk-QoL-DN: The Norfolk QOL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that assesses 6 domains: physical function, large-fiber neuropathy, activities of daily living, symptoms, small-fiber neuropathy, and autonomic neuropathy. The total score ranges from -4 points (best possible quality of life) to 136 points (worst possible quality of life).
The KCCQ is a 23-item self-administered questionnaire developed to independently measure the patient's perception of health status, which includes heart failure symptoms, impact on physical and social function, and how their heart failure impacts their quality of life within a 2-week recall period. The KCCQ quantifies 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary \[OS\] scores).
The R-ODS is a 24-item self-administered questionnaire for assessment of the disability a patient experiences. It uses a linearly weighted categorical rating scale that specifically captures domains of activity and social participation limitations in patients.
Study Overview
The purpose of this study is to:
* Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients
* Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting
* Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant
Contacts and Locations
Clinical Trial Site
La Jolla, CA 92037
United States
Clinical Trial Site
Los Angeles, CA 90095
United States
Clinical Trial Site
Jacksonville, FL 32224
United States
Clinical Trial Site
Iowa City, IA 52242
United States
Clinical Trial Site
Kansas City, KS 66160
United States
Clinical Trial Site
Baltimore, MD 21224
United States
Clinical Trial Site
Boston, MA 02127
United States
Clinical Trial Site
New York, NY 10034
United States
Clinical Trial Site
Durham, NC 27710
United States
Clinical Trial Site
Columbus, OH 43210
United States
Clinical Trial Site
Philadelphia, PA 19104
United States
Clinical Trial Site
Austin, TX 78756
United States
Clinical Trial Site
Houston, TX 77030
United States
Clinical Trial Site
Salvador
Brazil
Clinical Trial Site
Arhus
Denmark
Clinical Trial Site
Copenhagen
Denmark
Clinical Trial Site
Bordeaux
France
Clinical Trial Site
Bron
France
Clinical Trial Site
Le Kremlin-Bicêtre
France
Clinical Trial Site
Marseille
France
Clinical Trial Site
Hanover
Germany
Clinical Trial Site
Jerusalem
Israel
Clinical Trial Site
Ramat Gan
Israel
Clinical Trial Site
Milan
Italy
Clinical Trial Site
Naples
Italy
Clinical Trial Site
Palermo
Italy
Clinical Trial Site
Roma
Italy
Clinical Trial Site
Groningen
Netherlands
Clinical Trial Site
Lisbon
Portugal
Clinical Trial Site
Porto
Portugal
Clinical Trial Site
Barcelona
Spain
Clinical Trial Site
Madrid
Spain
Clinical Trial Site
Palma
Spain
Clinical Trial Site
New Taipei City
Taiwan
Clinical Trial Site
Taipei
Taiwan
Participation Criteria
Inclusion Criteria:
* Diagnosis of ATTR amyloidosis or documented known disease-causing TTR variant for the cohort of pre-symptomatic carriers
* Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis
Exclusion Criteria:
* Current enrollment in a clinical trial for any investigational agent
Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, and pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study.