Clinical Trial
Study Details
Status
RECRUITING
Study ID #
NCT04561518
Study Start
Primary Completion
Study Completion
Enrollment
1500
Study Type
Observational
Primary Outcomes
Measure
Incidence of Adverse Events
Timeframe
From time of enrollment for up to 10 years
Measure
Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) Amyloidosis (ATTRv Amyloidosis)
Description

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.

Timeframe
From 1 year prior to enrollment for up to 10 years
Measure
Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score
Description

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.

Timeframe
Up to 11 years
Measure
HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score
Description

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.

Timeframe
Up to 11 years
Measure
HCP-Assessed Neuropathy Impairment Score (NIS)
Description

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.

Timeframe
Up to 11 years
Measure
HCP-Assessed Cardiomyopathy
Description

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.

Timeframe
Up to 11 years
Measure
HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance
Timeframe
Up to 11 years
Measure
Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score
Description

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).

Timeframe
Up to 11 years
Measure
Kansas City Cardiomyopathy Questionnaire (KCCQ)
Description

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).

Timeframe
Up to 11 years
Measure
Rasch-built Overall Disability Scale (R-ODS)
Description

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.

Timeframe
Up to 11 years
Summary

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

Conditions
Transthyretin-Mediated Amyloidosis
ATTR Amyloidosis
Study Contact
Name
Alnylam Clinical Trial Information Line
Role
Contact
Phone
1-877-ALNYLAM
Email
clinicaltrials@alnylam.com
Name
Alnylam Clinical Trial Information Line
Role
Contact
Phone
1-877-256-9526
Email
clinicaltrials@alnylam.com
Locations
Facility

Clinical Trial Site
La Jolla, CA 92037
United States

Facility

Clinical Trial Site
Los Angeles, CA 90095
United States

Facility

Clinical Trial Site
Jacksonville, FL 32224
United States

Facility

Clinical Trial Site
Iowa City, IA 52242
United States

Facility

Clinical Trial Site
Kansas City, KS 66160
United States

Facility

Clinical Trial Site
Baltimore, MD 21224
United States

Facility

Clinical Trial Site
Boston, MA 02127
United States

Facility

Clinical Trial Site
New York, NY 10034
United States

Facility

Clinical Trial Site
Durham, NC 27710
United States

Facility

Clinical Trial Site
Columbus, OH 43210
United States

Facility

Clinical Trial Site
Philadelphia, PA 19104
United States

Facility

Clinical Trial Site
Austin, TX 78756
United States

Facility

Clinical Trial Site
Houston, TX 77030
United States

Facility

Clinical Trial Site
Salvador
Brazil

Facility

Clinical Trial Site
Arhus
Denmark

Facility

Clinical Trial Site
Copenhagen
Denmark

Facility

Clinical Trial Site
Bordeaux
France

Facility

Clinical Trial Site
Bron
France

Facility

Clinical Trial Site
Le Kremlin-Bicêtre
France

Facility

Clinical Trial Site
Marseille
France

Facility

Clinical Trial Site
Hanover
Germany

Facility

Clinical Trial Site
Jerusalem
Israel

Facility

Clinical Trial Site
Ramat Gan
Israel

Facility

Clinical Trial Site
Milan
Italy

Facility

Clinical Trial Site
Naples
Italy

Facility

Clinical Trial Site
Palermo
Italy

Facility

Clinical Trial Site
Roma
Italy

Facility

Clinical Trial Site
Groningen
Netherlands

Facility

Clinical Trial Site
Lisbon
Portugal

Facility

Clinical Trial Site
Porto
Portugal

Facility

Clinical Trial Site
Barcelona
Spain

Facility

Clinical Trial Site
Madrid
Spain

Facility

Clinical Trial Site
Palma
Spain

Facility

Clinical Trial Site
New Taipei City
Taiwan

Facility

Clinical Trial Site
Taipei
Taiwan

Eligibility 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

Sexes Eligible for Study
ALL
Eligibility Std Ages
Child
Adult
Older Adult
Study Population

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.