ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

study id #: NCT04561518

condition: Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis

status: Recruiting

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 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) mutation

results: https://clinicaltrials.gov/ct2/show/results/NCT04561518

last updated: March 10, 2024

start date: November 23, 2020

estimated completion: September 2030

last updated: February 13, 2024

size / enrollment: 1500

primary outcomes:

• Incidence of Adverse Events
  
• From time of enrollment for up to 10 years
  Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) Amyloidosis (ATTRv Amyloidosis)
  Selected events of interest are defined as hepatic events, cardiovascular events, renal events, ocular events and infusion-related reactions in patients diagnosed with ATTRv amyloidosis.
• From 1 year prior to enrollment for up to 10 years
  Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score
  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.
• Up to 11 years
  HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score
  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.
• Up to 11 years
  HCP-Assessed Neuropathy Impairment Score of the Lower Limb (NIS-LL) Score
  The NIS-LL assesses muscle weakness, reflexes and sensation, scored separately for the left and right limbs. Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) are scored on 0 to 4 scale (0=normal, 4=paralysis). Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) are scored 0=normal, 1=decreased, or 2=absent. Total possible NIS-LL score ranges 0-88 with higher score=greater impairment.
• Up to 11 years
  HCP-Assessed Cardiomyopathy
  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.
• Up to 11 years
  Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score
  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).
• Up to 11 years
  Kansas City Cardiomyopathy Questionnaire (KCCQ)
  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).
• Up to 11 years
  Rasch-built Overall Disability Scale (R-ODS)
  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.
• Up to 11 years
  

inclusion criteria:

exclusion criteria: Criteria:

Current enrollment in a clinical trial for any investigational agent

sponsor: Alnylam Pharmaceuticals

contacts: Alnylam Clinical Trial Information Line, 1-877-ALNYLAM, [email protected]

investigators: Medical Director,Alnylam Pharmaceuticals

trial center locations:

• United States, California
  Clinical Trial Site
  
  
• United States, California
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• United States, Florida
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• United States, Iowa
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• United States, Kansas
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• United States, Maryland
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• United States, Massachusetts
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• United States, New York
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• United States, North Carolina
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• United States, Ohio
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• United States, Pennsylvania
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• United States, Texas
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• United States, Texas
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• Brazil
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• Denmark
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• Denmark
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• France
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• France
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• France
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• France
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• Germany
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• Israel
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• Israel
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• Italy
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• Italy
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• Italy
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• Italy
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• Netherlands
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• Portugal
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• Portugal
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• Spain
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• Spain
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• Spain
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• Taiwan
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• Taiwan
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