Observational Study of Transthyretin-Mediated Amyloidosis (ATTR Amyloidosis)
This is an observational study called ConTTRibute, looking at people with Transthyretin-Mediated Amyloidosis (ATTR amyloidosis). Researchers want to understand more about this condition, how it progresses, and how it's managed in real life. They will also observe the safety and effectiveness of two specific medications, patisiran and vutrisiran, when used in regular patient care. The study aims to enroll about 1500 participants, including those already diagnosed with ATTR amyloidosis or those who carry a gene variant that causes the disease but don't yet have symptoms. The study will track health events, including side effects, and changes in nerve damage over time. The current status of the study is unclear.
- Study design
- This is an observational study, meaning participants will be followed over time without receiving a specific study intervention. It aims to enroll about 1500 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 10 years for adverse events and other health-related events, and up to 11 years for nerve damage scores.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
At a glance
Conditions
Where it's being run
40 sites across 22 statesStudy leadership
- Medical Director · STUDY_DIRECTOR · Alnylam Pharmaceuticals
Who to contact
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Do you actually qualify for this trial?
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Inclusion
Exclusion
What this trial measures
- Incidence of Adverse EventsFrom time of enrollment for up to 10 years
- Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) or Wild-type Transthyretin-mediated (wtATTR) Amyloidosis (ATTRv Amyloidosis)From 1 year prior to enrollment for up to 10 years
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 or wtATTR amyloidosis.
- Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability ScoreUp to 11 years
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.
- HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) ScoreUp to 11 years
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.
- HCP-Assessed Neuropathy Impairment Score (NIS)Up to 11 years
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.
- HCP-Assessed CardiomyopathyUp to 11 years
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.
- HCP- Assessed Cardiopulmonary Exercise Testing (CPET) PerformanceUp to 11 years
- Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total ScoreUp to 11 years
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).
- Kansas City Cardiomyopathy Questionnaire (KCCQ)Up to 11 years
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).
- Rasch-built Overall Disability Scale (R-ODS)Up to 11 years
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.