An observational study in Transthyretin-Mediated Amyloidosis and ATTR Amyloidosis, sponsored by Alnylam Pharmaceuticals. Recruiting at 40 sites in 12 countries. Per ClinicalTrials.gov, last updated 2026-10-13.
Sponsored by Alnylam Pharmaceuticals · Observational
The purpose of this study is to:
290 studies on the registry are indexed under Amyloidosis; 92 are open to participants now.
This study's planned enrollment of 1,500 is above the median of 190 across 83 observational studies indexed under Amyloidosis.
Browse Amyloidosis studies →Alnylam Pharmaceuticals is the lead sponsor of 84 studies on the registry; 19 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 15 (83%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, will be eligible for the study and will follow routine clinical care.
Pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study and will follow routine clinical care.
Incidence of Adverse Events
Time frame: From 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)
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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 11 years
HCP-Assessed Neuropathy Impairment Score (NIS)
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.
Time frame: 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.
Time frame: Up to 11 years
HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance
Time frame: 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).
Time frame: 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).
Time frame: 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.
Time frame: Up to 11 years
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Plan to share: No
No publications or documents are linked to this record.
Started Nov 2020 (actual). Primary completion is estimated for Dec 2033, 157 months after the start.
Of 52 observational studies under Amyloidosis, half reached primary completion within 97 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 183. This study's planned 157 months is above that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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