ClinicalTrials.gov record NCT04561518
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This prospective observational study follows people diagnosed with transthyretin (TTR)-mediated amyloidosis and pre-symptomatic carriers of known disease-causing TTR variants. It collects data from routine clinical care over years to describe disease features, natural history, and real-world management. The study also records safety and effectiveness information for patisiran and vutrisiran when used in standard practice.
What it is studying. The study aims to describe epidemiological and clinical characteristics and the natural history of ATTR amyloidosis, to document real-world clinical management, to characterize the safety and effectiveness of patisiran and vutrisiran as used in routine care, and to describe how disease emerges or progresses in pre-symptomatic carriers of known disease-causing TTR variants.
What the researchers hope to learn. Describe epidemiology and clinical characteristics of ATTR amyloidosis; map its natural history; document real-world management practices; characterize safety and effectiveness of patisiran and vutrisiran in routine care; describe disease emergence and progression in pre-symptomatic carriers.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is an observational study: participants continue their usual medical care while the study team collects clinical information. Data collection may include reports of adverse events and selected clinical events, doctor‑assessed neuropathy and disability scores, cardiomyopathy assessments and exercise testing, and patient questionnaires about quality of life and disability. Follow-up and data collection are planned long term (the registry lists outcome timeframes up to about 10–11 years). The registry lists no study-specific invasive procedures; the study does not assign experimental treatments.
How the study is structured. A prospective observational cohort (registry) study. Participants continue their usual clinical care; the study collects medical data, healthcare events, clinician assessments, and patient questionnaires over time (registry follow-up reported up to about 10–11 years). No study treatments are assigned.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Brazil, Bulgaria, Denmark, France, Germany, Israel, Italy, Netherlands, Portugal, Spain, Taiwan, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on ATTRv amyloidosis
This trial is also listed on this site under wild-type ATTR amyloidosis.
Important dates
- Study start
- Nov 23, 2020
- Primary completion
- Dec 31, 2033 (estimated)
- Study completion
- Dec 31, 2033 (estimated)
- Target enrollment
- 1500 participants (estimated)
- Last updated on registry
- Sep 8, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Alnylam Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Alnylam Clinical Trial Information Line 📞 1-877-ALNYLAM ✉ clinicaltrials@alnylam.com
- Alnylam Clinical Trial Information Line 📞 1-877-256-9526 ✉ clinicaltrials@alnylam.com
Participating sites (40)
- Clinical Trial SiteRecruiting
La Jolla, California, 92037 · United States
- Clinical Trial SiteRecruiting
Los Angeles, California, 90095 · United States
- Clinical Trial SiteRecruiting
Jacksonville, Florida, 32224 · United States
- Clinical Trial SiteRecruiting
Iowa City, Iowa, 52242 · United States
- Clinical Trial SiteRecruiting
Kansas City, Kansas, 66160 · United States
- Clinical Trial SiteRecruiting
Baltimore, Maryland, 21224 · United States
- Clinical Trial SiteCompleted
Boston, Massachusetts, 02127 · United States
- Clinical Trial SiteRecruiting
New York, New York, 10034 · United States
- Clinical Trial SiteRecruiting
Durham, North Carolina, 27710 · United States
- Clinical Trial SiteRecruiting
Columbus, Ohio, 43210 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- natural history
- A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
- cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- clinical trial
- A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
- investigational agent
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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