Long-term global follow-up of adults with ATTR-CM treated with vutrisiran or other approved therapies
Official title: DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy in a Real-World Setting
Run by Alnylam Pharmaceuticals · NCT07358078 · Data updated 2026-11-12
This project will follow adults with ATTR-CM who start vutrisiran or another approved treatment to see how they do over up to five years in routine clinical care.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 2000 (estimated) |
| Sponsor | Alnylam Pharmaceuticals Industry-sponsored |
| Locations | 27 sites (Tucson, Arizona and 26 other locations) |
| Status | Recruiting |
| Started | January 9, 2026 |
| Main results expected | October 23, 2030 |
| Study ends | October 23, 2030 |
| Registry record updated | October 9, 2026 |
| Trial ID | NCT07358078 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults with a diagnosis of ATTR-CM who are starting treatment with vutrisiran or another approved ATTR-CM therapy and who are not enrolled in an interventional clinical trial are ideal candidates.
✕Probably not a fit
Patients who are not diagnosed with ATTR-CM, who are not initiating an approved ATTR-CM therapy, or who are already enrolled in an interventional clinical trial are unlikely to benefit from participating.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a global, observational, multicenter registry that enrolls adults diagnosed with transthyretin-mediated amyloid cardiomyopathy who are initiating vutrisiran or another approved ATTR-CM therapy. Sites will collect clinical characteristics, treatment patterns, health-related quality of life, and healthcare resource use during routine care. Participants will be followed for up to five years or until withdrawal, loss to follow-up, death, or study end. The dataset will be used to compare long-term effectiveness of vutrisiran versus other approved therapies in a real-world setting.
Why it matters
Potential benefit: If successful, the project could clarify how vutrisiran performs in everyday clinical practice and help doctors choose the best approved treatments for patients with ATTR-CM.
How similar studies have gone: Randomized trials have shown efficacy for RNAi therapies like vutrisiran and other approved ATTR-CM treatments, and some registries exist, but long-term head-to-head real-world comparisons remain limited.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Has a diagnosis of ATTR-CM * Is initiating treatment with vutrisiran or another approved ATTR-CM therapy Exclusion Criteria: * Is enrolled in an interventional clinical trial
Where it's running
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Clinical Trial Site
Tucson, Arizona, United States Recruiting -
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Beverly Hills, California, United States Recruiting -
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La Jolla, California, United States Recruiting -
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Naples, Florida, United States Recruiting -
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Honolulu, Hawaii, United States Recruiting -
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Kansas City, Kansas, United States Recruiting
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Boston, Massachusetts, United States Recruiting -
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St Louis, Missouri, United States Recruiting -
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New Brunswick, New Jersey, United States Recruiting -
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Charlotte, North Carolina, United States Recruiting -
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Philadelphia, Pennsylvania, United States Recruiting -
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Memphis, Tennessee, United States Recruiting -
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Dallas, Texas, United States Recruiting -
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Heidelberg, Germany Recruiting -
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Munich, Germany Recruiting -
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Münster, Germany Recruiting -
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Würzburg, Germany Recruiting -
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Bunkyō City, Japan Recruiting -
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Kumamoto, Japan Recruiting -
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Kurume, Japan Recruiting -
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Matsumoto, Japan Recruiting -
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Sendai, Japan Recruiting -
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Shinjuku, Japan Recruiting -
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Suita, Japan Recruiting -
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Tsukuba, Japan Recruiting -
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Ube, Japan Recruiting -
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Yufu, Japan Recruiting
Study contacts
- Study coordinator: Clinical Trial Information Line
- Email: clinicaltrials@alnylam.com
- Phone: 1-877-ALNYLAM
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Long-term safety of eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplant or severe liver impairment
This study will see if eplontersen remains safe over time for adults with transthyretin amyloidosis, including people who have had a liver transplant or have severe liver problems.
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Nucresiran for transthyretin amyloidosis with cardiomyopathy
This trial will test whether nucresiran can lower deaths and major heart-related events in people with transthyretin amyloidosis that affects the heart.
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Acoramidis for ATTR-CM: Cardiac Imaging Study
This study tests whether acoramidis can improve heart structure and function in people with transthyretin amyloid cardiomyopathy.
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Evaluating NTLA-2001 for treating heart issues in patients with ATTR amyloidosis
This study is testing if a new treatment called NTLA-2001 can improve heart function and health for people with heart issues caused by ATTR amyloidosis.
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Annular ligament biopsy during endoscopic carpal tunnel surgery to screen for transthyretin amyloidosis
This study tests whether a biopsy of the annular ligament during carpal tunnel surgery can reliably indicate transthyretin amyloidosis and match the results of the standard synovial biopsy.
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Eplontersen exposure during pregnancy and breastfeeding: outcomes
This project collects reports to see if exposure to eplontersen during pregnancy or breastfeeding affects mothers, fetuses, or infants with transthyretin amyloidosis.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.