Long-term global follow-up of adults with ATTR-CM treated with vutrisiran or other approved therapies
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy in a Real-World Setting
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 |
|---|---|
| Enrollment | 2000 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Alnylam Pharmaceuticals Industry-sponsored |
| Locations | 4 sites (Naples, Florida and 3 other locations) |
| Trial ID | NCT07358078 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Naples, Florida and 3 other locations
- Clinical Trial Site — Naples, Florida, United States (Recruiting)
- Clinical Trial Site — Boston, Massachusetts, United States (Recruiting)
- Clinical Trial Site — New Brunswick, New Jersey, United States (Recruiting)
- Clinical Trial Site — Memphis, Tennessee, United States (Recruiting)
Study contacts
- Study coordinator: Clinical Trial Information Line
- Email: clinicaltrials@alnylam.com
- Phone: 1-877-ALNYLAM
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.