A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
- Registration Number
- NCT06679946
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 800
- Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003
- Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
- Future or current participation in another investigational device or drug study, scheduled to occur during this study
- Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Vutrisiran 25 mg Vutrisiran Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).
- Primary Outcome Measures
Name Time Method Frequency of Adverse Events (AEs) Up to 36 Months An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
- Secondary Outcome Measures
Name Time Method Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Baseline up to 36 Months The KCCQ is a 23-item self-administered questionnaire quantifying 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 are transformed to a range of 0-100, in which higher scores reflect better health status.
Change from Baseline in New York Heart Association (NYHA) Class Baseline up to 36 Months NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea).
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) Baseline up to 36 Months NT-proBNP will be assessed through measurement of serum levels.
Change From Baseline in the Cardiac Biomarker: Troponin I Baseline up to 36 Months Troponin I will be assessed through measurement of serum levels.
All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) Up to 36 Months All deaths, hospitalizations and urgent HF visits will be recorded throughout the study as part of SAE and AE monitoring
Change from baseline in serum TTR level Baseline, Month 6 and Month 12
Trial Locations
- Locations (1)
Clinical Trial Site
🇬🇧Manchester, United Kingdom