Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Conditions
Keywords
ATTR, Cardiomyopathy, Amylodosis, TTR, Transthyretin, TTR-mediated amyloidosis, Amyloidosis, Hereditary, Transthyretin-Related, RNAi therapeutic, Transthyretin amyloid cardiomyopathy, TTR cardiomyopathy, ATTR-CM, Wild-type TTR, V122I, TTR amyloidosis, Amyloidosis, Wild Type, hATTR amyloidosis with cardiomyopathy, ATTR amyloidosis with cardiomyopathy, wtATTR amyloidosis with cardiomyopathy
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.
Interventions
DRUGVutrisiran
Vutrisiran administered SC q3M
Sponsors
Alnylam Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
Countries
Argentina, Austria, Belgium, Brazil, Czechia, Denmark, France, Italy, Netherlands, Norway, Portugal, South Korea, Spain, Sweden, Taiwan, United Kingdom, United States
Contacts
STUDY_DIRECTORMedical Director
Alnylam Pharmaceuticals
Outcome results
None listed