Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Conditions
Brief summary
Safety parameters to be assessed: treatment- emergent serious adverse events (SAEs) and adverse events (AEs), AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal electrocardiogram (ECG) parameters of clinical relevance, and changes in clinical safety laboratory parameters of clinical relevance
Detailed description
All-cause mortality and CV mortality, Change from Baseline in distance walked during the 6MWT (6MWD), Change from Baseline in KCCQ Overall Summary Score (KCCQ-OS), CV-related hospitalization, Change from baseline in TTR level (an in vivo measure of TTR stabilization), TTR stabilization measured in established ex vivo assays (FPE and D39Western blot) in the PK-PD substudy
Interventions
Sponsors
Eidos Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety parameters to be assessed: treatment- emergent serious adverse events (SAEs) and adverse events (AEs), AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal electrocardiogram (ECG) parameters of clinical relevance, and changes in clinical safety laboratory parameters of clinical relevance | — |
Secondary
| Measure | Time frame |
|---|---|
| All-cause mortality and CV mortality, Change from Baseline in distance walked during the 6MWT (6MWD), Change from Baseline in KCCQ Overall Summary Score (KCCQ-OS), CV-related hospitalization, Change from baseline in TTR level (an in vivo measure of TTR stabilization), TTR stabilization measured in established ex vivo assays (FPE and D39Western blot) in the PK-PD substudy | — |
Countries
Belgium, Czechia, Denmark, Greece, Ireland, Italy, Netherlands, Portugal, Spain
Outcome results
None listed