The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
32
Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion
Calculated volume to match active comparator
Clinical Site
Le Kremlin-Bicêtre, France
Clinical Site
Porto, Portugal
Clinical Site
Umeå, Sweden
Clinical Site
London, United Kingdom
The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
Time frame: Up to 28 Days
Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Time frame: Up to 70 days
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)
Time frame: up to 70 days
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