The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of ALN-AT3SC in healthy volunteers and Hemophilia A or B patients.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
51
Ascending doses of ALN-AT3SC by subcutaneous (sc) injection
Calculated volume to match active comparator
Clinical Trial Site
Pittsburgh, Pennsylvania, United States
Clinical Trial Site
Plovdiv, Bulgaria
Clinical Trial Site
Sofia, Bulgaria
The safety of ALN-AT3SC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicities (DLTs), and AEs leading to study drug discontinuation.
Time frame: Part A (SAD phase): through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacokinetics (PK) of ALN-AT3SC as characterized by plasma PK profiles and urine samples.
Time frame: Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma AT levels.
Time frame: Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma TG.
Time frame: Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
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Clinical Trial Site
Varna, Bulgaria
Clinical Trial Site
Kirov, Russia
Clinical Trial Site
Moscow, Russia
Clinical Trial Site
Saint Petersburg, Russia
Clinical Trial Site
Sankt Gallen, Switzerland
Clinical Trial Site
Zurich, Switzerland
Clinical Trial Site
Glasgow, United Kingdom
...and 4 more locations