Study BT200-01 is a first in human (FIH) study in male and female normal human volunteers (NHVs) that uses an Integrated Protocol Design. This Phase 1 study will comprise 4 sub-parts: Part A, a single ascending dose (SAD) study; Part B, a multiple ascending dose (MAD) study; Part C, a desmopressin challenge study to explore (i) whether desmopressin could be used as an antidote, and/or (ii) whether desmopressin stimulated vonWillebrand Factor (VWF) release is overcome with increasing BT200 doses; and Part D, a relative bioavailability (BA) study. The primary objective of this study is to assess the safety and tolerability profile of BT200 in NHVs.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
112
BT200 is a PEGylated synthetic RNA oligonucleotide
Sterile solution for injection
Sterile saline for injection
Medical University of Vienna
Vienna, Austria
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
Any AE or bleeding event related to study treatment
Time frame: Baseline through 8 weeks after dosing up to 56 days
Measured Area Under the Curve (AUC)
Measured Area Under the Curve at timepoints pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d post dose
Time frame: Baseline through 8 weeks after dosing up to 56 days
Maximum Plasma Concentration (Cmax)
Maximum plasma Concentration measured at pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d
Time frame: Baseline through 8 weeks after dosing up to 56 days
Time to Maximum Plasma Concentration (Tmax)
Time to maximum plasma concentration measured at pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d
Time frame: Baseline through 8 weeks after dosing up to 56 days
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