Primary objective: To assess the safety and tolerability of single ascending intravenous (i.v.) doses, a single subcutaneous (s.c.) dose of caplacizumab (Part I), and multiple s.c. doses of caplacizumab (Part II) in Japanese subjects. Secondary objectives: * To compare the pharmacokinetic (PK) and pharmacodynamic (PD) profiles (total vWF:Ag concentration levels \[vWF:Ag\], coagulation factor VIII \[FVIII:C\], and ristocetin cofactor activity \[RICO\]) after single i.v. or s.c. administration of caplacizumab in Japanese and White subjects. * To evaluate the immunogenicity of caplacizumab (anti-drug antibodies \[ADA\]) in Japanese subjects.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
TRIPLE
Enrollment
60
Single intravenous (iv) administration of Caplacizumab Dose 1
Single intravenous (iv) administration of Caplacizumab Dose 2
Single subcutaneous (sc) administration of Caplacizumab Dose 2
Once daily subcutaneous (sc) administration of Caplacizumab Dose 2 during 7 consecutive days
Single intravenous (iv) administration of Placebo
Single subcutaneous (sc) administration of Placebo
Once daily subcutaneous (sc) administration of Placebo during 7 consecutive days
Investigator Site
Glendale, California, United States
The number of treatment-emergent adverse events (Safety and Tolerability)
Time frame: From signing of informed consent form until Day 28 (single-dose part) or Day 34 (multiple dose part)
Pharmacokinetics: concentration of caplacizumab in plasma
Time frame: From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
Pharmacokinetics: concentration of caplacizumab in urine
Time frame: From Day 1 to Day 4 (single dose part) or to Day 8 (multiple dose part)
Pharmacodynamics as measured by Ristocetin cofactor activity in plasma
Time frame: From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
Pharmacodynamics as measured by von Willebrand factor antigen in plasma
Time frame: From screening to Day 6 (single dose part) or to Day 12 (multiple dose part)
Pharmacodynamics as measured by Factor VIII clotting activity in plasma
Time frame: From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
Immunogenicity as measured by the concentration of anti-caplacizumab antibodies in serum
Time frame: From screening until Day 28 (single-dose part) or Day 34 (multiple dose part)
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