Caplacizumab Single and Multiple Dose Study in Healthy Japanese and White Subjects.
Phase 1
Completed
- Conditions
- Healthy Volunteers
- Interventions
- Biological: Caplacizumab Dose 2 iv (single-dose)Other: Placebo sc (single-dose)Biological: Caplacizumab Dose 2 sc (single-dose)Other: Placebo iv (single-dose)Biological: Caplacizumab Dose 2 sc (multiple-dose)Biological: Caplacizumab Dose 1 iv (single-dose)Other: Placebo sc (multiple-dose)
- Registration Number
- NCT03172208
- Lead Sponsor
- Ablynx, a Sanofi company
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 60
Inclusion Criteria
- BMI between ≥18 kg/m² and <30 kg/m² at time of screening
- Body weight between ≥45 kg and <100 kg
- Baseline vWF:Ag between ≥60% and <170% (0.6-1.7 IU/mL)
Exclusion Criteria
- History of and/or any sign or symptom indicating current abnormal hemostasis or blood dyscrasia, including but not limited to thrombocytopenia, thrombocytopathy, thromboasthenia, hemophilia, von Willebrand's disease, vascular purpura, bleeding gums and/or frequent nose bleeding, easy/spontaneous bruises, meno- or metrorrhagia, history of gastrointestinal bleeding or excessive bleeding after minor injury such as shaving.
- Family history of congenital vascular malformation (e.g., Marfan's Syndrome) and/or bleeding disorder (e.g., hemophilia, von Willebrand's disease, Christmas disease)
- Any surgical intervention (including tooth extraction) or trauma within the 4 weeks preceding screening for the study or any planned surgical intervention during the participation in the study
- Treatment with vitamin K, direct oral anticoagulant (DOAC), warfarin, high dose heparin within 2 weeks before screening
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group 2: White - Caplacizumab Dose 2 iv (SD) Caplacizumab Dose 2 iv (single-dose) Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to White participants Group 3: Japanese - Placebo sc (SD) Placebo sc (single-dose) Single dose (SD) of Placebo administered subcutaneously (sc) to Japanese participants Group 3: White - Caplacizumab Dose 2 sc (SD) Caplacizumab Dose 2 sc (single-dose) Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to White participants Group 2: Japanese - Caplacizumab Dose 2 iv (SD) Caplacizumab Dose 2 iv (single-dose) Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to Japanese participants Group 2: Japanese - Placebo iv (SD) Placebo iv (single-dose) Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants Group 2: White - Placebo iv (SD) Placebo iv (single-dose) Single dose (SD) of Placebo administered intravenously (iv) to White participants Group 3: White - Placebo sc (SD) Placebo sc (single-dose) Single dose (SD) Placebo administered subcutaneously (sc) to White participants Group 1: Japanese - Placebo iv (SD) Placebo iv (single-dose) Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants Group 4: Japanese - Caplacizumab Dose 2 sc (MD) Caplacizumab Dose 2 sc (multiple-dose) Multiple doses (MD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants Group 1: Japanese - Caplacizumab Dose 1 iv (SD) Caplacizumab Dose 1 iv (single-dose) Single dose (SD) of Caplacizumab Dose 1 administered intravenously (iv) to Japanese participants Group 3: Japanese - Caplacizumab Dose 2 sc (SD) Caplacizumab Dose 2 sc (single-dose) Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants Group 4: Japanese - Placebo sc (MD) Placebo sc (multiple-dose) Multiple doses (MD) of Placebo administered subcutaneously (sc) to Japanese participants
- Primary Outcome Measures
Name Time Method The number of treatment-emergent adverse events (Safety and Tolerability) From signing of informed consent form until Day 28 (single-dose part) or Day 34 (multiple dose part)
- Secondary Outcome Measures
Name Time Method Pharmacokinetics: concentration of caplacizumab in plasma 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 From screening to Day 6 (single dose part) or to Day 12 (multiple dose part) Pharmacodynamics as measured by Ristocetin cofactor activity in plasma From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part) Pharmacokinetics: concentration of caplacizumab in urine From Day 1 to Day 4 (single dose part) or to Day 8 (multiple dose part) Pharmacodynamics as measured by Factor VIII clotting activity in plasma 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 From screening until Day 28 (single-dose part) or Day 34 (multiple dose part)
Trial Locations
- Locations (1)
Investigator Site
🇺🇸Glendale, California, United States