A Phase I Single Center, Randomized, Double-blind, Placebo Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Caplacizumab in Japanese and White Healthy Volunteers.

Ablynx, a Sanofi company1 site in 1 country60 target enrollmentJune 5, 2017

ConditionsHealthy Volunteers

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Healthy Volunteers
Sponsor: Ablynx, a Sanofi company
Enrollment: 60
Locations: 1
Primary Endpoint: The number of treatment-emergent adverse events (Safety and Tolerability)
Status: Completed
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

June 5, 2017

End Date

October 19, 2017

Last Updated

8 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Ablynx, a Sanofi company

Responsible Party

Sponsor

Eligibility Criteria

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

Outcomes

Primary Outcomes

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)

Secondary Outcomes

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))