Caplacizumab Single and Multiple Dose Study in Healthy Japanese and White Subjects.

Phase 1

Completed

• Conditions: Healthy Volunteers

• 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

Study Timeline

• Study First Submitted: 2017-05-29
• Study First Submitted QC: 2017-05-31
• Study First Posted: 2017-06-01
• Study Start: 2017-06-05
• Primary Completion: 2017-10-19
• Study Completion: 2017-10-19
• Status Verified: 2017-11
• Last Update Submitted: 2017-11-09
• Last Update Posted: 2017-11-14

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

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