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

Healthy Volunteers Clinical Trial

Official title:

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03172208
• Other study ID #: ALX0681-C103
• Status: Completed
• Phase: Phase 1
• First received: May 29, 2017
• Last updated: November 9, 2017
• Start date: June 5, 2017
• Est. completion date: October 19, 2017

Study information

• Verified date: November 2017
• Source: Ablynx
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: October 19, 2017
• Est. primary completion date: October 19, 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Healthy Volunteers

Intervention

Biological:
• Caplacizumab Dose 1 iv (single-dose)
Single intravenous (iv) administration of Caplacizumab Dose 1
• Caplacizumab Dose 2 iv (single-dose)
Single intravenous (iv) administration of Caplacizumab Dose 2
• Caplacizumab Dose 2 sc (single-dose)
Single subcutaneous (sc) administration of Caplacizumab Dose 2
• Caplacizumab Dose 2 sc (multiple-dose)
Once daily subcutaneous (sc) administration of Caplacizumab Dose 2 during 7 consecutive days

Other:
• Placebo iv (single-dose)
Single intravenous (iv) administration of Placebo
• Placebo sc (single-dose)
Single subcutaneous (sc) administration of Placebo
• Placebo sc (multiple-dose)
Once daily subcutaneous (sc) administration of Placebo during 7 consecutive days

Locations

Country	Name	City	State
United States	Investigator Site	Glendale	California

Sponsors (1)

Lead Sponsor	Collaborator
Ablynx

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Pharmacokinetics: concentration of caplacizumab in plasma		From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
Secondary	Pharmacokinetics: concentration of caplacizumab in urine		From Day 1 to Day 4 (single dose part) or to Day 8 (multiple dose part)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)