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Completed

NCT Number: NCT03172208

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

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.

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Key information

Conditions

Healthy Volunteers

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Investigator Site

Glendale, California, 91206, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Caplacizumab Dose 1 iv (single-dose)

Biological

Single intravenous (iv) administration of Caplacizumab Dose 1

Caplacizumab Dose 2 iv (single-dose)

Biological

Single intravenous (iv) administration of Caplacizumab Dose 2

Caplacizumab Dose 2 sc (single-dose)

Biological

Single subcutaneous (sc) administration of Caplacizumab Dose 2

Caplacizumab Dose 2 sc (multiple-dose)

Biological

Once daily subcutaneous (sc) administration of Caplacizumab Dose 2 during 7 consecutive days

Placebo iv (single-dose)

Other

Single intravenous (iv) administration of Placebo

Placebo sc (single-dose)

Other

Single subcutaneous (sc) administration of Placebo

Placebo sc (multiple-dose)

Other

Once daily subcutaneous (sc) administration of Placebo during 7 consecutive days

Primary outcomes

  1. 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

  1. Pharmacokinetics: concentration of caplacizumab in plasma

    Time frame: From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)

  2. Pharmacokinetics: concentration of caplacizumab in urine

    Time frame: From Day 1 to Day 4 (single dose part) or to Day 8 (multiple dose part)

  3. 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)

  4. 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)

  5. 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)

  6. 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)

Sponsors and collaborators

Lead sponsor

Ablynx, a Sanofi company

Industry

Registry information

Official study 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.

Important dates

Study start
2017
Primary completion
2017
Study completion
2017
First posted
Jun 1, 2017
Registry last updated
Nov 14, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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