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

Sponsor
Ablynx (Industry)
Overall Status
Completed
CT.gov ID
NCT03172208
Collaborator
(none)
60
Enrollment
1
Location
12
Arms
4.5
Actual Duration (Months)
13.4
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Biological: Caplacizumab Dose 1 iv (single-dose)
  • Biological: Caplacizumab Dose 2 iv (single-dose)
  • Biological: Caplacizumab Dose 2 sc (single-dose)
  • Biological: Caplacizumab Dose 2 sc (multiple-dose)
  • Other: Placebo iv (single-dose)
  • Other: Placebo sc (single-dose)
  • Other: Placebo sc (multiple-dose)
 Phase 1

Study Design

Study Type:
Interventional
Actual Enrollment :
60 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose:
Other
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.
Actual Study Start Date :
Jun 5, 2017
Actual Primary Completion Date :
Oct 19, 2017
Actual Study Completion Date :
Oct 19, 2017

Arms and Interventions

Arm Intervention/Treatment
Experimental: Group 1: Japanese - Caplacizumab Dose 1 iv (SD)

Single dose (SD) of Caplacizumab Dose 1 administered intravenously (iv) to Japanese participants

Biological: Caplacizumab Dose 1 iv (single-dose)
Single intravenous (iv) administration of Caplacizumab Dose 1
Placebo Comparator: Group 1: Japanese - Placebo iv (SD)

Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants

Other: Placebo iv (single-dose)
Single intravenous (iv) administration of Placebo
Experimental: Group 2: Japanese - Caplacizumab Dose 2 iv (SD)

Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to Japanese participants

Biological: Caplacizumab Dose 2 iv (single-dose)
Single intravenous (iv) administration of Caplacizumab Dose 2
Experimental: Group 2: Japanese - Placebo iv (SD)

Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants

Other: Placebo iv (single-dose)
Single intravenous (iv) administration of Placebo
Experimental: Group 2: White - Caplacizumab Dose 2 iv (SD)

Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to White participants

Biological: Caplacizumab Dose 2 iv (single-dose)
Single intravenous (iv) administration of Caplacizumab Dose 2
Placebo Comparator: Group 2: White - Placebo iv (SD)

Single dose (SD) of Placebo administered intravenously (iv) to White participants

Other: Placebo iv (single-dose)
Single intravenous (iv) administration of Placebo
Experimental: Group 3: Japanese - Caplacizumab Dose 2 sc (SD)

Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants

Biological: Caplacizumab Dose 2 sc (single-dose)
Single subcutaneous (sc) administration of Caplacizumab Dose 2
Placebo Comparator: Group 3: Japanese - Placebo sc (SD)

Single dose (SD) of Placebo administered subcutaneously (sc) to Japanese participants

Other: Placebo sc (single-dose)
Single subcutaneous (sc) administration of Placebo
Experimental: Group 3: White - Caplacizumab Dose 2 sc (SD)

Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to White participants

Biological: Caplacizumab Dose 2 sc (single-dose)
Single subcutaneous (sc) administration of Caplacizumab Dose 2
Placebo Comparator: Group 3: White - Placebo sc (SD)

Single dose (SD) Placebo administered subcutaneously (sc) to White participants

Other: Placebo sc (single-dose)
Single subcutaneous (sc) administration of Placebo
Experimental: Group 4: Japanese - Caplacizumab Dose 2 sc (MD)

Multiple doses (MD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants

Biological: Caplacizumab Dose 2 sc (multiple-dose)
Once daily subcutaneous (sc) administration of Caplacizumab Dose 2 during 7 consecutive days
Placebo Comparator: Group 4: Japanese - Placebo sc (MD)

Multiple doses (MD) of Placebo administered subcutaneously (sc) to Japanese participants

Other: Placebo sc (multiple-dose)
Once daily subcutaneous (sc) administration of Placebo during 7 consecutive days

Outcome Measures

Primary Outcome Measures

  1. 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

  1. Pharmacokinetics: concentration of caplacizumab in plasma [From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)]

  2. Pharmacokinetics: concentration of caplacizumab in urine [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 [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 [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 [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 [From screening until Day 28 (single-dose part) or Day 34 (multiple dose part)]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
Yes
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Investigator Site Glendale California United States 91206

Sponsors and Collaborators

  • Ablynx

Investigators

  • Study Director: Ablynx Clinical Department, Ablynx

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Ablynx
ClinicalTrials.gov Identifier:
NCT03172208
Other Study ID Numbers:
  • ALX0681-C103
First Posted:
Jun 1, 2017
Last Update Posted:
Nov 14, 2017
Last Verified:
Nov 1, 2017
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No

Study Results

No Results Posted as of Nov 14, 2017