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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03172208
Recruitment Status : Completed
First Posted : June 1, 2017
Last Update Posted : November 14, 2017
Sponsor:
Information provided by (Responsible Party):
Ablynx

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
Healthy Volunteers 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

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Study Type : Interventional  (Clinical Trial)
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 : June 5, 2017
Actual Primary Completion Date : October 19, 2017
Actual Study Completion Date : October 19, 2017

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




Primary Outcome Measures :
  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 Outcome Measures :
  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) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03172208


Locations
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United States, California
Investigator Site
Glendale, California, United States, 91206
Sponsors and Collaborators
Ablynx
Investigators
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Study Director: Ablynx Clinical Department Ablynx
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Responsible Party: Ablynx
ClinicalTrials.gov Identifier: NCT03172208    
Other Study ID Numbers: ALX0681-C103
First Posted: June 1, 2017    Key Record Dates
Last Update Posted: November 14, 2017
Last Verified: November 2017

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No