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

November 9, 2017 updated by: Ablynx

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.

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
60

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Glendale, California, United States, 91206
        • Investigator Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
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)
From signing of informed consent form until Day 28 (single-dose part) or Day 34 (multiple dose part)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Pharmacokinetics: concentration of caplacizumab in plasma
Time Frame: From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
Pharmacokinetics: concentration of caplacizumab in urine
Time Frame: From Day 1 to Day 4 (single dose part) or to Day 8 (multiple dose part)
From Day 1 to Day 4 (single dose part) or to Day 8 (multiple dose part)
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)
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
Time Frame: From screening to Day 6 (single dose part) or to Day 12 (multiple dose part)
From screening to Day 6 (single dose part) or to Day 12 (multiple dose part)
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)
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
Time Frame: From screening until Day 28 (single-dose part) or Day 34 (multiple dose part)
From screening until Day 28 (single-dose part) or Day 34 (multiple dose part)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ablynx

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 5, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 19, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 19, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 29, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 31, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 1, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 14, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 9, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2017

More Information

Terms related to this study

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALX0681-C103

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Healthy Volunteers

Clinical Trials on Caplacizumab Dose 1 iv (single-dose)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings