Healthy VolunteersPhase 1NCT03172208
Caplacizumab Single and Multiple Dose Study in Healthy Japanese and White Subjects.
A Phase 1 study testing Caplacizumab Dose 1 iv (single-dose), Caplacizumab Dose 2 iv (single-dose) and Caplacizumab Dose 2 sc (single-dose) in people with Healthy Volunteers, run by Ablynx, a Sanofi company at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteers
- What is being tested?
- Caplacizumab Dose 1 iv (single-dose), Caplacizumab Dose 2 iv (single-dose) and Caplacizumab Dose 2 sc (single-dose)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jun 2017to 19 Oct 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
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.
- Conditions
- Healthy Volunteers
- Being tested
- Caplacizumab Dose 1 iv (single-dose), Caplacizumab Dose 2 iv (single-dose), Caplacizumab Dose 2 sc (single-dose), Caplacizumab Dose 2 sc (multiple-dose), Placebo iv (single-dose)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| Group 1: Japanese - Caplacizumab Dose 1 iv (SD) | Experimental | Single dose (SD) of Caplacizumab Dose 1 administered intravenously (iv) to Japanese participants Receives: Biological: Caplacizumab Dose 1 iv (single-dose) |
| Group 1: Japanese - Placebo iv (SD) | Placebo comparator | Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants Receives: Other: Placebo iv (single-dose) |
| Group 2: Japanese - Caplacizumab Dose 2 iv (SD) | Experimental | Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to Japanese participants Receives: Biological: Caplacizumab Dose 2 iv (single-dose) |
| Group 2: Japanese - Placebo iv (SD) | Experimental | Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants Receives: Other: Placebo iv (single-dose) |
| Group 2: White - Caplacizumab Dose 2 iv (SD) | Experimental | Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to White participants Receives: Biological: Caplacizumab Dose 2 iv (single-dose) |
| Group 2: White - Placebo iv (SD) | Placebo comparator | Single dose (SD) of Placebo administered intravenously (iv) to White participants Receives: Other: Placebo iv (single-dose) |
| Group 3: Japanese - Caplacizumab Dose 2 sc (SD) | Experimental | Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants Receives: Biological: Caplacizumab Dose 2 sc (single-dose) |
| Group 3: Japanese - Placebo sc (SD) | Placebo comparator | Single dose (SD) of Placebo administered subcutaneously (sc) to Japanese participants Receives: Other: Placebo sc (single-dose) |
| Group 3: White - Caplacizumab Dose 2 sc (SD) | Experimental | Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to White participants Receives: Biological: Caplacizumab Dose 2 sc (single-dose) |
| Group 3: White - Placebo sc (SD) | Placebo comparator | Single dose (SD) Placebo administered subcutaneously (sc) to White participants Receives: Other: Placebo sc (single-dose) |
| Group 4: Japanese - Caplacizumab Dose 2 sc (MD) | Experimental | Multiple doses (MD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants Receives: Biological: Caplacizumab Dose 2 sc (multiple-dose) |
| Group 4: Japanese - Placebo sc (MD) | Placebo comparator | Multiple doses (MD) of Placebo administered subcutaneously (sc) to Japanese participants Receives: Other: Placebo sc (multiple-dose) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of treatment-emergent adverse events (Safety and Tolerability)
Measured: From signing of informed consent form until Day 28 (single-dose part) or Day 34 (multiple dose part)
Secondary outcomes (6)
Pharmacokinetics: concentration of caplacizumab in plasma
Measured: From Day -1 to Day 6 (single dose part) or to Day 12 (multiple dose part)
Pharmacokinetics: concentration of caplacizumab in urine
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: From screening until Day 28 (single-dose part) or Day 34 (multiple dose part)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2017
- Study start5 Jun 2017
- Primary completion19 Oct 2017
- Study completion19 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy VolunteersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ablynx, a Sanofi company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ablynx Clinical Department, Study director, Ablynx, a Sanofi company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03172208
- Sponsor's protocol ID
- ALX0681-C103
- Status last verified
- Nov 2017
- First submitted
- 29 May 2017
- Last updated
- 14 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT03172208
- Acronym / sponsor ID
- ALX0681-C103
- Conditions
- Healthy Volunteers
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Ablynx, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 5 Jun 2017 (actual)
- Primary completion
- 19 Oct 2017 (actual)
- Study completion
- 19 Oct 2017 (actual)
- First posted
- 1 Jun 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 12
| Arm | Type | Receives |
|---|---|---|
| Group 1: Japanese - Caplacizumab Dose 1 iv (SD) | Experimental | Biological: Caplacizumab Dose 1 iv (single-dose)Single dose (SD) of Caplacizumab Dose 1 administered intravenously (iv) to Japanese participants |
| Group 1: Japanese - Placebo iv (SD) | Placebo comparator | Other: Placebo iv (single-dose)Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants |
| Group 2: Japanese - Caplacizumab Dose 2 iv (SD) | Experimental | Biological: Caplacizumab Dose 2 iv (single-dose)Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to Japanese participants |
| Group 2: Japanese - Placebo iv (SD) | Experimental | Other: Placebo iv (single-dose)Single dose (SD) of Placebo administered intravenously (iv) to Japanese participants |
| Group 2: White - Caplacizumab Dose 2 iv (SD) | Experimental | Biological: Caplacizumab Dose 2 iv (single-dose)Single dose (SD) of Caplacizumab Dose 2 administered intravenously (iv) to White participants |
| Group 2: White - Placebo iv (SD) | Placebo comparator | Other: Placebo iv (single-dose)Single dose (SD) of Placebo administered intravenously (iv) to White participants |
| Group 3: Japanese - Caplacizumab Dose 2 sc (SD) | Experimental | Biological: Caplacizumab Dose 2 sc (single-dose)Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants |
| Group 3: Japanese - Placebo sc (SD) | Placebo comparator | Other: Placebo sc (single-dose)Single dose (SD) of Placebo administered subcutaneously (sc) to Japanese participants |
| Group 3: White - Caplacizumab Dose 2 sc (SD) | Experimental | Biological: Caplacizumab Dose 2 sc (single-dose)Single dose (SD) of Caplacizumab Dose 2 administered subcutaneously (sc) to White participants |
| Group 3: White - Placebo sc (SD) | Placebo comparator | Other: Placebo sc (single-dose)Single dose (SD) Placebo administered subcutaneously (sc) to White participants |
| Group 4: Japanese - Caplacizumab Dose 2 sc (MD) | Experimental | Biological: Caplacizumab Dose 2 sc (multiple-dose)Multiple doses (MD) of Caplacizumab Dose 2 administered subcutaneously (sc) to Japanese participants |
| Group 4: Japanese - Placebo sc (MD) | Placebo comparator | Other: Placebo sc (multiple-dose)Multiple doses (MD) of Placebo administered subcutaneously (sc) to Japanese participants |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Caplacizumab Dose 1 iv (single-dose)Single intravenous (iv) administration of Caplacizumab Dose 1 | Biological | Group 1: Japanese - Caplacizumab Dose 1 iv (SD) |
| Caplacizumab Dose 2 iv (single-dose)Single intravenous (iv) administration of Caplacizumab Dose 2 | Biological | Group 2: Japanese - Caplacizumab Dose 2 iv (SD), Group 2: White - Caplacizumab Dose 2 iv (SD) |
| Caplacizumab Dose 2 sc (single-dose)Single subcutaneous (sc) administration of Caplacizumab Dose 2 | Biological | Group 3: Japanese - Caplacizumab Dose 2 sc (SD), Group 3: White - Caplacizumab Dose 2 sc (SD) |
| Caplacizumab Dose 2 sc (multiple-dose)Once daily subcutaneous (sc) administration of Caplacizumab Dose 2 during 7 consecutive days | Biological | Group 4: Japanese - Caplacizumab Dose 2 sc (MD) |
| Placebo iv (single-dose)Single intravenous (iv) administration of Placebo | Other | Group 1: Japanese - Placebo iv (SD), Group 2: Japanese - Placebo iv (SD), Group 2: White - Placebo iv (SD) |
| Placebo sc (single-dose)Single subcutaneous (sc) administration of Placebo | Other | Group 3: Japanese - Placebo sc (SD), Group 3: White - Placebo sc (SD) |
| Placebo sc (multiple-dose)Once daily subcutaneous (sc) administration of Placebo during 7 consecutive days | Other | Group 4: Japanese - Placebo sc (MD) |
Primary outcome
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)
6 secondary outcomes
Pharmacokinetics: concentration of caplacizumab in plasma
Time frame: 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)
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)
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)
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)
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2017.
Related trials for Healthy Volunteers
Not yet recruiting
Neuroergonomic Analysis of a Brain-Computer Interface Device (BCI)
Healthy Volunteers - Male and Female
RecruitingPhase 1
First-in-Human Trial to Assess REGN20423 in Healthy Adult Participants and Adult Participants With Atopic Dermatitis
Healthy Volunteer, Atopic Dermatitis
Not yet recruitingObservational study
Establishment of Biobank of Nasal Epithelium Samples From Healthy Volunteers
Healthy
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
Not yet recruitingObservational study
Screening Protocol
Pain
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ablynx Clinical Department study director
Ablynx, a Sanofi company
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Caplacizumab Dose 1 iv (single-dose), Caplacizumab Dose 2 iv (single-dose) and Caplacizumab Dose 2 sc (single-dose) in people with Healthy Volunteers, run by Ablynx, a Sanofi company at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Nov 2017.