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Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) (TITAN)

Primary Purpose

Acquired Thrombotic Thrombocytopenic Purpura

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Caplacizumab
Placebo
Sponsored by
Ablynx, a Sanofi company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acquired Thrombotic Thrombocytopenic Purpura

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 18 years of age or older (adults) or aged 12 to < 18 years (adolescents)
  • Male or female subject, willing to accept an acceptable contraceptive regimen
  • Subject with a clinical diagnosis of TTP
  • Requiring PE (one single PE session prior to randomization into the study was allowed)
  • Subject accessible to follow-up
  • Subject able to provide signed and dated informed consent and assent (if applicable, for adolescents)

Exclusion Criteria:

  • Platelet count ≥ 100,000/µL
  • Severe active infection indicated by sepsis (requirement for pressors with or without positive blood cultures)
  • Clinical evidence of enteric infection with Escherichia coli 0157 or related organism
  • Anti-phospholipid syndrome
  • Diagnosis of disseminated intravascular coagulation (DIC)
  • Pregnancy or breast-feeding
  • Hematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy
  • Known with congenital TTP
  • Active bleeding or high risk of bleeding
  • Uncontrolled arterial hypertension

  • Known chronic treatment with anticoagulant treatment that cannot be stopped safely, including but not limited to:

    • vitamin K antagonists
    • heparin or low molecular weight heparin (LMWH)
    • non-acetyl salicylic acid non-steroidal anti-inflammatory molecules
  • Severe or life threatening clinical condition other than TTP that would impair participation in the study
  • Subjects with malignancies resulting in a life expectation of less than 3 months
  • Subjects with known or suspected bone marrow carcinosis
  • Subjects who cannot comply with study protocol requirements and procedures
  • Known hypersensitivity to the active substance or to excipients of the study drug

  • Severe liver impairment, corresponding to grade 3 toxicity defined by the CTCAE scale. For the key liver parameters, this is defined as follows:

    • bilirubin > 3 x upper limit of normal (ULN) (needed to differentiate isolated increase in indirect bilirubin due to hemolysis, this was not an exclusion parameter but disease-related)
    • alanine transaminase (ALT)/ aspartate transaminase (AST) > 5 x ULN
    • alkaline phosphatase (ALP) > 5 x ULN
    • gamma-glutamyl transpeptidase (GGT) > 5 x ULN
  • Severe chronic renal impairment, as defined by glomerular filtration rate < 30 mL/min

Note that the use of another investigational drug or device within 30 days prior to screening was not allowed. Participation in non-interventional/observational studies and registries during the study period was allowed. Participation in another clinical study was not allowed until the end of the follow-up period or within 30 days after the last study treatment in case of early subject withdrawal from the study. Subjects who had already participated in the current study and had either completed the study per protocol or had discontinued prematurely, were not allowed to be re-included.

Sites / Locations

  • Investigator Site
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  • Investigator Site
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  • Investigator Site
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  • Investigator Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Caplacizumab

Placebo

Arm Description

Caplacizumab 10 mg once daily

Placebo once daily

Outcomes

Primary Outcome Measures

Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL
Time-to-response, defined by the achievement of platelet count response, confirmed at 48 hours after the initial reporting of this response. Platelet response was defined as recovery of platelets ≥ 150,000/µL. This response had to be confirmed at 48 hours after the initial reporting of platelet recovery ≥ 150,000/µL by a de novo measure of platelets ≥ 150,000/µL and lactate dehydrogenase (LDH) ≤ 2x upper limit of normal (ULN) (i.e., 'confirmed platelet response').

Secondary Outcome Measures

Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE)
Number and percentage of subjects with complete remission (defined as confirmed platelet count response and absence of exacerbation) following initial daily PE.
Number and Percentage of Subjects With Exacerbations of TTP
Number and percentage of subjects with exacerbations of TTP (defined as recurrent thrombocytopenia following a confirmed platelet count response and requiring a re-initiation of daily PE treatment after ≥ 1 day but ≤ 30 days of end of daily PE treatment. Time to first exacerbation of TTP was also examined as part of this end point analysis; the median time to first exacerbation could not be determined because of the small number of events.
Number and Percentage of Subjects With Relapse of TTP
Number and percentage of subjects with relapse of TTP (defined as de novo TTP event that occurred later than 30 days after the last daily PE) was evaluated.
Number of Daily PE Sessions During the Initial Daily PE Period
Number of daily PE sessions during the initial daily PE period which could include more than 1 PE per day was evaluated.
Total Volume of Plasma Administered During the Initial Daily PE Period
The total volume of plasma administered during the initial daily PE period was measured.
Number of Days With at Least One PE Administration During the Total Course of the Study
Number of days for PE was evaluated. This implies the number of days with at least one PE administration during the total course of the study.
The Maximum Number of Consecutive Days Per Subject Where There Was no Interruption of PE During the Initial Daily PE Period
The maximum number of consecutive days per subject where there was no interruption of PE during the initial daily PE period.
Resolution of Non-focal Neurological Symptoms
Resolution of non-focal neurological symptoms as defined by neurocognitive function at complete remission, measured by a Computerised Neuropsychological Test Battery(CNTB) (adults only). The CNTB included 6 modules: word list learning and selective reminding (WLL/SR), choice reaction time (CRT), visual memory (VMEM), simple reaction time (SRT), working memory (WMEM), and word list learning and delayed recall (WLL/DR). The 6 tasks are rated as a percentage correct (for CRT and SRT, the percentage correct corresponds to the percentage hits) and the mean score from these provides the CNTB summary score (range: 0-100). A higher CNTB summary score indicates better neuropsychological functioning.
Number of Participants With Resolution of TTP-related Signs or Symptoms
Resolution or improvement (improvement of ≥ 1 grade in the Common Terminology Criteria for Adverse Events [CTCAE] v4.0 scale) of TTP-related signs and symptoms as captured on physical examination and as adverse events. This endpoint was only evaluated for "resolution".
Mortality
Total mortality up to 1 month follow-up.
Number of PE Related Adverse Events
Number of PE treatment-related adverse events (AEs).
Number and Percentage of Subjects With PE Related AEs
Number and percentage of subjects with PE related AEs.
Number of Treatment-emergent Adverse Events (TEAEs) by Severity
Number and severity of TEAEs were evaluated. The severity grades of AEs were defined as: mild, moderate, and severe. Note: the numbers listed do not include the TEAEs with missing severity.
Number and Percentage of Subjects With TEAEs by Severity
Number and percentage of subjects with TEAEs by severity. The severity grades of AEs were defined as: mild, moderate, and severe.
Number of TEAEs and Their Relationship to Study Drug
Number of TEAEs and their relationship to study drug were evaluated. Relationship of AEs to study drug was: related, possibly related, and unlikely/not related.
Number of Participants Who Developed Treatment-emergent Anti-Drug Antibodies (ADA)
The development of anti-drug antibodies (ADA) was monitored from the start of the study until last follow-up visit.
Plasma Concentrations of Caplacizumab
The concentration of caplacizumab in plasma was determined at different time points. pharmacokinetics (PK) Population: the PK Population consisted of all subjects who received the study drug and for whom the primary PK data are considered to be sufficient and interpretable.
Pharmacodynamics (PD): Ristocetin Cofactor (RICO) Activity Over Time
The change from baseline in RICO activity was measured at different time points.
Pharmacodynamics: Von Willebrand Factor Antigen (vWF:Ag) Over Time
The change from baseline in vWF:Ag concentration was measured at different time points.
PD: Coagulation Factor VIII Clotting Activity (FVIII:C) Over Time
The change from baseline in FVIII:C concentration was measured at different ime points.

Full Information

First Posted
June 25, 2010
Last Updated
March 31, 2023
Sponsor
Ablynx, a Sanofi company
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1. Study Identification

Unique Protocol Identification Number
NCT01151423
Brief Title
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Acronym
TITAN
Official Title
A Phase II, Single-blind, Randomized, Placebo-controlled Trial to Study the Efficacy and Safety of Anti-von Willebrand Factor Nanobody Administered as Adjunctive Treatment to Patients With Acquired Thrombotic Thrombocytopenic Purpura
Study Type
Interventional

2. Study Status

Record Verification Date
May 2019
Overall Recruitment Status
Completed
Study Start Date
January 2011 (Actual)
Primary Completion Date
March 2014 (Actual)
Study Completion Date
March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ablynx, a Sanofi company

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This study was a Phase II, single-blind, randomized, placebo-controlled trial to determine whether anti-vWF Nanobody is safe and effective as adjunctive treatment in patients with aTTP. Patients received either placebo or anti-vWF Nanobody as adjunctive therapy to plasma exchange (PE).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acquired Thrombotic Thrombocytopenic Purpura

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Participant
Masking Description
A single-blinded study design was initiated because, in the initial versions of the protocol, the dosing regimen could be adjusted dependent on the results of the Ristocetin Cofactor (RICO) test following the initial dose. Therefore, a double-blind design was not feasible because the results of the RICO test effectively unblinded the Investigator. Single-blind design was maintained due to the objective nature of the primary endpoint.
Allocation
Randomized
Enrollment
75 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Caplacizumab
Arm Type
Experimental
Arm Description
Caplacizumab 10 mg once daily
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo once daily
Intervention Type
Biological
Intervention Name(s)
Caplacizumab
Other Intervention Name(s)
anti-vWF Nanobody, ALX-0081
Intervention Description
Subjects received a first intravenous (i.v.) bolus of 10 mg (filled at 5 mg/mL) caplacizumab via push injection within 6 hours to 15 minutes prior to the first PE on study. The first PE on study could either be the very first PE session for the current episode of aTTP (if the subject was randomized prior to the initiation of PE) or the second PE session (if the subject was randomized after a single PE session). The first PE on study was followed by subcutaneous (s.c.) administration of 10 mg study drug within 30 minutes after the end of the PE procedure. All subsequent study drug administrations were daily s.c. injections within 30 minutes after the end of the PE procedure (if applicable) or within 24 hours of the previous dose. Subjects received caplacizumab up to 30 days after the last PE session.
Intervention Type
Biological
Intervention Name(s)
Placebo
Intervention Description
Subjects received a first i.v. bolus of placebo via push injection within 6 hours to 15 minutes prior to the first PE on study. The first PE on study could either be the very first PE session for the current episode of aTTP (if the subject was randomized prior to the initiation of PE) or the second PE session (if the subject was randomized after a single PE session). The first PE on study was followed by s.c. administration of placebo within 30 minutes after the end of the PE procedure. All subsequent study drug administrations were daily s.c. injections within 30 minutes after the end of the PE procedure (if applicable) or within 24 hours of the previous dose. Subjects received placebo up to 30 days after the last PE session.
Primary Outcome Measure Information:
Title
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL
Description
Time-to-response, defined by the achievement of platelet count response, confirmed at 48 hours after the initial reporting of this response. Platelet response was defined as recovery of platelets ≥ 150,000/µL. This response had to be confirmed at 48 hours after the initial reporting of platelet recovery ≥ 150,000/µL by a de novo measure of platelets ≥ 150,000/µL and lactate dehydrogenase (LDH) ≤ 2x upper limit of normal (ULN) (i.e., 'confirmed platelet response').
Time Frame
From the day of first study drug administration up to 30 days after first study drug administration
Secondary Outcome Measure Information:
Title
Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE)
Description
Number and percentage of subjects with complete remission (defined as confirmed platelet count response and absence of exacerbation) following initial daily PE.
Time Frame
From the day of first study drug administration up to 30 days after first study drug administration
Title
Number and Percentage of Subjects With Exacerbations of TTP
Description
Number and percentage of subjects with exacerbations of TTP (defined as recurrent thrombocytopenia following a confirmed platelet count response and requiring a re-initiation of daily PE treatment after ≥ 1 day but ≤ 30 days of end of daily PE treatment. Time to first exacerbation of TTP was also examined as part of this end point analysis; the median time to first exacerbation could not be determined because of the small number of events.
Time Frame
Within 30 days of last day of initial daily PE
Title
Number and Percentage of Subjects With Relapse of TTP
Description
Number and percentage of subjects with relapse of TTP (defined as de novo TTP event that occurred later than 30 days after the last daily PE) was evaluated.
Time Frame
Later than 30 days after the last daily PE
Title
Number of Daily PE Sessions During the Initial Daily PE Period
Description
Number of daily PE sessions during the initial daily PE period which could include more than 1 PE per day was evaluated.
Time Frame
During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days
Title
Total Volume of Plasma Administered During the Initial Daily PE Period
Description
The total volume of plasma administered during the initial daily PE period was measured.
Time Frame
During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days
Title
Number of Days With at Least One PE Administration During the Total Course of the Study
Description
Number of days for PE was evaluated. This implies the number of days with at least one PE administration during the total course of the study.
Time Frame
During the total course of the study (from Screening till the 12-month follow-up [FU] visit)
Title
The Maximum Number of Consecutive Days Per Subject Where There Was no Interruption of PE During the Initial Daily PE Period
Description
The maximum number of consecutive days per subject where there was no interruption of PE during the initial daily PE period.
Time Frame
During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days
Title
Resolution of Non-focal Neurological Symptoms
Description
Resolution of non-focal neurological symptoms as defined by neurocognitive function at complete remission, measured by a Computerised Neuropsychological Test Battery(CNTB) (adults only). The CNTB included 6 modules: word list learning and selective reminding (WLL/SR), choice reaction time (CRT), visual memory (VMEM), simple reaction time (SRT), working memory (WMEM), and word list learning and delayed recall (WLL/DR). The 6 tasks are rated as a percentage correct (for CRT and SRT, the percentage correct corresponds to the percentage hits) and the mean score from these provides the CNTB summary score (range: 0-100). A higher CNTB summary score indicates better neuropsychological functioning.
Time Frame
From Baseline till the 12-month FU visit
Title
Number of Participants With Resolution of TTP-related Signs or Symptoms
Description
Resolution or improvement (improvement of ≥ 1 grade in the Common Terminology Criteria for Adverse Events [CTCAE] v4.0 scale) of TTP-related signs and symptoms as captured on physical examination and as adverse events. This endpoint was only evaluated for "resolution".
Time Frame
End of daily PE treatment period (median [min, max] duration of exposure to study drug of 6 [2, 36] days), end of study treatment period (median [min, max] duration of exposure to study drug of 36.5 [2, 90] days) and at 1 month follow-up
Title
Mortality
Description
Total mortality up to 1 month follow-up.
Time Frame
From the start of the study up to 1 month follow-up
Title
Number of PE Related Adverse Events
Description
Number of PE treatment-related adverse events (AEs).
Time Frame
From the start of the study up to 1 month follow-up
Title
Number and Percentage of Subjects With PE Related AEs
Description
Number and percentage of subjects with PE related AEs.
Time Frame
From the start of the study up to 1 month follow-up
Title
Number of Treatment-emergent Adverse Events (TEAEs) by Severity
Description
Number and severity of TEAEs were evaluated. The severity grades of AEs were defined as: mild, moderate, and severe. Note: the numbers listed do not include the TEAEs with missing severity.
Time Frame
From the start of the study up to 1 month follow-up
Title
Number and Percentage of Subjects With TEAEs by Severity
Description
Number and percentage of subjects with TEAEs by severity. The severity grades of AEs were defined as: mild, moderate, and severe.
Time Frame
From the start of the study up to 1 month follow-up
Title
Number of TEAEs and Their Relationship to Study Drug
Description
Number of TEAEs and their relationship to study drug were evaluated. Relationship of AEs to study drug was: related, possibly related, and unlikely/not related.
Time Frame
From the start of the study up to 1 month follow-up
Title
Number of Participants Who Developed Treatment-emergent Anti-Drug Antibodies (ADA)
Description
The development of anti-drug antibodies (ADA) was monitored from the start of the study until last follow-up visit.
Time Frame
From the start of the study until last follow-up visit
Title
Plasma Concentrations of Caplacizumab
Description
The concentration of caplacizumab in plasma was determined at different time points. pharmacokinetics (PK) Population: the PK Population consisted of all subjects who received the study drug and for whom the primary PK data are considered to be sufficient and interpretable.
Time Frame
From the start of the study up to 1 month follow-up (i.e., at Baseline, Days 1 and 2 of daily PE, last day of daily PE, Day 1 after daily PE, Weeks 1, 2, 3, and 4 after daily PE, Days 3 and 7 of FU and at the 1-month FU visit).
Title
Pharmacodynamics (PD): Ristocetin Cofactor (RICO) Activity Over Time
Description
The change from baseline in RICO activity was measured at different time points.
Time Frame
From the start of the study up to 1 month follow-up (i.e., at Baseline, Days 1 and 2 of daily PE, last day of daily PE, Day 1 after daily PE, Weeks 1, 2, 3, and 4 after daily PE, Days 3 and 7 of FU and at the 1-month FU visit).
Title
Pharmacodynamics: Von Willebrand Factor Antigen (vWF:Ag) Over Time
Description
The change from baseline in vWF:Ag concentration was measured at different time points.
Time Frame
From the start of the study drug up to 1 month follow-up (i.e., at Baseline, Days 1 and 2 of daily PE, last day of daily PE, Day 1 after daily PE, Weeks 1, 2, 3, and 4 after daily PE, Days 3 and 7 of FU and at the 1-month FU visit).
Title
PD: Coagulation Factor VIII Clotting Activity (FVIII:C) Over Time
Description
The change from baseline in FVIII:C concentration was measured at different ime points.
Time Frame
From the start of the study up to 1 month follow-up (i.e., at Baseline, Days 1 and 2 of daily PE, last day of daily PE, Day 1 after daily PE, Weeks 1, 2, 3, and 4 after daily PE, Days 3 and 7 of FU and at the 1-month FU visit).

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 18 years of age or older (adults) or aged 12 to < 18 years (adolescents) Male or female subject, willing to accept an acceptable contraceptive regimen Subject with a clinical diagnosis of TTP Requiring PE (one single PE session prior to randomization into the study was allowed) Subject accessible to follow-up Subject able to provide signed and dated informed consent and assent (if applicable, for adolescents) Exclusion Criteria: Platelet count ≥ 100,000/µL Severe active infection indicated by sepsis (requirement for pressors with or without positive blood cultures) Clinical evidence of enteric infection with Escherichia coli 0157 or related organism Anti-phospholipid syndrome Diagnosis of disseminated intravascular coagulation (DIC) Pregnancy or breast-feeding Hematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy Known with congenital TTP Active bleeding or high risk of bleeding Uncontrolled arterial hypertension Known chronic treatment with anticoagulant treatment that cannot be stopped safely, including but not limited to: vitamin K antagonists heparin or low molecular weight heparin (LMWH) non-acetyl salicylic acid non-steroidal anti-inflammatory molecules Severe or life threatening clinical condition other than TTP that would impair participation in the study Subjects with malignancies resulting in a life expectation of less than 3 months Subjects with known or suspected bone marrow carcinosis Subjects who cannot comply with study protocol requirements and procedures Known hypersensitivity to the active substance or to excipients of the study drug Severe liver impairment, corresponding to grade 3 toxicity defined by the CTCAE scale. For the key liver parameters, this is defined as follows: bilirubin > 3 x upper limit of normal (ULN) (needed to differentiate isolated increase in indirect bilirubin due to hemolysis, this was not an exclusion parameter but disease-related) alanine transaminase (ALT)/ aspartate transaminase (AST) > 5 x ULN alkaline phosphatase (ALP) > 5 x ULN gamma-glutamyl transpeptidase (GGT) > 5 x ULN Severe chronic renal impairment, as defined by glomerular filtration rate < 30 mL/min Note that the use of another investigational drug or device within 30 days prior to screening was not allowed. Participation in non-interventional/observational studies and registries during the study period was allowed. Participation in another clinical study was not allowed until the end of the follow-up period or within 30 days after the last study treatment in case of early subject withdrawal from the study. Subjects who had already participated in the current study and had either completed the study per protocol or had discontinued prematurely, were not allowed to be re-included.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Ablynx NV
Official's Role
Study Director
Facility Information:
Facility Name
Investigator Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Investigator Site
City
Washington
State/Province
District of Columbia
Country
United States
Facility Name
Investigator Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Investigator Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Investigator Site
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Investigator Site
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Investigator Site
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Facility Name
Investigator Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Investigator Site
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Investigator Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Investigator Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Investigator Site
City
Dallas
State/Province
Texas
Country
United States
Facility Name
Investigator Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Investigator Site
City
Garran
State/Province
Australian Capital Territory
Country
Australia
Facility Name
Investigator Site
City
Liverpool
Country
Australia
Facility Name
Investigator Site
City
Melbourne
Country
Australia
Facility Name
Investigator Site
City
Woolloongabba
Country
Australia
Facility Name
Investigator Site
City
Graz
Country
Austria
Facility Name
Investigator Site
City
Vienna
Country
Austria
Facility Name
Investigator Site
City
Antwerp
Country
Belgium
Facility Name
Investigator Site
City
Brussels
Country
Belgium
Facility Name
Investigator Site
City
Leuven
Country
Belgium
Facility Name
Investigator Site
City
Namur
Country
Belgium
Facility Name
Investigator Site
City
Sofia
Country
Bulgaria
Facility Name
Investigator Site
City
Caen
Country
France
Facility Name
Investigator Site
City
Ulm
State/Province
Baden-Wuerttemberg
Country
Germany
Facility Name
Investigator Site
City
Aachen
Country
Germany
Facility Name
Investigator Site
City
Berlin
Country
Germany
Facility Name
Investigator Site
City
Dortmund
Country
Germany
Facility Name
Investigator Site
City
Hannover
Country
Germany
Facility Name
Investigator Site
City
Köln
Country
Germany
Facility Name
Investigator Site
City
Mainz
Country
Germany
Facility Name
Investigator Site
City
Mannheim
Country
Germany
Facility Name
Investigator Site
City
Munchen
Country
Germany
Facility Name
Investigator Site
City
Haifa
Country
Israel
Facility Name
Investigator Site
City
Jerusalem
Country
Israel
Facility Name
Investigator Site
City
Petach Tikva
Country
Israel
Facility Name
Investigator Site
City
Catania
Country
Italy
Facility Name
Investigator Site
City
Foggia
Country
Italy
Facility Name
Investigator Site
City
Milan
Country
Italy
Facility Name
Investigator Site
City
Reggio Emilia
Country
Italy
Facility Name
Investigator Site
City
Rome
Country
Italy
Facility Name
Investigator Site
City
Bucharest
Country
Romania
Facility Name
Investigator Site
City
Badalona
Country
Spain
Facility Name
Investigator Site
City
Sevilla
Country
Spain
Facility Name
Investigator Site
City
Valencia
Country
Spain
Facility Name
Investigator Site
City
Bern
Country
Switzerland
Facility Name
Investigator Site
City
Lausanne
Country
Switzerland
Facility Name
Investigator Site
City
Zurich
Country
Switzerland
Facility Name
Investigator Site
City
Liverpool
Country
United Kingdom
Facility Name
Investigator Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Citations:
PubMed Identifier
33881463
Citation
Peyvandi F, Cataland S, Scully M, Coppo P, Knoebl P, Kremer Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Minkue Mi Edou J, De Winter H, Callewaert F. Caplacizumab prevents refractoriness and mortality in acquired thrombotic thrombocytopenic purpura: integrated analysis. Blood Adv. 2021 Apr 27;5(8):2137-2141. doi: 10.1182/bloodadvances.2020001834.
Results Reference
derived
PubMed Identifier
26863353
Citation
Peyvandi F, Scully M, Kremer Hovinga JA, Cataland S, Knobl P, Wu H, Artoni A, Westwood JP, Mansouri Taleghani M, Jilma B, Callewaert F, Ulrichts H, Duby C, Tersago D; TITAN Investigators. Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2016 Feb 11;374(6):511-22. doi: 10.1056/NEJMoa1505533.
Results Reference
derived
PubMed Identifier
22475545
Citation
Holz JB. The TITAN trial--assessing the efficacy and safety of an anti-von Willebrand factor Nanobody in patients with acquired thrombotic thrombocytopenic purpura. Transfus Apher Sci. 2012 Jun;46(3):343-6. doi: 10.1016/j.transci.2012.03.027. Epub 2012 Apr 3.
Results Reference
derived

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Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

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