Acquired Thrombotic Thrombocytopenic PurpuraPhase 2TITANNCT01151423
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
A Phase 2 study testing Caplacizumab in people with Acquired Thrombotic Thrombocytopenic Purpura, run by Ablynx, a Sanofi company at 51 sites in 13 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acquired Thrombotic Thrombocytopenic Purpura
- What is being tested?
- Caplacizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 75 participants
- Where
- 51 sitesin 13 countries
- Study dates
- Jan 2011to Mar 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Show all 26Show fewer
- 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.
Trial locations and contacts
51 sites in 13 countries.
51 sites in 13 countries
United States13 sites
Investigator Site
Atlanta, Georgia, 30322
Investigator Site
Columbus, Ohio, 43210
Investigator Site
Dallas, Texas
Investigator Site
Durham, North Carolina, 27710
Investigator Site
Los Angeles, California, 90033
Investigator Site
New York, New York, 10021
Investigator Site
Pittsburgh, Pennsylvania, 15224
Investigator Site
Rochester, New York, 14642
Investigator Site
Salt Lake City, Utah, 84132
Investigator Site
St Louis, Missouri, 63110
Investigator Site
Valhalla, New York, 10595
Investigator Site
Washington D.C., District of Columbia
Investigator Site
Winston-Salem, North Carolina, 27157
Germany9 sites
Investigator Site
Aachen
Investigator Site
Berlin
Investigator Site
Cologne
Investigator Site
Dortmund
Investigator Site
Hanover
Investigator Site
Mainz
Investigator Site
Mannheim
Investigator Site
München
Investigator Site
Ulm, Baden-Wurttemberg
Italy5 sites
Investigator Site
Catania
Investigator Site
Foggia
Investigator Site
Milan
Investigator Site
Reggio Emilia
Investigator Site
Rome
Australia4 sites
Investigator Site
Garran, Australian Capital Territory
Investigator Site
Liverpool
Investigator Site
Melbourne
Investigator Site
Woolloongabba
Belgium4 sites
Investigator Site
Antwerp
Investigator Site
Brussels
Investigator Site
Leuven
Investigator Site
Namur
Israel3 sites
Investigator Site
Haifa
Investigator Site
Jerusalem
Investigator Site
Petah Tikva
Spain3 sites
Investigator Site
Badalona
Investigator Site
Seville
Investigator Site
Valencia
Switzerland3 sites
Investigator Site
Bern
Investigator Site
Lausanne
Investigator Site
Zurich
United Kingdom2 sites
Investigator Site
Liverpool
Investigator Site
London
About this study
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
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).
- Being tested
- Caplacizumab, Placebo
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CaplacizumabBiological
Also called anti-vWF Nanobody, ALX-0081
- 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.
PlaceboBiological
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Caplacizumab | Experimental | Caplacizumab 10 mg once daily Receives: Biological: Caplacizumab |
| Placebo | Placebo comparator | Placebo once daily Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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').
Measured: From the day of first study drug administration up to 30 days after first study drug administration
Secondary outcomes (20)
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.
Measured: From the day of first study drug administration up to 30 days after first study drug administration
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.
Measured: Within 30 days of last day of initial daily PE
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.
Measured: Later than 30 days after the last daily PE
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.
Measured: During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days
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.
Measured: During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days
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.
Measured: During the total course of the study (from Screening till the 12-month follow-up [FU] visit)
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.
Measured: During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days
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.
Measured: From Baseline till the 12-month FU visit
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".
Measured: 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
Mortality
Total mortality up to 1 month follow-up.
Measured: From the start of the study up to 1 month follow-up
Number of PE Related Adverse Events
Number of PE treatment-related adverse events (AEs).
Measured: From the start of the study up to 1 month follow-up
Number and Percentage of Subjects With PE Related AEs
Number and percentage of subjects with PE related AEs.
Measured: From the start of the study up to 1 month follow-up
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.
Measured: From the start of the study up to 1 month follow-up
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.
Measured: From the start of the study up to 1 month follow-up
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.
Measured: From the start of the study up to 1 month follow-up
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.
Measured: 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.
Measured: 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).
Pharmacodynamics (PD): Ristocetin Cofactor (RICO) Activity Over Time
The change from baseline in RICO activity was measured at different time points.
Measured: 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).
Pharmacodynamics: Von Willebrand Factor Antigen (vWF:Ag) Over Time
The change from baseline in vWF:Ag concentration was measured at different time points.
Measured: 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).
PD: Coagulation Factor VIII Clotting Activity (FVIII:C) Over Time
The change from baseline in FVIII:C concentration was measured at different ime points.
Measured: 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).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2010 (estimated)
- Study startJan 2011
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Acquired Thrombotic Thrombocytopenic PurpuraStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ablynx, a Sanofi company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Ablynx NV
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01151423
- Sponsor's protocol ID
- ALX0681-2.1/10
- Other IDs
- 2010-019375-30
- Acronym
- TITAN
- Status last verified
- May 2019
- First submitted
- 25 Jun 2010
- Last updated
- 4 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: 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
Publications
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.
PubMed 33881463Peyvandi 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.
PubMed 26863353Holz 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.
PubMed 22475545
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01151423
- Acronym / sponsor ID
- TITAN / ALX0681-2.1/10
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Ablynx, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- Jan 2011 (actual)
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 28 Jun 2010 (estimated)
- Results
- Posted 29 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Caplacizumab | Experimental | Biological: CaplacizumabCaplacizumab 10 mg once daily |
| Placebo | Placebo comparator | Biological: PlaceboPlacebo once daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (26 listed)
- 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)
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 |
|---|---|---|
| CaplacizumabAlso: anti-vWF Nanobody, ALX-0081* 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. | Biological | Caplacizumab |
| Placebo* 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. | Biological | Placebo |
Primary outcome
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL
Time frame: From the day of first study drug administration up to 30 days after first study drug administration
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').
20 secondary outcomes
Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE)
Time frame: From the day of first study drug administration up to 30 days after first study drug administration
Number and Percentage of Subjects With Exacerbations of TTP
Time frame: Within 30 days of last day of initial daily PE
Number and Percentage of Subjects With Relapse of TTP
Time frame: Later than 30 days after the last daily PE
Number of Daily PE Sessions 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
Total Volume of Plasma Administered 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
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)
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
Resolution of Non-focal Neurological Symptoms
Time frame: From Baseline till the 12-month FU visit
Number of Participants With Resolution of TTP-related Signs or Symptoms
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
Mortality
Time frame: From the start of the study up to 1 month follow-up
Number of PE Related Adverse Events
Time frame: From the start of the study up to 1 month follow-up
Number and Percentage of Subjects With PE Related AEs
Time frame: From the start of the study up to 1 month follow-up
Number of Treatment-emergent Adverse Events (TEAEs) by Severity
Time frame: From the start of the study up to 1 month follow-up
Number and Percentage of Subjects With TEAEs by Severity
Time frame: From the start of the study up to 1 month follow-up
Number of TEAEs and Their Relationship to Study Drug
Time frame: From the start of the study up to 1 month follow-up
Number of Participants Who Developed Treatment-emergent Anti-Drug Antibodies (ADA)
Time frame: From the start of the study until last follow-up visit
Plasma Concentrations of Caplacizumab
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).
Pharmacodynamics (PD): Ristocetin Cofactor (RICO) Activity Over Time
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).
Pharmacodynamics: Von Willebrand Factor Antigen (vWF:Ag) Over Time
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).
PD: Coagulation Factor VIII Clotting Activity (FVIII:C) Over Time
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).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33881463Peyvandi 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.
- Derived from this study: PubMed 26863353Peyvandi 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.
- Derived from this study: PubMed 22475545Holz 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.
- Individual participant dataSharing planned: Yes. 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
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Acquired Thrombotic Thrombocytopenic Purpura
RecruitingObservational study
Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura
Thrombotic Thrombocytopenic Purpura, Acquired
Recruiting
Association of TNFAIP3 With Immune-mediated TTP
Thrombotic Thrombocytopenic Purpura, Acquired
Active, not recruitingObservational study
Improvement of Immunologic and Molecular Techniques for the Diagnosis and Follow-up of Patients With Thrombotic Thrombocytopenic Purpura
Acquired Thrombotic Thrombocytopenic Purpura
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.
Medical Monitor study director
Ablynx NV
51 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Caplacizumab in people with Acquired Thrombotic Thrombocytopenic Purpura, run by Ablynx, a Sanofi company at 51 sites in 13 countries.
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 6 inclusion and 26 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 4 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in United States, Germany, Italy, Australia, Belgium and 8 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL (time frame: From the day of first study drug administration up to 30 days after first study drug administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01151423, which also lists 3 linked publications.
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 4 Apr 2023.