A Phase 2 interventional study of Caplacizumab and Placebo in Acquired Thrombotic Thrombocytopenic Purpura, sponsored by Ablynx, a Sanofi company. Completed at 51 sites in 13 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-04-04.
Sponsored by Ablynx, a Sanofi company · Phase 2, Interventional, and Treatment
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).
263 studies on the registry are indexed under Purpura; 27 are open to participants now.
This study's enrollment of 75 is above the median of 50 across 171 interventional studies indexed under Purpura.
Browse Purpura studies →Ablynx, a Sanofi company is the lead sponsor of 24 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Known chronic treatment with anticoagulant treatment that cannot be stopped safely, including but not limited to:
Severe liver impairment, corresponding to grade 3 toxicity defined by the CTCAE scale. For the key liver parameters, this is defined as follows:
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.
Caplacizumab 10 mg once daily
Biological: Caplacizumab
Placebo once daily
Biological: Placebo
* 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.
Also known as: anti-vWF Nanobody, ALX-0081
* 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.
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').
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 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.
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
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
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.
Time frame: 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.
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
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
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.
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
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
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
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".
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
Total mortality up to 1 month follow-up.
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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)
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
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.
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
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).
Pharmacodynamics: Von Willebrand Factor Antigen (vWF:Ag) Over Time
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).
PD: Coagulation Factor VIII Clotting Activity (FVIII:C) Over Time
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).
Subjects with aTTP were recruited from 11 countries in Europe, Australia, Asia and United States (US). Only adults were recruited; no adolescent patients were enrolled, although the trial was open for adolescent patients. A total of 75 patients were included in the trial.
| Milestone | Caplacizumab | Placebo |
|---|---|---|
| Started | 36 | 39 |
| Received study drug | 35 | 37 |
| Completed | 20 | 21 |
| Not completed | 16 | 18 |
| Withdrew: Study terminated by sponsor | 9 | 10 |
| Withdrew: Protocol violation | 0 | 1 |
| Withdrew: Physician decision | 1 | 1 |
| Withdrew: Pregnancy | 0 | 1 |
| Withdrew: Death | 0 | 1 |
| Withdrew: Adverse event | 3 | 0 |
| Withdrew: Withdrawal by subject | 1 | 3 |
| Withdrew: Lost to follow-up | 1 | 0 |
| Withdrew: Diagnosed as non-ttp patient | 0 | 1 |
| Withdrew: Participated in other clinical trial | 1 | 0 |
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').
| days | Caplacizumab | Placebo |
|---|---|---|
| YES - One PE session prior to randomization | 2.4 (1.9 to 3) | 4.3 (2.9 to 5.7) |
| NO - No PE session prior to randomization | 3 (2.7 to 3.9) | 4.9 (3.2 to 6.6) |
Number and percentage of subjects with complete remission (defined as confirmed platelet count response and absence of exacerbation) following initial daily PE.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | 29 | 18 |
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.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Number and Percentage of Subjects With Exacerbations of TTP | 3 | 11 |
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.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Number and Percentage of Subjects With Relapse of TTP | 11 | 3 |
Number of daily PE sessions during the initial daily PE period which could include more than 1 PE per day was evaluated.
| PE sessions | Caplacizumab | Placebo |
|---|---|---|
| Number of Daily PE Sessions During the Initial Daily PE Period | 6.7 ± 3.69 | 8.4 ± 6.74 |
The total volume of plasma administered during the initial daily PE period was measured.
| mL | Caplacizumab | Placebo |
|---|---|---|
| Total Volume of Plasma Administered During the Initial Daily PE Period | 22481.8 ± 15914.85 | 28358.4 ± 21344.16 |
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.
| days | Caplacizumab | Placebo |
|---|---|---|
| Number of Days With at Least One PE Administration During the Total Course of the Study | 11.8 ± 7.43 | 12.6 ± 9.15 |
The maximum number of consecutive days per subject where there was no interruption of PE during the initial daily PE period.
| days | Caplacizumab | Placebo |
|---|---|---|
| The Maximum Number of Consecutive Days Per Subject Where There Was no Interruption of PE During the Initial Daily PE Period | 6.6 ± 3.35 | 8.1 ± 6.46 |
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.
| score on a scale | Caplacizumab | Placebo |
|---|---|---|
| Baseline | 66.76 ± 11.88 | 49.62 ± 12.91 |
| 12 months post discharge | 62.10 ± 12.06 | 58.36 ± 10.23 |
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".
| Participants | Caplacizumab | Placebo |
|---|---|---|
| End of daily PE treatment period | 29 | 29 |
| End of study treatment period | 30 | 33 |
| At 1 month follow-up | 31 | 27 |
Total mortality up to 1 month follow-up.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Mortality | 0 | 2 |
Number of PE treatment-related adverse events (AEs).
| adverse events | Caplacizumab | Placebo |
|---|---|---|
| Number of PE Related Adverse Events | 72 | 44 |
Number and percentage of subjects with PE related AEs.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Number and Percentage of Subjects With PE Related AEs | 20 | 20 |
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.
| adverse events | Caplacizumab | Placebo |
|---|---|---|
| Mild | 348 | 299 |
| Moderate | 154 | 173 |
| Severe | 37 | 23 |
Number and percentage of subjects with TEAEs by severity. The severity grades of AEs were defined as: mild, moderate, and severe.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Mild | 31 | 36 |
| Moderate | 27 | 31 |
| Severe | 18 | 14 |
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.
| adverse events | Caplacizumab | Placebo |
|---|---|---|
| Related | 12 | 6 |
| Possibly related | 59 | 9 |
| Unlikely/not related | 486 | 524 |
The development of anti-drug antibodies (ADA) was monitored from the start of the study until last follow-up visit.
| Participants | Caplacizumab | Placebo |
|---|---|---|
| Number of Participants Who Developed Treatment-emergent Anti-Drug Antibodies (ADA) | 3 | 0 |
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.
| ng/mL | Caplacizumab | Placebo |
|---|---|---|
| Baseline | 100 ± 0 | — |
| Day 1 of daily PE, 5 - 10 min postdose | 1765.9 ± 185.04 | — |
| Day 1 of daily PE, 3 - 6 hours postdose | 450.4 ± 36.15 | — |
| Day 1 of daily PE, 8 - 24 hours postdose | 562 ± 36.8 | — |
| Day 2 of daily PE, predose | 288 ± 24.05 | — |
| Day 2 of daily PE, 1 - 6 hours postdose | 415.8 ± 24.75 | — |
| Day 2 of daily PE, 6 - 12 hours postdose | 570.7 ± 52.22 | — |
| Day 2 of daily PE, 18 - 24 hours postdose | 489.3 ± 32.54 | — |
| Last day of daily PE, predose | 348.4 ± 38.32 | — |
| Day 1 after daily PE | 521.9 ± 31.52 | — |
| Week 1 after daily PE | 490.6 ± 36.11 | — |
| Week 2 after daily PE | 524.9 ± 39.37 | — |
| Week 3 after daily PE | 499.6 ± 35.1 | — |
| Week 4 after daily PE | 503.4 ± 31.21 | — |
| Day 3 of follow-up period | 346.7 ± 25.43 | — |
| Day 7 of follow-up period | 162.3 ± 20.2 | — |
| 1 month follow-up | 100 ± 0 | — |
The change from baseline in RICO activity was measured at different time points.
| percentage of RICO activity | Caplacizumab | Placebo |
|---|---|---|
| Baseline | 76.2 ± 4.95 | 82.1 ± 3.76 |
| Day 1 of daily PE, post dose | 16.2 ± 0.72 | 84.4 ± 6.09 |
| Day 2 of daily PE, post dose | 21.4 ± 3.64 | 94.4 ± 3.69 |
| Last day of daily PE, post dose | 15.4 ± 0.39 | 118.2 ± 1.78 |
| Day 1 after daily PE | 19.2 ± 3.94 | 105.2 ± 4.31 |
| Week 1 after daily PE | 15 ± 0 | 107.3 ± 3.05 |
| Week 2 after daily PE | 18.9 ± 2.87 | 109.2 ± 2.8 |
| Week 3 after daily PE | 17.4 ± 2.34 | 104 ± 3.46 |
| Week 4 after daily PE | 15.1 ± 0.07 | 105.5 ± 3.59 |
| Day 3 of follow-up period | 42.3 ± 6.33 | 99.1 ± 4.56 |
| Day 7 of follow-up period | 88.3 ± 4.99 | 99.7 ± 4.06 |
| 1 month follow-up | 94.6 ± 3.78 | 94.7 ± 5.35 |
The change from baseline in vWF:Ag concentration was measured at different time points.
| Percentage of vWF:Ag | Caplacizumab | Placebo |
|---|---|---|
| Baseline | 185.1 ± 15.09 | 204.4 ± 14.52 |
| Day 1 of daily PE, post dose | 120.6 ± 7.98 | 166.6 ± 9.24 |
| Day 2 of daily PE, post dose | 94.6 ± 4.83 | 140.2 ± 6.16 |
| Last day of daily PE, post dose | 93.6 ± 6.01 | 151.2 ± 14.25 |
| Day 1 after daily PE | 86.2 ± 6.15 | 166 ± 10.05 |
| Week 1 after daily PE | 93.4 ± 6.4 | 234.9 ± 20.91 |
| Week 2 after daily PE | 115.9 ± 12.42 | 242.6 ± 19.43 |
| Week 3 after daily PE | 102.4 ± 6.45 | 224.2 ± 18.62 |
| Week 4 after daily PE | 100.1 ± 5 | 204.3 ± 17.08 |
| Day 3 of follow-up period | 137.8 ± 9.32 | 184.1 ± 13.89 |
| Day 7 of follow-up period | 190.2 ± 12.9 | 190.3 ± 14.81 |
| 1 month follow-up | 176.3 ± 15.63 | 167.2 ± 15.46 |
The change from baseline in FVIII:C concentration was measured at different ime points.
| Percentage of FVIII:C activity | Caplacizumab | Placebo |
|---|---|---|
| Baseline | 144.18 ± 11.14 | 156.8 ± 12.54 |
| Day 1 of daily PE, post dose | 104 ± 6.95 | 149 ± 9.16 |
| Day 2 of daily PE, post dose | 90.7 ± 5.49 | 152.9 ± 9.41 |
| Last day of daily PE, post dose | 102.4 ± 10.91 | 169.5 ± 17.79 |
| Day 1 after daily PE | 116.3 ± 13.33 | 234.2 ± 16.05 |
| Week 1 after daily PE | 116.4 ± 11.73 | 296.8 ± 26.07 |
| Week 2 after daily PE | 125.2 ± 16.88 | 291.1 ± 18.25 |
| Week 3 after daily PE | 106.3 ± 9.7 | 273.1 ± 20.35 |
| Week 4 after daily PE | 95.8 ± 6.09 | 249.1 ± 18.27 |
| Day 3 of follow-up period | 146.3 ± 12.59 | 227.7 ± 17.32 |
| Day 7 of follow-up period | 208.6 ± 15.54 | 237.5 ± 15.65 |
| 1 month follow-up | 212.2 ± 17.33 | 200.1 ± 17.11 |
Collected over From time of the first study drug administration up to the last follow-up visit (12 months). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Caplacizumab | 0/35 (0%) | 20/35 (57.1%) | 33/35 (94.3%) |
| Placebo | 2/37 (5.4%) | 19/37 (51.4%) | 37/37 (100%) |
| Event | Caplacizumab | Placebo |
|---|---|---|
| Thrombotic thrombocytopenic purpuraBlood and lymphatic system disorders | 13/35 | 13/37 |
| DizzinessNervous system disorders | 2/35 | 0/37 |
| AnemiaBlood and lymphatic system disorders | 1/35 | 0/37 |
| Retinal hemorrhageEye disorders | 1/35 | 0/37 |
| HypertransaminasemiaHepatobiliary disorders | 1/35 | 0/37 |
| Bacterial infectionInfections and infestations | 1/35 | 0/37 |
| Muscle abscessInfections and infestations | 1/35 | 0/37 |
| SepsisInfections and infestations | 1/35 | 0/37 |
| Urinary tract infectionInfections and infestations | 1/35 | 0/37 |
| Alanine aminotransferase increasedInvestigations | 1/35 | 0/37 |
| Event | Caplacizumab | Placebo |
|---|---|---|
| HeadacheNervous system disorders | 11/35 | 10/37 |
| EpistaxisRespiratory, thoracic and mediastinal disorders | 11/35 | 4/37 |
| NauseaGastrointestinal disorders | 10/35 | 10/37 |
| ConstipationGastrointestinal disorders | 7/35 | 10/37 |
| HypokalemiaMetabolism and nutrition disorders | 9/35 | 8/37 |
| AnemiaBlood and lymphatic system disorders | 2/35 | 8/37 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 3/35 | 8/37 |
| ParaesthesiaNervous system disorders | 7/35 | 8/37 |
| VomitingGastrointestinal disorders | 7/35 | 7/37 |
| MyalgiaMusculoskeletal and connective tissue disorders | 7/35 | 1/37 |
| Age, Categorical(Participants) | Caplacizumab | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 35 | 38 | 73 |
| >=65 years | 1 | 1 | 2 |
| Age, Continuous(years) | Caplacizumab | Placebo | Total |
|---|---|---|---|
| Mean | 40.6 ± 12.7 | 42.5 ± 13.18 | 41.6 ± 12.9 |
| Sex: Female, Male(Participants) | Caplacizumab | Placebo | Total |
|---|---|---|---|
| Female | 24 | 20 | 44 |
| Male | 12 | 19 | 31 |
| Race/Ethnicity, Customized(Participants) | Caplacizumab | Placebo | Total |
|---|---|---|---|
| Race/Ethnicity — Caucasian | 32 | 34 | 66 |
| Race/Ethnicity — Black | 4 | 5 | 9 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: 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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