Trial · NCT01151423
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
NCT01151423, Phase 2, Completed, Ablynx, a Sanofi company; results first posted 2019-05-29
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP). Trials, record NCT01151423. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01151423
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
- NCT number
- NCT01151423
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 75 participants, actual
- Lead sponsor
- Ablynx, a Sanofi company
- Conditions
- Acquired Thrombotic Thrombocytopenic Purpura
- Interventions
- Caplacizumab, Placebo
- First posted
- 2010-06-28
- Last updated
- 2023-04-04
- Other registries' IDs
- eudract:2010-019375-30
- Results first posted
- 2019-05-29
- Limitations and caveats
The study was stopped prematurely before meeting its enrollment target. The study is not considered to have been terminated as sites were requested to perform the assessments until 1-month follow-up if they had subjects in the study.
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL | 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'). |
| Secondary | Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | 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 (defined as confirmed platelet count response and absence of exacerbation) following initial daily PE. |
| Secondary | Number and Percentage of Subjects With Exacerbations of TTP | Within 30 days of last day of initial daily PE | 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. |
| Secondary | Number and Percentage of Subjects With Relapse of TTP | Later than 30 days after the last daily PE | 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. |
| Secondary | Number of Daily PE Sessions During the Initial Daily PE Period | During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days | Number of daily PE sessions during the initial daily PE period which could include more than 1 PE per day was evaluated. |
| Secondary | Total Volume of Plasma Administered During the Initial Daily PE Period | During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days | The total volume of plasma administered during the initial daily PE period was measured. |
| Secondary | Number of Days With at Least One PE Administration During the Total Course of the Study | During the total course of the study (from Screening till the 12-month follow-up [FU] visit) | 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. |
| Secondary | The Maximum Number of Consecutive Days Per Subject Where There Was no Interruption of PE During the Initial Daily PE Period | During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days | The maximum number of consecutive days per subject where there was no interruption of PE during the initial daily PE period. |
| Secondary | Resolution of Non-focal Neurological Symptoms | From Baseline till the 12-month FU visit | 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. |
| Secondary | Number of Participants With Resolution of TTP-related Signs or Symptoms | 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 | 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". |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL | From the day of first study drug administration up to 30 days after first study drug administration | ITT Population | days | MEDIAN |
| SECONDARY | Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | From the day of first study drug administration up to 30 days after first study drug administration | ITT Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number and Percentage of Subjects With Exacerbations of TTP | Within 30 days of last day of initial daily PE | ITT Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number and Percentage of Subjects With Relapse of TTP | Later than 30 days after the last daily PE | ITT Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Daily PE Sessions During the Initial Daily PE Period | During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days | Number of subjects from the ITT Population with data available | PE sessions | MEAN |
| SECONDARY | Total Volume of Plasma Administered During the Initial Daily PE Period | During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days | Number of subjects from the ITT Population with data available | mL | MEAN |
| SECONDARY | Number of Days With at Least One PE Administration During the Total Course of the Study | During the total course of the study (from Screening till the 12-month follow-up [FU] visit) | Number of subjects from the ITT Population with data available | days | MEAN |
| SECONDARY | The Maximum Number of Consecutive Days Per Subject Where There Was no Interruption of PE During the Initial Daily PE Period | During the initial daily PE period, with a median (min, max) duration of exposure to study drug of 6 (2, 36) days | Number of subjects from the ITT Population with data available | days | MEAN |
| SECONDARY | Resolution of Non-focal Neurological Symptoms | From Baseline till the 12-month FU visit | The Computerized Neuropsychological Test Battery (CNTB) was completed by a low proportion of subjects with baseline data available for only 3 subjects in the caplacizumab and 4 subjects in the placebo group and 12 month post-discharge data available for 10 and 6 subjects, respectively. | score on a scale | MEAN |
| SECONDARY | Number of Participants With Resolution of TTP-related Signs or Symptoms | 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 | ITT Population | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group143
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL | Caplacizumab | YES - One PE session prior to randomization | 2.4 | 95% Confidence Interval 1.9 to 3 | days |
| Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL | Placebo | YES - One PE session prior to randomization | 4.3 | 95% Confidence Interval 2.9 to 5.7 | days |
| Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL | Caplacizumab | NO - No PE session prior to randomization | 3 | 95% Confidence Interval 2.7 to 3.9 | days |
| Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL | Placebo | NO - No PE session prior to randomization | 4.9 | 95% Confidence Interval 3.2 to 6.6 | days |
| Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | Caplacizumab | 29 | Participants | ||
| Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | Placebo | 18 | Participants | ||
| Number and Percentage of Subjects With Exacerbations of TTP | Caplacizumab | 3 | Participants | ||
| Number and Percentage of Subjects With Exacerbations of TTP | Placebo | 11 | Participants | ||
| Number and Percentage of Subjects With Relapse of TTP | Caplacizumab | 11 | Participants | ||
| Number and Percentage of Subjects With Relapse of TTP | Placebo | 3 | Participants |
1 to 10 of 143Source ClinicalTrials.gov
See all 143 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Caplacizumab | 36 | |
| Overall Study | STARTED | Placebo | 39 | |
| Overall Study | Received Study Drug | Caplacizumab | 35 | |
| Overall Study | Received Study Drug | Placebo | 37 | |
| Overall Study | COMPLETED | Caplacizumab | 20 | |
| Overall Study | COMPLETED | Placebo | 21 | |
| Overall Study | NOT COMPLETED | Caplacizumab | 16 | |
| Overall Study | NOT COMPLETED | Placebo | 18 |
Source ClinicalTrials.gov
Adverse events280
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | Caplacizumab | 2 | 35 | 2 |
| other | Anemia | Blood and lymphatic system disorders | Placebo | 8 | 37 | 34 |
| other | Leukocytosis | Blood and lymphatic system disorders | Caplacizumab | 0 | 35 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | Placebo | 2 | 37 | 2 |
| other | Neutrophilia | Blood and lymphatic system disorders | Caplacizumab | 0 | 35 | 0 |
| other | Neutrophilia | Blood and lymphatic system disorders | Placebo | 2 | 37 | 2 |
| other | Acute myocardial infarction | Cardiac disorders | Caplacizumab | 0 | 35 | 0 |
| other | Acute myocardial infarction | Cardiac disorders | Placebo | 2 | 37 | 2 |
| other | Cardiac disorder | Cardiac disorders | Caplacizumab | 2 | 35 | 3 |
| other | Cardiac disorder | Cardiac disorders | Placebo | 1 | 37 | 1 |
1 to 10 of 280Source ClinicalTrials.gov
See all 280 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Caplacizumab | 0 | 35 |
| Total, all-cause mortality | Placebo | 2 | 37 |
| Total, other adverse events | Caplacizumab | 33 | 35 |
| Total, other adverse events | Placebo | 37 | 37 |
| Total, serious adverse events | Caplacizumab | 20 | 35 |
| Total, serious adverse events | Placebo | 19 | 37 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons8
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Caplacizumab vs Placebo: Number of Participants Who Developed Treatment-emergent Anti-Drug Antibodies (ADA) | Trial comparisons |
| Caplacizumab vs Placebo: Mortality | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With PE Related AEs | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With Exacerbations of TTP | Trial comparisons |
| Caplacizumab vs Placebo: deaths from any cause | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With Relapse of TTP | Trial comparisons |
| Caplacizumab vs Placebo: serious adverse events | Trial comparisons |
Tests3
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| Choice Reaction Time | Secondary outcome | ConductCognition |
| Simple Reaction Time | Secondary outcome | ConductCognition |
| Verbal Learning | Secondary outcome | ConductCognition |
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01151423
GET https://conductscience.org/api/trials/v1/NCT01151423/registered?page=2