Trial · NCT02553317
Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura
NCT02553317, Phase 3, Completed, Ablynx, a Sanofi company; results first posted 2019-05-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura. Trials, record NCT02553317. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02553317
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura
- NCT number
- NCT02553317
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 145 participants, actual
- Lead sponsor
- Ablynx, a Sanofi company
- Conditions
- Acquired Thrombotic Thrombocytopenic Purpura
- Interventions
- Caplacizumab, Placebo
- First posted
- 2015-09-17
- Last updated
- 2023-04-05
- Other registries' IDs
- eudract:2015-001098-42
- Results first posted
- 2019-05-22
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time to Platelet Count Response | Only data from the DB daily PE period (median = 5 days) up to the cut-off were used. The cut-off point was defined by whichever occured first: 1) 45 days of daily PE after start of study drug, 2) stop of daily PE, 3) stop of study drug (median = 34 days) | Platelet count response was defined as initial platelet count ≥ 150,000/μL with subsequent stop of daily PE within 5 days. It refers to the first time both conditions, platelet count ≥ 150,000/μL and the stop of daily PE within 5 days, were met. |
| Secondary | Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | The study drug treatment period, a median (min, max) of 36 (2, 82) days. For both treatment groups, only events that occurred prior to a switch to open-label caplacizumab were evaluated for this analysis. | Number and percentage of subjects with TTP-related death, a recurrence of TTP, or at least one treatment-emergent major thromboembolic event during the study drug treatment period (i.e., first key secondary endpoint). |
| Secondary | Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | The overall study period (covers both the overall treatment period and the follow-up period), a median (min, max) of 65 (2, 110) days. | Number and percentage of subjects with a recurrence of TTP during the Overall Study Period (i.e., including follow-up \[FU\]) (i.e., second key secondary endpoint). |
| Secondary | Number and Percentage of Subjects With Refractory Disease | The study drug treatment period, a median (min, max) of 36 (2, 82) days. | Number and percentage of subjects with refractory TTP, defined as absence of platelet count doubling after 4 days of standard treatment, and lactate dehydrogenase (LDH) \> upper limit of normal (ULN) (i.e., third key secondary endpoint). |
| Secondary | Time to Normalization of Organ Damage Marker Levels | Overall study period, a median (min, max) of 65 (2, 110) days. For both treatment groups, normalizations occurring during the open-label period were not evaluated in this analysis. | Time to first normalization of LDH, cardiac troponin I (cTnI) and serum creatinine was defined as: first time of LDH ≤ ULN and cTnI ≤ ULN and serum creatinine ≤ ULN - time of first i.v. loading dose of study drug after randomization + 1 minute. Subjects in either initial treatment group who switched to open-label caplacizumab before having reached the endpoint were censored at time of switch. Of note, the key secondary endpoints were hierarchically ordered to allow statistical testing for these endpoints at the same nominal significance level of 5% without adjustment, as long as the tests occurred in the pre-defined sequential order, and given that all null hypotheses tested for endpoints with a higher rank (including the primary endpoint) were rejected. No confirmatory testing was done for this fourth key secondary endpoint, as the statistical test was not significant for the proportion of subjects with refractory disease (i.e., the third key secondary endpoint). |
| Other | Number of Days of Plasma Exchange | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | The number of days of PE during the overall study drug treatment period, including the number of days of PE during the open-label study drug treatment period. Data were analyzed according to the initial treatment allocation (both before and after switch to open-label caplacizumab). |
| Other | Total Volume of Plasma Exchange | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | The total volume of PE during the overall study drug treatment period, including the total volume of PE during the open-label study drug treatment period. Data were analyzed according to the initial treatment allocation (both before and after switch to open-label caplacizumab). |
| Other | Number of Days in Intensive Care Unit | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | The number of days in intensive care unit (ICU) during the overall study drug treatment period, including the number of days in ICU during the open-label study drug treatment period. Data were analyzed according to the initial treatment allocation (both before and after switch to open-label caplacizumab). |
| Other | Number of Days in Hospital | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | The number of days in hospital during the overall study drug treatment period, including the number of days in hospital during the open-label study drug treatment period. Data were analyzed according to the initial treatment allocation (both before and after switch to open-label caplacizumab). |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time to Platelet Count Response | Only data from the DB daily PE period (median = 5 days) up to the cut-off were used. The cut-off point was defined by whichever occured first: 1) 45 days of daily PE after start of study drug, 2) stop of daily PE, 3) stop of study drug (median = 34 days) | Intent-to-treat (ITT) population (for the respective study period, i.e., DB treatment period) | days | MEDIAN |
| SECONDARY | Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | The study drug treatment period, a median (min, max) of 36 (2, 82) days. For both treatment groups, only events that occurred prior to a switch to open-label caplacizumab were evaluated for this analysis. | ITT Population (for the respective study period, i.e., overall treatment period) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | The overall study period (covers both the overall treatment period and the follow-up period), a median (min, max) of 65 (2, 110) days. | ITT Population (for the respective study period, i.e., overall study period) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number and Percentage of Subjects With Refractory Disease | The study drug treatment period, a median (min, max) of 36 (2, 82) days. | ITT Population (for the respective study period, i.e., overall treatment period) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to Normalization of Organ Damage Marker Levels | Overall study period, a median (min, max) of 65 (2, 110) days. For both treatment groups, normalizations occurring during the open-label period were not evaluated in this analysis. | ITT Population (for the respective study period, i.e., overall study period) with biomarker level data available | days | MEDIAN |
| OTHER_PRE_SPECIFIED | Number of Days of Plasma Exchange | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | ITT Population (for the respective study period, i.e., overall treatment period) | days | MEAN |
| OTHER_PRE_SPECIFIED | Total Volume of Plasma Exchange | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | ITT Population (for the respective study period, i.e., overall treatment period) | liter(s) | MEAN |
| OTHER_PRE_SPECIFIED | Number of Days in Intensive Care Unit | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | ITT Population (for the respective study period, i.e., overall treatment period) | days | MEAN |
| OTHER_PRE_SPECIFIED | Number of Days in Hospital | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. | ITT Population (for the respective study period, i.e., overall treatment period) | days | MEAN |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time to Platelet Count Response | Caplacizumab | 2.69 | 95% Confidence Interval 1.89 to 2.83 | days | |
| Time to Platelet Count Response | Placebo | 2.88 | 95% Confidence Interval 2.68 to 3.56 | days | |
| Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | Caplacizumab | 9 | Participants | ||
| Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | Placebo | 36 | Participants | ||
| Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | Caplacizumab | 9 | Participants | ||
| Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | Placebo | 28 | Participants | ||
| Number and Percentage of Subjects With Refractory Disease | Caplacizumab | 0 | Participants | ||
| Number and Percentage of Subjects With Refractory Disease | Placebo | 3 | Participants | ||
| Time to Normalization of Organ Damage Marker Levels | Caplacizumab | 2.86 | 95% Confidence Interval 1.93 to 3.86 | days | |
| Time to Normalization of Organ Damage Marker Levels | Placebo | 3.36 | 95% Confidence Interval 1.88 to 7.71 | days |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow20
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Treatment Period + Follow-up | STARTED | Caplacizumab | 72 | |
| Overall Treatment Period + Follow-up | STARTED | Placebo | 73 | |
| Overall Treatment Period + Follow-up | Received Study Drug | Caplacizumab | 71 | |
| Overall Treatment Period + Follow-up | Received Study Drug | Placebo | 73 | |
| Overall Treatment Period + Follow-up | COMPLETED | Caplacizumab | 58 | |
| Overall Treatment Period + Follow-up | COMPLETED | Placebo | 50 | |
| Overall Treatment Period + Follow-up | NOT COMPLETED | Caplacizumab | 14 | |
| Overall Treatment Period + Follow-up | NOT COMPLETED | Placebo | 23 | |
| Double-blind (DB) Treatment Period | STARTED | Caplacizumab | 71 | |
| Double-blind (DB) Treatment Period | STARTED | Placebo | 73 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 participant flowAdverse events321
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 | Catheter site hemorrhage | General disorders | Double-blind Caplacizumab | 5 | 71 | 6 |
| other | Catheter site hemorrhage | General disorders | Double-blind Placebo | 5 | 73 | 5 |
| other | Catheter site hemorrhage | General disorders | Open-label Caplacizumab | 8 | 28 | 10 |
| other | Fatigue | General disorders | Double-blind Caplacizumab | 10 | 71 | 10 |
| other | Fatigue | General disorders | Double-blind Placebo | 6 | 73 | 6 |
| other | Fatigue | General disorders | Open-label Caplacizumab | 2 | 28 | 2 |
| other | Pyrexia | General disorders | Double-blind Caplacizumab | 10 | 71 | 12 |
| other | Pyrexia | General disorders | Double-blind Placebo | 6 | 73 | 6 |
| other | Pyrexia | General disorders | Open-label Caplacizumab | 1 | 28 | 1 |
| other | Oedema peripheral | General disorders | Double-blind Caplacizumab | 4 | 71 | 4 |
1 to 10 of 321Source ClinicalTrials.gov
See all 321 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Double-blind Caplacizumab | 1 | 71 |
| Total, all-cause mortality | Double-blind Placebo | 3 | 73 |
| Total, all-cause mortality | Open-label Caplacizumab | 0 | 28 |
| Total, other adverse events | Double-blind Caplacizumab | 67 | 71 |
| Total, other adverse events | Double-blind Placebo | 65 | 73 |
| Total, other adverse events | Open-label Caplacizumab | 25 | 28 |
| Total, serious adverse events | Double-blind Caplacizumab | 28 | 71 |
| Total, serious adverse events | Double-blind Placebo | 39 | 73 |
| Total, serious adverse events | Open-label Caplacizumab | 7 | 28 |
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Caplacizumab vs Placebo: Number and Percentage of Subjects With Refractory Disease | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
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/NCT02553317
GET https://conductscience.org/api/trials/v1/NCT02553317/events?page=2