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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-001098-42 | EudraCT Number |
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The study was a Phase III, double-blind, placebo-controlled, randomized study to evaluate the efficacy of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Caplacizumab | Experimental | Caplacizumab 10 mg once daily |
|
| Placebo | Placebo Comparator | Placebo once daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Caplacizumab | Biological |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time to Platelet Count Response | 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. | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | 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). | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Days of Plasma Exchange | 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). | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Ablynx NV | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator Site | Birmingham | Alabama | 35249 | United States | ||
| Investigator Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33881463 | Derived | 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. | |
| 31691462 |
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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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Of the 149 subjects screened, 4 were screen failures and 145 were randomly assigned to treatment (Intent-to-treat [ITT] population). All, except 1 subject who withdrew consent, received study drug and were included in the safety population and in the modified ITT (mITT) population.
A total of 145 subjects was randomized at 55 sites located in Europe (34 sites; 91 subjects), Asia (4 sites, 6 subjects), Australia (3 sites, 3 subjects), and North America (14 sites; 45 subjects). Consent was obtained from the first subject on 19 November 2015; the last subject completed the final visit on 16 August 2017.
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| ID | Title | Description |
|---|---|---|
| FG000 | Caplacizumab | Caplacizumab 10 mg once daily Caplacizumab:
|
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Treatment Period + Follow-up |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 20, 2016 | Feb 25, 2019 |
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|
|
| Placebo | Biological |
|
|
|
| Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | 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). | 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 Refractory Disease | 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). | The study drug treatment period, a median (min, max) of 36 (2, 82) days. |
| Time to Normalization of Organ Damage Marker Levels | 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). | 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. |
| Total Volume of Plasma Exchange | 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). | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
| Number of Days in Intensive Care Unit | 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). | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
| Number of Days in Hospital | 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). | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
| Los Angeles |
| California |
| 90033 |
| United States |
| Investigator Site | Atlanta | Georgia | 30322 | United States |
| Investigator Site | Boston | Massachusetts | 02114 | United States |
| Investigator Site | St Louis | Missouri | 63110 | United States |
| Investigator Site | Rochester | New York | 14642 | United States |
| Investigator Site | Rochester | New York | 55905 | United States |
| Investigator Site | Valhalla | New York | 10595 | United States |
| Investigator Site | Chapel Hill | North Carolina | 27599 | United States |
| Investigator Site | Durham | North Carolina | 27710 | United States |
| Investigator Site | Winston-Salem | North Carolina | 27157 | United States |
| Investigator SIte | Cleveland | Ohio | 44195 | United States |
| Investigator Site | Columbus | Ohio | 43210 | United States |
| Investigator Site | Oklahoma City | Oklahoma | 73104 | United States |
| Investigator Site | Pittsburgh | Pennsylvania | 15213 | United States |
| Investigator Site | Charleston | South Carolina | 29425 | United States |
| Investigator Site | Greenville | South Carolina | 27834 | United States |
| Investigator Site | Houston | Texas | 77030 | United States |
| Investigator Site | Salt Lake City | Utah | 84112 | United States |
| Investigator Site | Brisbane | Australia |
| Investigator Site 1 | Melbourne | Australia |
| Investigator Site 2 | Melbourne | Australia |
| Investigator Site 3 | Melbourne | Australia |
| Investigator Site 4 | Melbourne | Australia |
| Investigator Site 5 | Melbourne | Australia |
| Investigator Site | Perth | Australia |
| Investigator Site 1 | Sydney | Australia |
| Investigator Site 2 | Sydney | Australia |
| Investigator Site | Vienna | Austria |
| Investigator Site | Antwerp | Belgium |
| Investigator Site | Brussels | Belgium |
| Investigator Site | Haine-Saint-Paul | Belgium |
| Investigator Site | La Louvière | Belgium |
| Investigator Site | Leuven | Belgium |
| Invesigator Site | Montreal | Quebec | Canada |
| Investigator Site | London | Canada |
| Investigator Site | Toronto | Canada |
| Investigator Site | Brno | Czechia |
| Investigator Site | Hradec Králové | Czechia |
| Investigator Site | Olomouc | Czechia |
| Investigator Site | Ostrava-Poruba | Czechia |
| Investigator Site | Caen | France |
| Investigator Site | Lille | France |
| Investigator Site | Marseille | France |
| Investigator Site | Nantes | France |
| Investigator Site 1 | Paris | France |
| Investigator Site 2 | Paris | France |
| Investigator Site 3 | Paris | France |
| Investigator Site 4 | Paris | France |
| Investigator site 5 | Paris | France |
| Investigator Site | Rouen | France |
| Investigator Site | Salouël | France |
| Investigator site | Cologne | Germany |
| Investigator site 1 | Dresden | Germany |
| Investigator site 2 | Dresden | Germany |
| Investigator site | Erlangen | Germany |
| Investigator site | Göppingen | Germany |
| Investigator site | Kiel | Germany |
| Investigator site | Leipzig | Germany |
| Investigator site | Würzburg | Germany |
| Investigator Site 1 | Budapest | Hungary |
| Investigator Site 2 | Budapest | Hungary |
| Investigator Site | Debrecen | Hungary |
| Investigator Site 1 | Be’er Ya‘aqov | Israel |
| Investigator Site 2 | Be’er Ya‘aqov | Israel |
| Investigator Site | Haifa | Israel |
| Investigator Site 1 | Jerusalem | Israel |
| Investigator Site 2 | Jerusalem | Israel |
| Investigator Site | Nahariya | Israel |
| Investigator Site | Petah Tikva | Israel |
| Investigator Site | Tel Aviv | Israel |
| Investigator Site | Catania | Italy |
| Investigator Site 1 | Milan | Italy |
| Investigator Site 2 | Milan | Italy |
| Investigator Site | Pesaro | Italy |
| Investigator Site | Rome | Italy |
| Investigator Site | Vicenza | Italy |
| Investigator Site | Amersfoort | Netherlands |
| Investigator Site | Leiden | Netherlands |
| Investigator Site | Rotterdam | Netherlands |
| Investigator Site | Veldhoven | Netherlands |
| Investigator Site 1 | Barcelona | Spain |
| Investigator Site 2 | Barcelona | Spain |
| Investigator Site | Madrid | Spain |
| Investigator Site | Seville | Spain |
| Investigator Site 1 | Valencia | Spain |
| Investigator Site 2 | Valencia | Spain |
| Investigator site 3 | Valencia | Spain |
| Investigator Site | Bern | Switzerland |
| Investigator Site | Zurich | Switzerland |
| Investigator Site | Ankara | Turkey (Türkiye) |
| Investigator Site 1 | Denizli | Turkey (Türkiye) |
| Investigator Site 2 | Denizli | Turkey (Türkiye) |
| Investigator site 3 | Denizli | Turkey (Türkiye) |
| Investigator Site | Istanbul | Turkey (Türkiye) |
| Investigator Site | Kayseri | Turkey (Türkiye) |
| Investigator Site | Trabzon | Turkey (Türkiye) |
| Investigator Site | Bristol | United Kingdom |
| Investigator Site | Liverpool | United Kingdom |
| Investigator Site 1 | London | United Kingdom |
| Investigator Site 2 | London | United Kingdom |
| Knoebl P, Cataland S, Peyvandi F, Coppo P, Scully M, Kremer Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Minkue Mi Edou J, De Winter H, Callewaert F. Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study. J Thromb Haemost. 2020 Feb;18(2):479-484. doi: 10.1111/jth.14679. Epub 2019 Dec 9. |
| 30625070 | Derived | Scully M, Cataland SR, Peyvandi F, Coppo P, Knobl P, Kremer Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Callewaert F, Biswas D, De Winter H, Zeldin RK; HERCULES Investigators. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019 Jan 24;380(4):335-346. doi: 10.1056/NEJMoa1806311. Epub 2019 Jan 9. |
| FG001 | Placebo | Placebo once daily Placebo:
|
| Received Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Double-blind (DB) Treatment Period |
|
| Open-label (OL) Treatment Period |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Caplacizumab | Caplacizumab 10 mg once daily Caplacizumab:
|
| BG001 | Placebo | Placebo once daily Placebo:
|
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Time to Platelet Count Response | 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. | Intent-to-treat (ITT) population (for the respective study period, i.e., DB treatment period) | Posted | Median | 95% Confidence Interval | days | 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) |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period | 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). | ITT Population (for the respective study period, i.e., overall treatment period) | Posted | Count of Participants | Participants | 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. |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period | 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). | ITT Population (for the respective study period, i.e., overall study period) | Posted | Count of Participants | Participants | The overall study period (covers both the overall treatment period and the follow-up period), a median (min, max) of 65 (2, 110) days. |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percentage of Subjects With Refractory Disease | 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). | ITT Population (for the respective study period, i.e., overall treatment period) | Posted | Count of Participants | Participants | The study drug treatment period, a median (min, max) of 36 (2, 82) days. |
|
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| Secondary | Time to Normalization of Organ Damage Marker Levels | 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). | ITT Population (for the respective study period, i.e., overall study period) with biomarker level data available | Posted | Median | 95% Confidence Interval | days | 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. |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Days of Plasma Exchange | 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). | ITT Population (for the respective study period, i.e., overall treatment period) | Posted | Mean | Standard Error | days | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Total Volume of Plasma Exchange | 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). | ITT Population (for the respective study period, i.e., overall treatment period) | Posted | Mean | Standard Error | liter(s) | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
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| Other Pre-specified | Number of Days in Intensive Care Unit | 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). | ITT Population (for the respective study period, i.e., overall treatment period) | Posted | Mean | Standard Error | days | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Days in Hospital | 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). | ITT Population (for the respective study period, i.e., overall treatment period) | Posted | Mean | Standard Error | days | Overall study drug treatment period, a median (min, max) of 36 (2, 82) days. |
|
|
From time of first study drug administration until the subject's study completion/discontinuation date, a median (min, max) of 65 (2, 110) days.
Double-Blind Caplacizumab/Double-Blind placebo groups (subjects randomized to caplacizumab/placebo, respectively): adverse events (AEs) starting in DB or FU Periods for subjects with no OL Period. Only AEs starting in the DB Period for subjects with an OL Period.
OL Caplacizumab group (all subjects with OL Period): AEs starting in the OL or FU Periods.
The 3 subjects who died in the placebo group died during treatment and the subject who died in the caplacizumab group died during follow-up.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Double-blind Caplacizumab | Caplacizumab 10 mg once daily | 1 | 71 | 28 | 71 | 67 | 71 |
| EG001 | Double-blind Placebo | Placebo once daily | 3 | 73 | 39 | 73 | 65 | 73 |
| EG002 | Open-label Caplacizumab | In case an exacerbation during the 30-day treatment period or a relapse during the treatment extension period occurred (first exacerbation or relapse), subjects were to receive open label caplacizumab 10 mg daily together with re-initiation of daily PE and optimized immunosuppressive treatment. Caplacizumab treatment schedule and visit schedule were the same as for the initial study drug treatment period (covering daily PE [variable duration] and 30-day post-daily PE period) and the possible treatment extension period. | 0 | 28 | 7 | 28 | 25 | 28 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Systemic inflammatory response syndrome | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Upper gastrointestinal hemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gastric ulcer hemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gastrointestinal necrosis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hematemesis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Intestinal ischemia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Intestinal perforation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cerebral ischemia | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hemorrhagic transformation stroke | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hemiparesis | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash erythematosus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Thrombotic thrombocytopenic purpura | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 20.0 | Systematic Assessment | Verbatim term of serious adverse event (SAE) with fatal outcome: septic shock |
|
| Bacteremia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anaphylactic transfusion reaction | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Subarachnoid hemorrhage | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Jugular vein thrombosis | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arteriospasm coronary | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cardiac tamponade | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cardiogenic shock | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ventricular fibrillation | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hemorrhagic ovarian cyst | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gallbladder necrosis | Hepatobiliary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Serum sickness | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Catheter site hemorrhage | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Injection site hematoma | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hematochezia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lip hemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Paresthesia | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypokalemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyperglycemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypocalcemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hematoma | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Hematuria | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vaginal hemorrhage | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 20.0 | Systematic Assessment |
|
Publication of any results from this study will be according to the principles of the Declaration of Helsinki, in particular point 30, and will require prior review and written agreement of the Sponsor.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Ablynx | +32 (9) 262 00 00 | clinicaltrials@ablynx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 14, 2017 | Feb 25, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D011697 | Purpura, Thrombotic Thrombocytopenic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D019851 | Thrombophilia |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C585343 | caplacizumab |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| United States |
|
| Czechia |
|
| United Kingdom |
|
| Switzerland |
|
| Spain |
|
| Canada |
|
| Austria |
|
| Netherlands |
|
| Turkey |
|
| Belgium |
|
| Italy |
|
| Israel |
|
| Australia |
|
| France |
|
| Time to platelet count response was analyzed using a Cox proportional hazards regression model with time to platelet count response as dependent variable, and treatment group and GCS category as independent variables. The hazard (or platelet count normalization rate) ratio from the Cox model was reported along with 95% CI. | Hazard Ratio (HR) | 1.55 | 2-Sided | 95 | 1.095 | 2.195 | The HR was estimated from a Cox proportional Hazards regression model. | Superiority |
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Placebo once daily
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