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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-005453-76 | EudraCT Number |
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The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fostamatinib Disodium | Experimental | Fostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks. |
|
| Placebo | Other | Placebo tablet PO bid (morning and evening) over the course of 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fostamatinib Disodium | Drug | Fostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Stable Platelet Response of at Least 50,000/µL | Stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the 6 visits between Weeks 14-24 | Baseline to Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Platelet Count ≥ 50,000/µL at Week 12 | Platelet Count ≥ 50,000/µL at Week 12 | Baseline to Week 12 |
| Number of Participants With Platelet Count ≥ 50,000/µL at Week 24 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rigel Pharmaceuticals, Inc. | Rigel Pharmaceuticals,Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hematology Oncology Associates of Rockland Division of Highland Medical PC | Nyack | New York | 10960 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33995988 | Derived | Cooper N, Altomare I, Thomas MR, Nicolson PLR, Watson SP, Markovtsov V, Todd LK, Masuda E, Bussel JB. Assessment of thrombotic risk during long-term treatment of immune thrombocytopenia with fostamatinib. Ther Adv Hematol. 2021 Apr 30;12:20406207211010875. doi: 10.1177/20406207211010875. eCollection 2021. |
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74 patients were enrolled from January 2015 to August 2016
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| ID | Title | Description |
|---|---|---|
| FG000 | Fostamatinib Recipient | Fostamatinib (100 mg PO bid or 150 mg PO bid) |
| FG001 | Placebo Recipient | Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Placebo | Drug | Placebo tablet PO bid (morning and evening) |
|
Platelet Count ≥ 50,000/µL at Week 24
| Baseline to Week 24 |
| Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 12 | Among subjects with a baseline platelet count < 15,000/μL, achievement of a count ≥ 30,000/μL and at least 20,000/μL above baseline at Week 12. | Baseline to Week 12 |
| Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 24 | Among subjects with a baseline platelet count < 15,000/μL, achievement of a count ≥ 30,000/μL and at least 20,000/μL above baseline at Week 24 | Baseline to Week 24 |
| Frequency and Severity of Bleeding According to the ITP Bleeding Score (IBLS) | The ITP Bleeding Scale (IBLS) is an immune thrombocytopenic purpura (ITP)-specific bleeding score used to analyze the correlation of clinical and laboratory platelet variables with bleeding. The IBLS comprises of 11 grades from 0 (none) to 2 (marked bleeding) by history over the previous week or by exam; 2 being worse. These 11 grades include: skin by physical exam, oral by physical exam, skin by history, oral by history, epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage. After each grade is scored, the mean value for all 11 grades is calculated (lowest score being 0 and highest score being 2) for each subject visit. LOCF method was used to impute any missing data. The mean of the IBLS scores across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint. | Baseline to Week 24 |
| Frequency and Severity of Bleeding According to the World Health Organization (WHO) Bleeding Scale | The World Health Organization (WHO) bleeding scale is a standardized grading scale created to measure the severity of bleeding. The scale is a clinical investigator-assessed five-point scale with a score range starting at the lowest 0=No bleeding, 1 = Petechiae, 2=Mild blood loss, 3=Gross blood loss, to the worse 4=Debilitating blood loss. The WHO bleeding scale is scored by history over the previous-week or by exam. After each grade is scored, the mean value is calculated (lowest score being 0 [no bleeding] to the highest score being 4 [debilitating blood loss]) for each visit. LOCF method was used to impute any missing data. The mean of the WHO bleeding scale across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint. | Baseline to Week 24 |
| Medizinische Universitaet Wien / AKH Wien |
| Vienna |
| AU |
| 1090 |
| Austria |
| Hanusch-Krankenhaus Wiener Gebietskrankenkasse | Vienna | AU | 1140 | Austria |
| LKH Feldkirch at LKH Rankweil | Rankweil | 6830 | Austria |
| Specialized Hospital for Active Treatment of Hematology Diseases, EAD, Sofia, Department of Chemotherapy, Hemotherapy and Blood Inherited Diseases to Clinic of Clinical Hematology; | Sofia | BG | 1756 | Bulgaria |
| MHAT Hristo Botev, AD, Vratsa, First Internal Department | Vratsa | BG | 3000 | Bulgaria |
| UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology | Pleven | 5800 | Bulgaria |
| UMHAT Aleksandrovska, EAD, Clinic of Clinical Hematology | Sofia | 1431 | Bulgaria |
| Fakultni nemocnice Brno | Brno | CZ | 625 00 | Czechia |
| Hospital Kyjov | Kyjov | CZ | 69733 | Czechia |
| Fakultni nemocnice Ostrava | Ostrava-Poruba | 708 52 | Czechia |
| Fakultni nemocnice Kralovske Vinohrady | Prague | 100 34 | Czechia |
| Vseobecna fakultni nemocnice, Linterní Klinika, Klinika hematologie | Prague | 128 08 | Czechia |
| Universitaetsklinikum Giessen und Marburg (UKGM) | Giessen | DE | 35392 | Germany |
| Werlhof Institut GmbH | Hanover | DE | 30159 | Germany |
| Charit Berlin - Campus Benjamin Franklin | Berlin | 12203 | Germany |
| Marien Hospital Duesseldorf | Düsseldorf | 40479 | Germany |
| Universittsklinikum Essen | Essen | 45147 | Germany |
| Haukeland University Hospital | Bergen | 5021 | Norway |
| Sykehuset Østfold Fredrikstad | Fredrikstad | 1606 | Norway |
| Uniwersyteckie Centrum Kliniczne | Gdansk | 80-952 | Poland |
| Szpital Uniwersytecki | Krakow | 31-501 | Poland |
| Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi | Lodz | 93-510 | Poland |
| Specjalistyczny Gabinet Lekarski | Lublin | 20-081 | Poland |
| Szpital Wojewodzki w Opolu | Opole | 45-064 | Poland |
| Wojewodzki Szpital Specjalistyczny im. J. Korczaka | Słupsk | 76-200 | Poland |
| Instytut Hematologii I Transfuzjologii | Warsaw | 02-776 | Poland |
| Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw | Wroclaw | 50-367 | Poland |
| Spitalul Clinic Judetean de Urgenta Tirgu-Mures, Sectia Medicina Interna 1, Compartiment Hematologie | Târgu Mureş | Mureș County | 540136 | Romania |
| Spitalul Clinic Colentina, Hematologie | Bucharest | 020125 | Romania |
| Spitalul Clinic Coltea, Hematologie | Bucharest | 030171 | Romania |
| Hospital Universitari Vall D'Hebron | Barcelona | 08035 | Spain |
| Hospital Universitari Germans Trias i Pujol | Barcelona | 08916 | Spain |
| Hospital Universitariola Paz | Madrid | 28046 | Spain |
| Hospital Universitari i Politécnic La Fe de Valencia | Valencia | 46026 | Spain |
| COMPLETED |
|
| NOT COMPLETED |
|
ITT Population
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Fostamatinib Recipient | Fostamatinib (100 mg PO bid or 150 mg PO bid) |
| BG001 | Placebo Recipient | 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, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | 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 | Number of Participants With Stable Platelet Response of at Least 50,000/µL | Stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the 6 visits between Weeks 14-24 | Posted | Count of Participants | Participants | Baseline to Week 24 |
|
|
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Platelet Count ≥ 50,000/µL at Week 12 | Platelet Count ≥ 50,000/µL at Week 12 | Posted | Count of Participants | Participants | Baseline to Week 12 |
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Platelet Count ≥ 50,000/µL at Week 24 | Platelet Count ≥ 50,000/µL at Week 24 | Posted | Count of Participants | Participants | Baseline to Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 12 | Among subjects with a baseline platelet count < 15,000/μL, achievement of a count ≥ 30,000/μL and at least 20,000/μL above baseline at Week 12. | Posted | Count of Participants | Participants | Baseline to Week 12 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 24 | Among subjects with a baseline platelet count < 15,000/μL, achievement of a count ≥ 30,000/μL and at least 20,000/μL above baseline at Week 24 | Posted | Count of Participants | Participants | Baseline to Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Frequency and Severity of Bleeding According to the ITP Bleeding Score (IBLS) | The ITP Bleeding Scale (IBLS) is an immune thrombocytopenic purpura (ITP)-specific bleeding score used to analyze the correlation of clinical and laboratory platelet variables with bleeding. The IBLS comprises of 11 grades from 0 (none) to 2 (marked bleeding) by history over the previous week or by exam; 2 being worse. These 11 grades include: skin by physical exam, oral by physical exam, skin by history, oral by history, epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage. After each grade is scored, the mean value for all 11 grades is calculated (lowest score being 0 and highest score being 2) for each subject visit. LOCF method was used to impute any missing data. The mean of the IBLS scores across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint. | Posted | Mean | Standard Deviation | scores on a scale | Baseline to Week 24 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Frequency and Severity of Bleeding According to the World Health Organization (WHO) Bleeding Scale | The World Health Organization (WHO) bleeding scale is a standardized grading scale created to measure the severity of bleeding. The scale is a clinical investigator-assessed five-point scale with a score range starting at the lowest 0=No bleeding, 1 = Petechiae, 2=Mild blood loss, 3=Gross blood loss, to the worse 4=Debilitating blood loss. The WHO bleeding scale is scored by history over the previous-week or by exam. After each grade is scored, the mean value is calculated (lowest score being 0 [no bleeding] to the highest score being 4 [debilitating blood loss]) for each visit. LOCF method was used to impute any missing data. The mean of the WHO bleeding scale across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint. | Posted | Mean | Standard Deviation | scores on a scale | Baseline to Week 24 |
|
|
24 weeks
One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Fostamatinib Recipient | Fostamatinib (100 mg PO bid or 150 mg PO bid) | 1 | 51 | 5 | 51 | 36 | 51 |
| EG001 | Placebo Recipient | Placebo | 0 | 23 | 6 | 23 | 18 | 23 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (18.1) | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA (18.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (18.1) | Systematic Assessment |
| |
| Muscle rupture | Injury, poisoning and procedural complications | MedDRA (18.1) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (18.1) | Systematic Assessment |
| |
| Plasma cell myeloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (18.1) | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA (18.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (18.1) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (18.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (18.1) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Anne-Marie Duliege, MD | Rigel | 6506241100 | 6506241100 | clinicaltrials@rigel.com |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| 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 |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
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| ID | Term |
|---|---|
| C523665 | fostamatinib |
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| Male |
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| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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|
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| Units |
|---|
| Counts |
|---|
| Participants |
|
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| Participants |
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