A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
- Conditions
- Immune Thrombocytopenic Purpura
- Interventions
- Drug: Placebo
- Registration Number
- NCT02076412
- Lead Sponsor
- Rigel Pharmaceuticals
- Brief Summary
The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 74
- Clinical diagnosis of persistent/chronic ITP for at least 3 months
- Average platelet count< 30,000/µL (and none > 35,000 unless as a result of rescue therapy) from at least 3 qualifying counts
- Clinical diagnosis of autoimmune hemolytic anemia
- Uncontrolled or poorly controlled hypertension
- History of coagulopathy including prothrombotic conditions
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Fostamatinib Disodium Fostamatinib Disodium Fostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks. Placebo Placebo Placebo tablet PO bid (morning and evening) over the course of 24 weeks
- Primary Outcome Measures
Name Time Method Number of Participants With Stable Platelet Response of at Least 50,000/µL Baseline to Week 24 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
- Secondary Outcome Measures
Name Time Method Number of Participants With Platelet Count ≥ 50,000/µL at Week 12 Baseline to Week 12 Platelet Count ≥ 50,000/µL at Week 12
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24 Baseline to Week 24 Platelet Count ≥ 50,000/µL at Week 24
Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 12 Baseline to 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.
Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 24 Baseline to 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
Frequency and Severity of Bleeding According to the ITP Bleeding Score (IBLS) Baseline to Week 24 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.Frequency and Severity of Bleeding According to the World Health Organization (WHO) Bleeding Scale Baseline to Week 24 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.
Trial Locations
- Locations (35)
Haukeland University Hospital
🇳🇴Bergen, Norway
Medizinische Universitaet Wien / AKH Wien
🇦🇹Wien, AU, Austria
Spitalul Clinic Coltea, Hematologie
🇷🇴Bucuresti, Romania
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, Bulgaria
Sykehuset Østfold Fredrikstad
🇳🇴Fredrikstad, Norway
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdansk, Poland
Instytut Hematologii I Transfuzjologii
🇵🇱Warszawa, Poland
Hematology Oncology Associates of Rockland Division of Highland Medical PC
🇺🇸Nyack, New York, United States
Fakultni nemocnice Ostrava
🇨🇿Ostrava-Poruba, Czechia
Fakultni nemocnice Kralovske Vinohrady
🇨🇿Praha 10, Czechia
Charit Berlin - Campus Benjamin Franklin
🇩🇪Berlin, Germany
Szpital Uniwersytecki
🇵🇱Krakow, Poland
Hospital Universitari Vall D'Hebron
🇪🇸Barcelona, Spain
Hanusch-Krankenhaus Wiener Gebietskrankenkasse
🇦🇹Vienna, AU, Austria
Marien Hospital Duesseldorf
🇩🇪Duesseldorf, Germany
Universittsklinikum Essen
🇩🇪Essen, Germany
UMHAT Aleksandrovska, EAD, Clinic of Clinical Hematology
🇧🇬Sofia, Bulgaria
Werlhof Institut GmbH
🇩🇪Hannover, DE, Germany
LKH Feldkirch at LKH Rankweil
🇦🇹Rankweil, Austria
MHAT Hristo Botev, AD, Vratsa, First Internal Department
🇧🇬Vratsa, BG, Bulgaria
UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology
🇧🇬Pleven, Bulgaria
Universitaetsklinikum Giessen und Marburg (UKGM)
🇩🇪Giessen, DE, Germany
Hospital Kyjov
🇨🇿Kyjov, CZ, Czechia
Fakultni nemocnice Brno
🇨🇿Brno, CZ, Czechia
Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi
🇵🇱Lodz, Poland
Specjalistyczny Gabinet Lekarski
🇵🇱Lublin, Poland
Szpital Wojewodzki w Opolu
🇵🇱Opole, Poland
Wojewodzki Szpital Specjalistyczny im. J. Korczaka
🇵🇱Slupsk, Poland
Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw
🇵🇱Wroclaw, Poland
Spitalul Clinic Judetean de Urgenta Tirgu-Mures, Sectia Medicina Interna 1, Compartiment Hematologie
🇷🇴Targu Mures, Mures, Romania
Spitalul Clinic Colentina, Hematologie
🇷🇴Bucuresti, Romania
Hospital Universitariola Paz
🇪🇸Madrid, Spain
Hospital Universitari Germans Trias i Pujol
🇪🇸Barcelona, Spain
Hospital Universitari i Politécnic La Fe de Valencia
🇪🇸Valencia, Spain
Vseobecna fakultni nemocnice, Linterní Klinika, Klinika hematologie
🇨🇿Praha 2, Czechia