A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
- Conditions
- Immune Thrombocytopenic Purpura
- Interventions
- Drug: Placebo
- Registration Number
- NCT02076399
- 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
- 76
- 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 Subjects begin with Fostamatinib Disodium tablet 100 mg PO bid and increase to 150 mg big after week 4 based on platelet count and tolerability. 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 (Count of ≥50,000/µL on at Least 4 of the Last 6 Scheduled Visits Between Weeks 14 and 24) From Week 14 to Week 24 A stable platelet response by Week 24 defined as a platelet count of at least 50,000/μL on at least 4 of the last 6 scheduled visits between Weeks 14 and 24
- Secondary Outcome Measures
Name Time Method Number of Participants With Platelet Count ≥ 50,000/µL at Week 12 Week 12 Platelet Count ≥ 50,000/µL at Week 12
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24 Week 24 Platelet Count ≥ 50,000/µL at Week 24
Mean of the ITP Bleeding Score (IBLS) Assessed over the 24-week study period 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.Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 12. Baseline to Week 12 Number of subjects with baseline platelet count \<15,000/μL who showed platelet count increase to ≥30,000/μL and ≥20,000/μL from baseline count at Week 12.
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 24. Baseline to Week 24 Number of subjects with baseline platelet count \<15,000/μL who showed platelet count increase to ≥30,000/μL and ≥20,000/μL from baseline count at Week 24.
Mean of World Health Organization (WHO) Bleeding Scale Assessed over the 24-week study period 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 (52)
University of Southern California
🇺🇸Los Angeles, California, United States
Colchester General Hospital
🇬🇧Colchester, Essex, United Kingdom
OspedaleCivile-ClinicaEmatologica/PUGD
🇮🇹Udine, Italy
Concord Repatriation General Hospital
🇦🇺Concord, New South Wales, Australia
Westmead Hospital
🇦🇺Westmead, New South Wales, Australia
Kent & Canterbury Hospital
🇬🇧Kent, Canterbury, United Kingdom
St George Hospital
🇦🇺Kogarah, New South Wales, Australia
Perth Blood Institute
🇦🇺Nedlands, Western Australia, Australia
Liverpool Hospital
🇦🇺Liverpool, New South Wales, Australia
Herlev Hospital University of Copenhagen, Department of Hematology L124
🇩🇰Herlev, DK, Denmark
Ematologia - Padigilione 8, Policinico S. Orsola Malpighi, Azienda Ospedaliero Universitaria di Bologna
🇮🇹Bologna, Italy
St. Michael's Hospital
🇨🇦Toronto, Ontario, Canada
Dept. of Haematology, Odense University Hospital
🇩🇰Odense C, DK, Denmark
Hematological department, Roskilde Hospital
🇩🇰Roskilde, DK, Denmark
Semmelweis University 1st
🇭🇺Budapest, Hungary
Universitã Federico II di Napoli
🇮🇹Napoli, Italy
St. James's Hospital
🇬🇧Leeds, United Kingdom
Royal Victoria Infirmary
🇬🇧Newcastle-upon-Tyne, United Kingdom
Ospedale San Raffaele S.r.l. Dipartimento di Oncoematologia
🇮🇹Milano, Italy
Royal London Hospital
🇬🇧London, United Kingdom
Manchester Royal Infirmary
🇬🇧Manchester, United Kingdom
University College Hospital
🇬🇧London, United Kingdom
Aarhus University Hospital
🇩🇰Aalborg, Denmark
James Paget University Hospital
🇬🇧Norfolk, United Kingdom
Arizona Oncology Associates
🇺🇸Tucson, Arizona, United States
Lakeland Regional Cancer Center
🇺🇸Lakeland, Florida, United States
Center for Cancer and Blood Disorders
🇺🇸Bethesda, Maryland, United States
Weill Cornell Medical College/New York Presbyterian Hospital
🇺🇸New York, New York, United States
Pitt County Memorial Hospital
🇺🇸Greenville, North Carolina, United States
Signal Point Clinical Research Center LLC
🇺🇸Middletown, Ohio, United States
University of Utah Health Sciences Center
🇺🇸Salt Lake City, Utah, United States
Prince of Wales Hospital
🇦🇺Randwick, New South Wales, Australia
Hamilton Health Sciences Corporation
🇨🇦Hamilton, Ontario, Canada
Ottawa Hospital Research Institute
🇨🇦Ottawa, Ontario, Canada
Pecs University
🇭🇺Pecs, Hungary
HAGA ziekenhuis
🇳🇱Haag, NL, Netherlands
ULSS 6 Vicenza-Ospedale San Bortolo di Vicenza
🇮🇹Vicenza, Italy
Leicester Royal Infirmary
🇬🇧Leicester, United Kingdom
Oxford University Hospital
🇬🇧Oxford, United Kingdom
University Hospital of North Staffordshire
🇬🇧Stoke-on-Trent, United Kingdom
Sandwell General Hospital
🇬🇧West Bromwich, United Kingdom
University of Virginia
🇺🇸Charlottesville, Virginia, United States
Bill Hefner VA Medical Center
🇺🇸Salisbury, North Carolina, United States
Bleeding & Clotting Disorders Institute
🇺🇸Peoria, Illinois, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Horizon Oncology Research, Inc
🇺🇸Lafayette, Indiana, United States
University of Debrecen
🇭🇺Debrecen, Hungary
Dept of Haematology, The Alfred Hospital and Monash Medical Centre
🇦🇺Melbourne, Victoria, Australia
Frankston Hospital
🇦🇺Frankston, Victoria, Australia
Launceston General Hospital
🇦🇺Launceston, Tasmania, Australia
Hammersmith Hospital, Catherine Lewis Centre
🇬🇧London, United Kingdom
Royal Liverpool University Hospital
🇬🇧Liverpool, UK, United Kingdom