A Phase 3 Study of Fostamatinib Disodium in the Treatment Immune Thrombocytopenic Purpura
- Conditions
- Persistent/Chronic Immune Thrombocytopenic PurpuraMedDRA version: 16.1Level: LLTClassification code 10067644Term: Immune-mediated thrombocytopenic purpuraSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2013-005452-15-GB
- Lead Sponsor
- Rigel Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 75
1.Subject must be willing and able to give written informed consent by signing an Ethics Committee approved Informed Consent Form prior to undergoing any study-specific procedures.
2.Subject must have had a diagnosis of ITP for at least 3 months and no known aetiology for thrombocytopenia.
3.Subject’s platelet count averages < 30,000/µL (and none > 35,000 unless as a result of rescue therapy) from at least 3 qualifying counts within the preceding 3 months. At least 2 of the qualifying counts must have been taken during the screening period.
4.Must have previously received at least 1 typical regimen for the treatment of ITP.
5.Male or female at least 18 years of age.
6.Performance status on Karnofsky performance status scale (KPS) > 70
7.Subject’s concurrent treatment for ITP may consist of either glucocorticoids (< 20 mg prednisone equivalent per day), or azathioprine or danazol. The dose of the concurrent medication must have been stable for 14 days prior to baseline and must be expected to remain stable throughout the study. No other concurrent medications for ITP are permitted.
8.Subject’s other therapeutic agents for ITP have been discontinued in accordance with the washout periods
9.Female subject must be either post-menopausal for at least 1 year or surgically sterile; or if female of child-bearing potential, must not be pregnant or lactating and must agree to use an acceptable method of birth control throughout the duration of the trial and for 30 days following the last dose. Acceptable methods of birth control are defined as: hormonal contraception (pill, injection or implant) used consistently for at least 30 days prior to screening, an intrauterine device (IUD), a double-barrier method (ie, condom and spermicide, or condom and diaphragm), or complete abstinence.
10.In the Investigator’s opinion, the subject has the ability to understand the nature of the study and any hazards of participation and to communicate satisfactorily with the Investigator.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15
1.Subject with ITP associated with lymphoma, chronic lymphocytic leukaemia, viral infection, autoimmune disorders, thyroid disease, human immunodeficiency virus, or hepatitis or induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
2.Subject with autoimmune haemolytic anaemia.
3.Subject has a history of or active, clinically significant, respiratory, gastrointestinal (pancreatitis), renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorders that, in the Investigator’s opinion, could affect the conduct of the study or the absorption, metabolism or excretion of the study drug.
4.Subject has had any major cardiovascular event within the 6 months prior to randomisation, including but not limited to; myocardial infarction, unstable angina, cerebrovascular accident, pulmonary embolism, or New York Heart Association Class III or IV heart failure.
5.Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood pressure = 140 mm Hg, or diastolic blood pressure = 90 mm Hg, whether or not the subject is receiving anti-hypertensive treatment. Subjects may be rescreened if the blood pressure is successfully and promptly controlled (within 30 days) using conventional anti-hypertensive therapy to achieve optimal blood pressure control (< 140/90 mmHg).
6.Subject has a history of coagulopathy including prothrombotic conditions such as Factor V Leiden, APC resistance, AT-III deficiency and lupus anticoagulant, or arterial or deep venous thrombosis within 6 months prior to randomisation.
7.In subjects with deep venous thrombosis greater than 6 months prior to randomisation, anticoagulants must have been discontinued for at least 30 days and subsequent D dimer must be within normal limits for the site’s local laboratory.
8.Subject has a bleeding assessment score of Grade 2 at any site by the ITP Bleeding Scale (IBLS).
9.Subject has 1 or more of the following laboratory abnormalities: leukocyte count < 2,500/µL, neutrophil count of < 1,500/µL, lymphocyte count < 750/µL, Hgb < 10 g/L without ongoing transfusion support, or transaminase levels (ALT, AST) > 1.5x ULN, total bilirubin > 2.0 mg/dL, or estimated glomerular filtration rate (eGFR) < 30 mL/min at the time of screening and/or baseline (Day 1).
10.Subject has a significant infection, or an acute infection such as influenza, or is known to have an active inflammatory process at the time of screening and/or baseline (Day 1).
11.Subject has acute gastrointestinal symptoms at the time of screening and/or baseline (eg, nausea, vomiting, diarrhea, abdominal pain).
12.Subject has increased the dose of, or added, prescription drugs within the 2 weeks prior to Day 1, unless agent is agreed to be not clinically relevant by both the Investigator and Sponsor.
13.Subject has had positive results for HIV, HBV, or HCV by standard serologic tests.
14.Subject has received any blood or blood products within the 2 weeks prior to randomization. (IVIg or anti-rho (D) immunoglobulin (anti-D) are allowed if used for rescue therapy, unless platelet count is > 30,000/µL at the time of randomization.)
15.Subject is currently enrolled in an investigational drug or device study or has used an investigational drug or device within 30 days or 5 half-lives (whichever is longer) of Day 1.
16.Subject has a history of alcohol or substance abuse that, in the judgment of the Investigator, may impair or risk th
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study is to establish the efficacy of fostamatinib as compared with placebo in achieving a stable platelet response in subjects with persistent/chronic ITP.;Secondary Objective: Secondary objectives include assessment of the incidence of bleeding complications in subjects receiving fostamatinib as compared with placebo, and assessment of the overall safety and tolerability of fostamatinib versus placebo in subjects with persistent/chronic ITP.;Primary end point(s): The primary efficacy endpoint is achievement of a 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.;Timepoint(s) of evaluation of this end point: visits between Weeks 14-24
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The secondary efficacy endpoints of this study are as follows:<br>• Achievement of a platelet response (a platelet count of at least 50,000/µL) at Weeks 12 and 24.<br>• 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 Weeks 12 and 24.<br>• Frequency and severity of bleeding according to the ITP Bleeding Score (IBLS) and World Health Organization (WHO) bleeding scale over the 24-week study period.;Timepoint(s) of evaluation of this end point: • at Weeks 12 and 24 <br>• over the 24-week study period