Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy
- Registration Number
- NCT04904276
- Lead Sponsor
- Rigel Pharmaceuticals
- Brief Summary
Evaluate patient characteristics, treatment patterns, clinical outcomes and safety of fostamatinib as second-line therapy in real-world clinical practice, for the treatment of ITP in adult patients who have an insufficient response to prior therapy (steroids ± immunoglobulins).
- Detailed Description
Evaluate patient characteristics, treatment patterns, clinical outcomes and safety of fostamatinib as second-line therapy in real-world clinical practice, for the treatment of ITP in adult patients who have an insufficient response to prior therapy (steroids ± immunoglobulins).
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 16
- Willing and able to provide written informed consent
- Diagnosis of ITP in adult patients (≥ 18 years of age) with an insufficient response to steroids ± immunoglobulins and requiring a change in therapy
- For Cohort 1, intent to initiate fostamatinib at enrollment as change in therapy
- For Cohort 2, currently undergoing treatment with fostamatinib as second line therapy for at least 12 weeks, with the intent to continue, at the time of enrollment
- Any prior ITP therapy/treatment other than steroids ± immunoglobulins (e.g. rituximab, thrombopoietin receptor agonists (TPO), splenectomy)
- Participation in any interventional study during the course of this study
- Lack of historical platelet counts, at time of fostamatinib initiation for those entering Cohort 2
- Pregnant and/or nursing
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Initiating treatment with fostamatinib as second-line therapy Fostamatinib - Treated with fostamatinib for at least 12 weeks as second-line therapy Fostamatinib -
- Primary Outcome Measures
Name Time Method Fostamatinib dosing 12-month observation period Changes in fostamatinib dosing
Use of ITP Rescue Medication 12-month observation period The number of patients with ITP rescue medication use at any time during the study.
Platelet counts over time during the course of fostamatinib therapy 12-month observation period Platelet count and change from baseline in platelet count over time.
Safety of fostamatinib 12-month observation period Evaluate continued safety of fostamatinib in ITP patients through adverse events
ITP-PAQ Quality of life measure 12-month observation period Use of the score from questionnaire to assess disease-specific quality of life in adults with ITP.
Treatment satisfaction measure (MSQ) 12-month observation period SF-36 Quality of life measure 12-month observation period Scale of functional health and well-being from the patient's point of view.
Use of concomitant medications related to ITP 12-month observation period Use of concomitant medications related to ITP during the study.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (11)
Saint Agnes Hospital
🇺🇸Baltimore, Maryland, United States
New York Medical College
🇺🇸Valhalla, New York, United States
Boston Medical Center
🇺🇸Boston, Massachusetts, United States
Advanced Research
🇺🇸Tamarac, Florida, United States
Maryland Oncology Hematology, P.A
🇺🇸Silver Spring, Maryland, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
Simmons Cancer Institute at Southern Illinois University
🇺🇸Springfield, Illinois, United States
The Center for Cancer and Blood Disorders
🇺🇸Bethesda, Maryland, United States
Regional Cancer Care Associates, LLC
🇺🇸Little Silver, New Jersey, United States
New York Medical College Westchester Medical Center
🇺🇸Valhalla, New York, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States