Clinical trial NCT04904276: Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy
Official title: Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy (FORTE)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: ITP
Immune Thrombocytopenia
Keywords: Immune Thrombocytopenia
ITP
Lead sponsor: Rigel Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-05-27
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: This two-cohort study will enroll approximately 45 patients diagnosed with ITP. Cohort 1 will enroll approximately 30 ITP patients who will begin treatment with fostamatinib as second-line therapy. Cohort 2 will enroll approximately 15 patients who have a record of at least 12 weeks of fostamatinib treatment as second-line therapy, with the intent to continue, at time of enrollment.

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).

Eligibility criteria: Inclusion Criteria:

1. Willing and able to provide written informed consent
2. Diagnosis of ITP in adult patients (≥ 18 years of age) with an insufficient response to steroids ± immunoglobulins and requiring a change in therapy
3. For Cohort 1, intent to initiate fostamatinib at enrollment as change in therapy
4. 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

Exclusion Criteria:

1. Any prior ITP therapy/treatment other than steroids ± immunoglobulins (e.g. rituximab, thrombopoietin receptor agonists (TPO), splenectomy)
2. Participation in any interventional study during the course of this study
3. Lack of historical platelet counts, at time of fostamatinib initiation for those entering Cohort 2
4. Pregnant and/or nursing

Primary outcomes:
measure: Fostamatinib dosing
timeFrame: 12-month observation period
measure: Platelet counts over time during the course of fostamatinib therapy
timeFrame: 12-month observation period
measure: Use of concomitant medications related to ITP
timeFrame: 12-month observation period
measure: Use of ITP Rescue Medication
timeFrame: 12-month observation period
measure: Safety of fostamatinib
timeFrame: 12-month observation period
measure: ITP-PAQ Quality of life measure
timeFrame: 12-month observation period
measure: Treatment satisfaction measure (MSQ)
timeFrame: 12-month observation period
measure: SF-36 Quality of life measure
timeFrame: 12-month observation period
Secondary outcomes:
Other outcomes:
Locations:
city: Tamarac
country: United States
facility: Advanced Research
state: Florida
city: Springfield
country: United States
facility: Simmons Cancer Institute at Southern Illinois University
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Saint Agnes Hospital
state: Maryland
city: Bethesda
country: United States
facility: The Center for Cancer and Blood Disorders
state: Maryland
city: Clinton
country: United States
facility: Maryland Oncology Hematology, P.A
state: Maryland
city: Columbia
country: United States
facility: Maryland Oncology Hematology, P.A
state: Maryland
city: Rockville
country: United States
facility: Maryland Oncology Hematology, P.A
state: Maryland
city: Silver Spring
country: United States
facility: Maryland Oncology Hematology, P.A
state: Maryland
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Little Silver
country: United States
facility: Regional Cancer Care Associates, LLC
state: New Jersey
city: Valhalla
country: United States
facility: New York Medical College Westchester Medical Center
state: New York
city: Valhalla
country: United States
facility: New York Medical College
state: New York
Study officials:
References: