Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy

Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy (FORTE)

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).

Study Overview

• Status: Terminated
• Conditions: Immune Thrombocytopenia; ITP
• Intervention / Treatment: Drug: Fostamatinib

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).

• Study Type: Observational
• Enrollment (Actual): 16

Contacts and Locations

Study Locations

• United States
  • Florida
    • Tamarac, Florida, United States, 33321
      • Advanced Research
  • Illinois
    • Springfield, Illinois, United States, 62702
      • Simmons Cancer Institute at Southern Illinois University
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21229
      • Saint Agnes Hospital
    • Bethesda, Maryland, United States, 20817
      • The Center for Cancer and Blood Disorders
    • Clinton, Maryland, United States, 20735
      • Maryland Oncology Hematology, P.A
    • Columbia, Maryland, United States, 21044
      • Maryland Oncology Hematology, P.A
    • Rockville, Maryland, United States, 20850
      • Maryland Oncology Hematology, P.A
    • Silver Spring, Maryland, United States, 20904
      • Maryland Oncology Hematology, P.A
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Boston Medical Center
  • Missouri
    • Saint Louis, Missouri, United States, 63130
      • Washington University
  • New Jersey
    • Little Silver, New Jersey, United States, 07739
      • Regional Cancer Care Associates, LLC
  • New York
    • Valhalla, New York, United States, 10595
      • New York Medical College
    • Valhalla, New York, United States, 10532
      • New York Medical College Westchester Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 98 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Initiating treatment with fostamatinib as second-line therapy	Drug: Fostamatinib; Fostamatinib disodium
Treated with fostamatinib for at least 12 weeks as second-line therapy	Drug: Fostamatinib; Fostamatinib disodium

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fostamatinib dosing	Changes in fostamatinib dosing	12-month observation period
Platelet counts over time during the course of fostamatinib therapy	Platelet count and change from baseline in platelet count over time.	12-month observation period
Use of concomitant medications related to ITP	Use of concomitant medications related to ITP during the study.	12-month observation period
Use of ITP Rescue Medication	The number of patients with ITP rescue medication use at any time during the study.	12-month observation period
Safety of fostamatinib	Evaluate continued safety of fostamatinib in ITP patients through adverse events	12-month observation period
ITP-PAQ Quality of life measure	Use of the score from questionnaire to assess disease-specific quality of life in adults with ITP.	12-month observation period
Treatment satisfaction measure (MSQ)		12-month observation period
SF-36 Quality of life measure	Scale of functional health and well-being from the patient's point of view.	12-month observation period

Collaborators and Investigators

• Sponsor: Rigel Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): May 18, 2021
• Primary Completion (Actual): November 17, 2022
• Study Completion (Actual): November 17, 2022

Study Registration Dates

• First Submitted: April 13, 2021
• First Submitted That Met QC Criteria: May 26, 2021
• First Posted (Actual): May 27, 2021

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 12, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Immune Thrombocytopenia; ITP
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Cytopenia; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: O-FOSTA-901

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No