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

NCT04904276 | Terminated DMC FDA Drug

• 1 other identifier: O-FOSTA-901
• Study Type: observational
• Target: 16
• Locations: 1 country
• Sites: 14

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).

Conditions

ITP; Immune Thrombocytopenia

Keywords

Immune Thrombocytopenia; ITP

Outcome Measures

Primary Outcomes (8)

• 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

Study Arms (2)

Initiating treatment with fostamatinib as second-line therapy

Drug: Fostamatinib

Treated with fostamatinib for at least 12 weeks as second-line therapy

Drug: Fostamatinib

Interventions

Fostamatinib DRUG

Fostamatinib disodium

Initiating treatment with fostamatinib as second-line therapy; Treated with fostamatinib for at least 12 weeks as second-line therapy

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Rigel Pharmaceuticals: lead

Study Sites (14)

Advanced Research

Tamarac, Florida, 33321, United States

Location

Simmons Cancer Institute at Southern Illinois University

Springfield, Illinois, 62702, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Saint Agnes Hospital

Baltimore, Maryland, 21229, United States

Location

The Center for Cancer and Blood Disorders

Bethesda, Maryland, 20817, United States

Location

Maryland Oncology Hematology, P.A

Clinton, Maryland, 20735, United States

Location

Maryland Oncology Hematology, P.A

Columbia, Maryland, 21044, United States

Location

Maryland Oncology Hematology, P.A

Rockville, Maryland, 20850, United States

Location

Maryland Oncology Hematology, P.A

Silver Spring, Maryland, 20904, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Washington University

St Louis, Missouri, 63130, United States

Location

Regional Cancer Care Associates, LLC

Little Silver, New Jersey, 07739, United States

Location

New York Medical College Westchester Medical Center

Valhalla, New York, 10532, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

fostamatinib

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 13, 2021
• First Posted: May 27, 2021
• Study Start: May 18, 2021
• Primary Completion: November 17, 2022
• Study Completion: November 17, 2022
• Last Updated: March 15, 2024

Record last verified: 2024-03

Locations

US (14)