Pilot Study of Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)
Condition: Purpura, Thrombocytopenic, Idiopathic
MeSH terms: Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
Intervention: Fostamatinib Disodium / R935788
Intervention description: R935788 tablets
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 18.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Adult Refractory Immune Thrombocytopenic Purpura (ITP)

Summary:

      The purpose of this study is to determine whether Fostamatinib Disodium is safe and effective
      in the treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients must be willing and able to give written informed consent by signing an
             IRB-approved Informed Consent Form prior to admission to this study.

          -  Patients may be male or female, between the ages of 18 75. Men, if sexually active,
             must agree to use at least one medically acceptable form of birth control. Women of
             childbearing potential must have a negative urine pregnancy test, and agree to use two
             independent methods of birth control, if sexually active.

          -  Patients must have a diagnosis of chronic refractory ITP for at least 3 months.
             Chronic refractory ITP is defined as:

               1. Platelet count < 30,000/mm3 consistently for 3 months (except for transient
                  nonsustained responses to various therapeutic regimens). There must be at least
                  three separate platelet counts (below 30,000/mm3) over this period, with at least
                  one extending back to three months or more prior to patient entry into the study.

               2. The following conditions will have been excluded either by history or appropriate
                  laboratory investigation: HIV infection (see below), lymphoproliferative
                  disorders, myelodysplasia, SLE, drug-induced or alloimmune thrombocytopenia, or
                  dysglobulinemias.

               3. The patient must have tried at least two typical regimens for the treatment of
                  ITP (George et al., Blood, 1996; Practice Guidelines, American Society of
                  Hematology). At least 50% of the enrolled patients will not be known to be
                  refractory to IVIg. Patients may or may not have been treated with IVIg in the
                  past.

          -  Subjects must test negative for HIV, HBV, and HCV by standard serologic tests within
             the previous six months.

        Exclusion Criteria:

          -  Patients who have a history or presence of substantial or clinically significant
             respiratory, gastrointestinal, renal, hepatic, neurological, cardiovascular,
             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 are excluded. Specifically
             excluded are lymphoma/chronic lymphocytic leukemia, hepatitis, or HIV associated with
             ITP.

          -  Patients who have a history of relevant drug hypersensitivity are excluded.

          -  Patients who have a history of substance abuse, drug addiction or alcoholism are
             excluded.

          -  Patients with the following laboratory abnormalities: a leukocyte count < 2,500/mm3, a
             neutrophil count of < 1,800/mm3, lymphocyte count < 750/mm3, Hgb < 10 g/L, or
             transaminase levels (ALT, AST) > 1.5xULN are excluded.