A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
Condition: Warm Antibody Autoimmune Hemolytic Anemia
MeSH terms: Anemia; Anemia, Hemolytic; Anemia, Hemolytic, Autoimmune; Hemolysis
Intervention: Fostamatinib disodium; Placebo
Intervention description: Fostamatinib (100mg PO bid or 150 mg PO bid)
The dose may be reduced at any time to a dose as low as fostamatinib 100 mg PO qd or matching placebo if dose limiting adverse events are observed.; Placebo
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 90.0
Sex: All
Minimum age: 18 Years
Maximum age: 100 Years
Healthy volunteers: No
Keywords: wAIHA; Warm Antibody Autoimmune Hemolytic Anemia; Warm Autoimmune Hemolytic Anemia

Summary:

      The primary objective of this study is to assess the efficacy of fostamatinib in subjects
      with warm antibody autoimmune hemolytic anemia (wAIHA).
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subject must have a diagnosis of primary or secondary warm Antibody Autoimmune
             Hemolytic Anemia (wAIHA) as documented by a positive direct antiglobulin test (DAT)
             specific for anti-IgG or anti-IgA.

          2. Have failed or not tolerated at least one prior wAIHA treatment regimen, including
             steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, MMF, danazol,
             vincristine, ESA or splenectomy (folate, iron or other supplements do not fulfill this
             criterion).

          3. Have haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.

          4. At screening, subject's hemoglobin level must be ≤9 g/dL OR if hemoglobin value >9
             g/dL and <10 g/dL, subject must be on an allowed wAIHA treatment AND the subject must
             have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue,
             shortness of breath, chest pain).

          5. Karnofsky performance status (KPS) ≥70.

          6. Subject's concurrent treatment for wAIHA may consist of no more than two of any of the
             following agents: azathioprine, steroids, ESAs, mycophenolate mofetil, dapsone or
             danazol at a stable dose

        Exclusion Criteria:

          1. Subject with other types of Antibody Autoimmune Hemolytic Anemia (AIHA) (e.g., cold
             antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold
             hemoglobinuria).

          2. Subject has AIHA secondary to autoimmune disease, including systemic lupus
             erythematosus (SLE), or lymphoid malignancy if the underlying disease is not stable or
             is not well-controlled on current therapy, per investigator medical judgement.

          3. Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood
             pressure ≥135 mmHg or diastolic blood pressure ≥85 mmHg, whether or not the subject is
             receiving anti-hypertensive treatment.

          4. Subject has one or more of the following laboratory abnormalities at screening:
             neutrophil count of <1,000/μL or platelet count of <30,000/μL, unless due to Evans
             syndrome; transaminase levels (i.e., alanine aminotransferase [ALT] or aspartate
             aminotransferase [AST]) >1.5 x ULN.

          5. Has documented active hepatitis B or hepatitis C infection or HIV infection.