A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA

• Register: ClinicalTrials.gov
• Last refreshed on: 5 June 2023
• Main ID: NCT03764618
• Date of registration: 03/12/2018
• Prospective Registration: Yes
• Primary sponsor: Rigel Pharmaceuticals
• Public title: A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
• Scientific title: A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
• Date of first enrolment: April 24, 2019
• Target sample size: 90
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03764618
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belarus, Belgium, Bulgaria, Canada, Czechia, Denmark, France, Georgia, Germany, Hungary, Italy, Netherlands, Norway, Romania, Russian Federation, Serbia, Spain, Ukraine, United Kingdom, United States

Key inclusion & exclusion 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 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.



Age minimum: 18 Years
Age maximum: 100 Years
Gender: All

Health Condition(s) or Problem(s) studied

Warm Antibody Autoimmune Hemolytic Anemia

Intervention(s)

Drug: Fostamatinib disodium

Drug: Placebo

Primary Outcome(s)

Durable Hemoglobin Response [Time Frame: 24 Weeks]

Secondary Outcome(s)

Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F) [Time Frame: 24 weeks]

A Hemoglobin Response by Week 24 [Time Frame: 24 weeks]

Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4 [Time Frame: 24 weeks]

Change From Baseline in Hemoglobin Level of 2 g/dL or Greater [Time Frame: 24 weeks]

Change in Hemoglobin From Baseline to End of Treatment [Time Frame: 24 weeks]

Secondary ID(s)

C-935788-057

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/05/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03764618