A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
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|ClinicalTrials.gov Identifier: NCT03764618|
Recruitment Status : Completed
First Posted : December 5, 2018
Results First Posted : May 25, 2023
Last Update Posted : May 25, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Warm Antibody Autoimmune Hemolytic Anemia||Drug: Fostamatinib disodium Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||90 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia|
|Actual Study Start Date :||April 24, 2019|
|Actual Primary Completion Date :||April 11, 2022|
|Actual Study Completion Date :||April 11, 2022|
Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.
Drug: Fostamatinib disodium
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 Comparator: Placebo
Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to placebo 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.
- Durable Hemoglobin Response [ Time Frame: 24 Weeks ]Proportion of subjects achieving a hemoglobin level ≥ 10 g/dL with an increase from Baseline in hemoglobin level of ≥ 2 g/dL on 3 consecutive available visits during the 24-week treatment period.
- A Hemoglobin Response by Week 24 [ Time Frame: 24 weeks ]Proportion of subjects with a hemoglobin response by Week 24.
- Change From Baseline in Hemoglobin Level of 2 g/dL or Greater [ Time Frame: 24 weeks ]Proportion of subjects with change from baseline in hemoglobin level of 2 g/dL or greater.
- Change in Hemoglobin From Baseline to End of Treatment [ Time Frame: 24 weeks ]Change in mean hemoglobin from baseline to end of treatment.
- Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4 [ Time Frame: 24 weeks ]Proportion of subjects free of rescue AIHA regimens used after Week 4.
- Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F) [ Time Frame: 24 weeks ]Change from Baseline to Week 24 in Functional Assessment of Chronic Illness Therapy - Fatigue scale (FACIT-F). The FACIT-F scale is a short, 13-item, easy to administer tool that measures an individual's level of fatigue during their usual daily activities over the past week. Each item is rated using a 5-point Likert-type scale. The scale range is 0 to 52, with 0 being the worst possible score and 52 being the best possible score indicating no fatigue. Total Score = [Sum of item scores] x [N of items in subscale] ÷ [N of items answered].
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|Ages Eligible for Study:||18 Years to 100 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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).
- Have haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.
- 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).
- Karnofsky performance status (KPS) ≥70.
- 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
- 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).
- 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.
- 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.
- 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.
- Has documented active hepatitis B or hepatitis C infection or HIV infection.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03764618
Documents provided by Rigel Pharmaceuticals:
|Responsible Party:||Rigel Pharmaceuticals|
|Other Study ID Numbers:||
|First Posted:||December 5, 2018 Key Record Dates|
|Results First Posted:||May 25, 2023|
|Last Update Posted:||May 25, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Warm Antibody Autoimmune Hemolytic Anemia
Warm Autoimmune Hemolytic Anemia
Anemia, Hemolytic, Autoimmune
Immune System Diseases