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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03764618
Recruitment Status : Completed
First Posted : December 5, 2018
Results First Posted : May 25, 2023
Last Update Posted : May 25, 2023
Sponsor:
Information provided by (Responsible Party):
Rigel Pharmaceuticals

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

Condition or disease Intervention/treatment Phase
Warm Antibody Autoimmune Hemolytic Anemia Drug: Fostamatinib disodium Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Fostamatinib
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.

Other Names:
  • R935788
  • Fostamatinib
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.
 Drug: Placebo
Placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. A Hemoglobin Response by Week 24 [ Time Frame: 24 weeks ]
    Proportion of subjects with a hemoglobin response by Week 24.

  2. 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.

  3. Change in Hemoglobin From Baseline to End of Treatment [ Time Frame: 24 weeks ]
    Change in mean hemoglobin from baseline to end of treatment.

  4. 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.

  5. 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].


Eligibility Criteria
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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
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.
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
Rigel Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Rigel Pharmaceuticals:
Study Protocol and Statistical Analysis Plan  [PDF] February 4, 2022

More Information
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Responsible Party: Rigel Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03764618    
Other Study ID Numbers: C-935788-057
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rigel Pharmaceuticals:
wAIHA
Warm Antibody Autoimmune Hemolytic Anemia
Warm Autoimmune Hemolytic Anemia
Additional relevant MeSH terms:
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Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
 Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases