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Completed

NCT Number: NCT03764618

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

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

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Key information

Conditions

Warm Antibody Autoimmune Hemolytic Anemia

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Concord Repatriation General Hospital, Sydney, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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.

Treatment and study plan

Fostamatinib disodium

Drug

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

Drug

Placebo

Primary outcomes

  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 outcomes

  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].

Sponsors and collaborators

Lead sponsor

Rigel Pharmaceuticals

Industry

Registry information

Official study title

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Important dates

Study start
2019
Primary completion
2022
Study completion
2022
First posted
Dec 5, 2018
Registry last updated
May 25, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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