A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Overview

The primary objectives of this study are: – To establish the efficacy of fostamatinib in placebo crossover subjects with warm antibody autoimmune hemolytic anemia (wAIHA) – To determine the ability to maintain a response in subjects receiving fostamatinib for wAIHA

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2021

Interventions

  • 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.

Arms, Groups and Cohorts

  • Experimental: Fostamatinib
    • Subjects who at any time during the C-935788-057 study achieved a hemoglobin response in the absence of rescue in the previous 4 weeks or a steroid dose greater than baseline will continue at their current dose (100mg or 150 mg) and regimen in the extension study. All other subjects who enter the extension study will initially receive fostamatinib 100 mg PO bid. Starting at Week 4, the initial fostamatinib dose of 100 mg PO bid will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug, based on the investigator’s judgment.

Clinical Trial Outcome Measures

Primary Measures

  • Durable Response
    • Time Frame: 104 weeks
    • A primary efficacy endpoint is the proportion of subjects who achieve a durable hemoglobin response.
  • Durable Response and Hemoglobin Response
    • Time Frame: 104 weeks
    • The proportion of any subjects who achieve a durable response and have a hemoglobin response at 48 weeks of exposure

Secondary Measures

  • Hemoglobin Response by Week 24
    • Time Frame: 24 weeks
    • Proportion of placebo crossover subjects who have achieved a hemoglobin response by week 24 of this study
  • Partial Hemoglobin Response
    • Time Frame: 24 Weeks
    • The proportion of any subjects who achieve a partial hemoglobin response
  • Median Hemoglobin Value at Week 24
    • Time Frame: 24 weeks
    • Median hemoglobin value at week 24 of this study, for placebo crossover subjects
  • Median change from baseline in hemoglobin at week 24
    • Time Frame: 24 Weeks
    • Median change from baseline in hemoglobin at week 24 of this study for placebo crossover subjects
  • Durable Response and Maintain a Hemoglobin Response at 24 and 36 Weeks
    • Time Frame: 36 Weeks
    • The proportion of any subjects who achieve a durable response and maintain a hemoglobin response at 24 and 36 weeks of exposure

Participating in This Clinical Trial

Inclusion Criteria

1. Subject must be willing and able to give written informed consent by signing an IRB approved Informed Consent Form prior to undergoing any study-specific procedures. 2. Subject must have completed all 24 weeks of participation in the study C-935788-057. Exclusion Criteria:

1. Any subject who discontinued participation in Study C-935788-057 prior to Week 24.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 100 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Rigel Pharmaceuticals
  • Provider of Information About this Clinical Study
    • Sponsor

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