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

• ClinicalTrials.gov Identifier: NCT04138927
• Recruitment Status: Enrolling by invitation
• First Posted: October 25, 2019
• Last Update Posted: March 17, 2022
• Sponsor: Rigel Pharmaceuticals
• Information provided by (Responsible Party): Rigel Pharmaceuticals

Tracking Information

• First Submitted Date: October 23, 2019
• First Posted Date: October 25, 2019
• Last Update Posted Date: March 17, 2022
• Actual Study Start Date: October 30, 2019
• Estimated Primary Completion Date: April 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 19, 2021)

• Adverse Events [ Time Frame: 104 weeks ]
  Incidence, frequency, seriousness, and severity of adverse events that occurred during the current study
• Blood Pressure [ Time Frame: 104 weeks ]
  Change from baseline in blood pressure over time
• Absolute Neutrophil Count (ANC) [ Time Frame: 104 weeks ]
  Change from baseline in absolute neutrophil count (ANC) over time

Original Primary Outcome Measures (submitted: October 23, 2019)

• 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

Current Secondary Outcome Measures (submitted: July 19, 2021)

• Achievement of Durable Hemoglobin Response [ Time Frame: 24 weeks ]
• Total Duration of Response [ Time Frame: During the Intervention period up to 104 weeks ]
• Corticosteroid dose [ Time Frame: During the Intervention period up to 104 weeks ]
  Net cumulative change from Baseline in corticosteroid dose

Original Secondary Outcome Measures (submitted: October 23, 2019)

• 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

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
• Official Title: A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Brief Summary

The primary objective of this study is:

• To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Warm Antibody Autoimmune Hemolytic Anemia

Intervention

Drug: Fostamatinib disodium

Fostamatinib is supplied in two (2) dosage strengths: 100 mg and 150 mg.

Other Names:

• R935788
• Fostamatinib

Study Arms

Experimental: Fostamatinib

Subjects who at any time during the C-935788-057 study achieved a hemoglobin response will continue at their dose and regimen from the Week 22 visit in the C-935788-057 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.

Intervention: Drug: Fostamatinib disodium

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: May 4, 2021): 90
• Original Estimated Enrollment (submitted: October 23, 2019): 80
• Estimated Study Completion Date: April 2024
• Estimated Primary Completion Date: April 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 100 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Austria, Belarus, Belgium, Bulgaria, Czechia, France, Georgia, Germany, Italy, Netherlands, Norway, Russian Federation, Serbia, Spain, Ukraine, United Kingdom, United States

Administrative Information

• NCT Number: NCT04138927
• Other Study ID Numbers: C-935788-058
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Rigel Pharmaceuticals
• Original Responsible Party: Same as current
• Current Study Sponsor: Rigel Pharmaceuticals
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Rigel Pharmaceuticals
• Verification Date: July 2021