Fostamatinib disodium in the treatment of wAIHA
Research type
Research Study
Full title
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia.
IRAS ID
262506
Contact name
Nichola Cooper
Contact email
Sponsor organisation
Rigel Pharmaceuticals, INC.
Eudract number
2018-004774-97
Duration of Study in the UK
1 years, 5 months, 11 days
Research summary
Research Summary
This is a Phase 3 (confirmatory) multi-center, randomized, double-blind, placebo-controlled, parallel group study to investigate the efficacy of 24 weeks of treatment with fostamatinib vs. placebo in increasing haemoglobin in subjects with warm antibody autoimmune haemolytic anemia (wAIHA), who have failed at least one prior treatment regimen.
The sponsor is aiming to conduct this study at 7 hospitals in the UK in adult patients with wAIHA. Approximately 80 subjects will be recruited globally and they will be randomised in a 1:1 ratio to 2 parallel treatment arms; 100mg fostamatinib taken orally twice a day or matching placebo. Randomisation will be stratified by concomitant steroid use at baseline and severity of anemia at screening. The subjects will self-administer the study drug in the morning an evening throughout a 24-week treatment period.
If the subjects tolerate the initial 100mg twice daily dose (or matching placebo) well, their dose will increase to 150mg fostamatinib twice daily (or matching placebo) starting at week 4.
Dose adjustments are allowed if dose-limiting adverse events are observed. The dose may be reduced at any time to a dose as low as 100mg fostamatinib (or matching placebo) once daily.Subjects are allowed to continue their stable concurrent steroid therapy and one other wAIHA therapy as per the protocol, while participating in this study.
During the study, the subjects are expected to visit the clinic about 15 times.
Blood samples will be collected at every visit for safety and efficacy assessments and haemoglobin levels to determine if a dose adjustment is required.
Rescue medication is allowed on the study if medically necessary (Hgb of >1.5 g/dL from baseline OR new or worsening symptoms of anemia).
Steroid use will be tapered if a durable response is observed at week 12.
Subjects who complete this study are encouraged to enrol in an open-label study with fostamatinib.
Summary of Results
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REC name
London - Surrey Borders Research Ethics Committee
REC reference
19/LO/1153
Date of REC Opinion
16 Sep 2019
REC opinion
Further Information Favourable Opinion