Open label extension study of Fostamatinib in patients with wAIHA
Research type
Research Study
Full title
A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
IRAS ID
274860
Contact name
Nichola Cooper
Contact email
Eudract number
2019-001882-34
Duration of Study in the UK
2 years, 8 months, 20 days
Research summary
This is a phase 3 multi-centre, open-label extension study to evaluate the long-term safety and efficacy of fostatmatinib in patients with warm Antibody Autoimmune hemolytic anemia (wAIHA) and who have completed 24 weeks of participation in study C-935788-057.
At entry into this extension study, subjects who at any time during the C-935788-057 study achieved a haemoglobin response (defined as a haemoglobin level of >10 g/dL and ≥2 g/dL higher than the baseline (Day 1) value if, during the previous 4 weeks, the steroid dose was maintained at the baseline level and rescue medication was not administered) 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 (orally twice a day) and starting at Week 4, 150 mg PO bid if subjects have adequately tolerated the study drug.
All subjects will remain blinded to their treatment assignment in study C-935788-057 (active or placebo).
Eligible participants will continue their study visits at the same NHS sites where they attended their visits for the C-935788-057 protocol. They will attend 25 visits for the C-935788-058 study.Participants will undergo physical examinations and vital sign measurements, serum pregnancy tests if required, complete questionnaires at the baseline visit (which is also the last visit of the C-935788-057 protocol) and have blood samples taken for safety and efficacy assessments.
Participants may continue a maximum of 2 concurrent wAIHA therapies while on the study (the study doctor will discuss the allowed medications for wAIHA with you).
Temporary steroid dose increases may be undertaken as rescue therapy during the study.
Study drug does levels can be reduced to as little as 100 mg once a day in the event of adverse events.
The trial will end when the last subject has completed their last study visit.
Lay summary of study results: The achievement of durable response rates was similar between treatment groups, and the average duration of response suggested that the fostamatinib benefit seemed durable. The duration of responses is well maintained in the extension study (C-935788-058) and have similar rate as C-935788-057 fostamatinib group. Similar results were observed in several efficacy endpoints in the extension study treatment groups compared to the fostamatinib treatment group in the parent study (C-935788-057). The results indicate that efficacy in wAIHA subjects from fostamatinib appears to be maintained in the extension study.
The long-term safety profile for fostamatinib observed in subjects with wAIHA support and expand the safety profile established in the primary Phase 3 studies in ITP, in prior studies in subjects with RA, and in the parent Phase 3 study C-935788-057 in the same subjects with wAIHA (24 weeks exposure). There were no new safety findings with up to 106 weeks exposure to fostamatinib as high as 150 mg po bid. Overall, the results of this Phase 3 open-label extension study support the long-term safety of fostamatinib administered at 100-150 mg BID in subjects with wAIHA.
Lay summary of study results: The achievement of durable response rates was similar between treatment groups, and the average duration of response suggested that the fostamatinib benefit seemed durable. The duration of responses is well maintained in the extension study (C-935788-058) and have similar rate as C-935788-057 fostamatinib group. Similar results were observed in several efficacy endpoints in the extension study treatment groups compared to the fostamatinib treatment group in the parent study (C-935788-057). The results indicate that efficacy in wAIHA subjects from fostamatinib appears to be maintained in the extension study.
The long-term safety profile for fostamatinib observed in subjects with wAIHA support and expand the safety profile established in the primary Phase 3 studies in ITP, in prior studies in subjects with RA, and in the parent Phase 3 study C-935788-057 in the same subjects with wAIHA (24 weeks exposure). There were no new safety findings with up to 106 weeks exposure to fostamatinib as high as 150 mg po bid. Overall, the results of this Phase 3 open-label extension study support the long-term safety of fostamatinib administered at 100-150 mg BID in subjects with wAIHA.
REC name
London - City & East Research Ethics Committee
REC reference
20/LO/0021
Date of REC Opinion
20 Jan 2020
REC opinion
Favourable Opinion