Phase 3
N=90
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
Warm Antibody Autoimmune Hemolytic Anemia
Bottom Line
View on ClinicalTrials.gov: NCT03764618 ↗Enrolled (actual)
90
Serious AEs
35.6%
Results posted
May 2023
Primary outcome: Primary: Durable Hemoglobin Response — 16; 12 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Fostamatinib disodium (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Rigel Pharmaceuticals
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Durable Hemoglobin Response |
16; 12 | — |
| SECONDARY A Hemoglobin Response by Week 24 |
21; 16 | — |
| SECONDARY Change From Baseline in Hemoglobin Level of 2 g/dL or Greater |
22; 16 | — |
| SECONDARY Change in Hemoglobin From Baseline to End of Treatment |
1.99; 1.99 | — |
| SECONDARY Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4 |
18; 18 | — |
| SECONDARY Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F) |
4.1; 2.2 | — |
Summary
The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
Eligibility Criteria
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 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 1.5 x ULN.
- Has documented active hepatitis B or hepatitis C infection or HIV infection.
Data sourced from ClinicalTrials.gov (NCT03764618). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.