Actively Recruiting
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-05-05
25
Participants Needed
1
Research Sites
73 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves. People with SCD are also at increased risk of forming blood clots in the veins and lungs, but the standard treatments for these clots can cause increased bleeding in people with SCD. Better treatments are needed. Objective: To test a drug (fostamatinib) in people with SCD. Eligibility: People aged 18 to 65 with SCD. Design: Participants will have 6 clinic visits over 12 weeks. Each visit will be 2 to 3 hours. Participants will be screened. They will have a physical exam with blood tests. They will tell the researchers about the medications they take. Fostamatinib is a tablet taken by mouth. Participants will take the drug at home, twice a day, for up to 6 weeks. Participants will have a clinic visit every 2 weeks while they are taking the drug. At each visit they will have a physical exam with blood tests. They will talk about any side effects the drug may be causing. If they are tolerating the drug well after the first 2 weeks, they may begin taking a higher dose. Participants will have a final visit 4 weeks after they stop taking the drug. They will have a physical exam and blood tests; they will be checked for any side effects of the drug.
CONDITIONS
Official Title
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide signed informed consent before any study procedures
- Be between 18 and 65 years old
- Have a clear diagnosis of sickle cell anemia (HbSS or HbSBeta0) confirmed by hemoglobin electrophoresis at least 60 days after any transfusion
- No blood transfusion in the 60 days before consent or no Hb A detected on hemoglobin analysis
- Have adequate organ function with AST and ALT less than or equal to 1.5 times the normal limit, neutrophil count at least 1.5 x 10^9/L, hemoglobin at least 7 g/dL, and platelet count at least 100 x 10^9/L
- If taking hydroxyurea, be on a stable dose for at least 3 months
- For women who can have children, have a negative pregnancy test during screening
- Agree to use two effective forms of contraception or be abstinent during the study and for 28 days after treatment
- Willing to follow all study procedures for the full study duration
You will not qualify if you...
- Had a pain crisis requiring parenteral treatment within 14 days before consent
- Have medical conditions that increase risk or affect study data, including neutropenia (except benign or hydroxyurea-related), history of posterior reversible encephalopathy syndrome, uncontrolled hypertension unless controlled for over 90 days, active hepatitis B or C infection, drug-induced cholestatic hepatitis, any primary cancer, active HIV infection with low CD4 count and high viral load, or psychiatric disorders affecting study cooperation
- Currently enrolled in another therapeutic clinical trial with ongoing treatment
- Used newly approved sickle cell therapies (L-glutamine, voxelotor, or crizanlizumab) within 7 days before consent
- Had a bone marrow or stem cell transplant
- Currently pregnant or breastfeeding
- Currently taking strong inhibitors or inducers of CYP3A4/5 that have not been stopped for the required time before consent
- Receiving erythropoiesis stimulating agents currently or recently
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
J
Jordan B Branch
CONTACT
S
Swee Lay Thein, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
BASIC_SCIENCE
Number of Arms
1
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