Fostamatinib for Stable Sickle Cell Disease

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Recruiting Phase 1 Interventional Study
Sickle Cell Disorders Sickle Cell Disease Hemoglobin S Hb-SS Disease Disease Sickle Cell Anemia
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
18 – 65
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Basic Science
Participants needed
35 (estimated)
Sponsor
National Heart, Lung, and Blood Institute (NHLBI) · NIH
Who this trial is looking for

This trial is looking for adults aged 18 to 65 with stable sickle cell disease. Participants will take a medication called fostamatinib at home for up to 6 weeks and have regular check-ups to monitor their health and any side effects.

Are You a Good Fit for This Trial?
Must be taking Hydroxyurea
Must not be taking L-glutamineCrizanlizumab
Rules you out NeutropeniaPsychiatric disorderCholestatic hepatitis

You may be able to join if

  • I am between 18 and 65 years old.
  • I have sickle cell disease confirmed by specific blood tests.
  • I haven't had a blood transfusion in the last 60 days.
  • My organ functions are stable based on blood tests.
  • I am willing to follow the study procedures.
  • I have provided signed consent to participate.

You may not be able to join if

  • I have had a pain crisis that needed treatment in the last 14 days.
  • I have a significant medical condition that could affect my participation.
  • I am currently pregnant or breastfeeding.
  • I have had a prior bone marrow or stem cell transplant.
  • I am taking strong medications that affect specific liver enzymes.
  • I have a history of certain serious infections like HIV or hepatitis.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial
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 …
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: Subjects will enroll onto the study and undergo screening. Subjects who do not meet any of the following criteria during screening will not receive the study intervention but will be counted toward study accrual. Screen failures may be rescreened at a later time. In order to b…
Contacts

Jordan B Branch

(301) 221-3820

jordan.branch@nih.gov

CONTACT

Swee Lay Thein, M.D.

(301) 435-2345

sweelay.thein@nih.gov

CONTACT