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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study ID
NCT05904093
Phase
PHASE1
Status
Recruiting
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Conditions

  • Disease Sickle Cell Anemia
  • Hb-SS Disease
  • Hemoglobin Beta Thalassemia Disease
  • Hemoglobin S
  • Sickle Cell Disease
  • Sickle Cell Disorders

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Accepted

Interventions

  • Fostamatinib — DRUG
    Fostamatinib will be administered orally, at a dose of 100 mg twice a day for 14 days and if tolerated, will be escalated to a dose of 150 mg, orally, twice a day for 28 days (total 42 days).

Study Details

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.

Key Dates

Start date
Dec 18, 2024
Status verified
Jan 2026
Primary completion
May 14, 2027
Completion
May 14, 2027

Study Design

Enrollment
25 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE

Arms

  • Experimental: Fostamatinib in participants with Sickle Cell Disease
    Participants with Sickle Cell Disease will receive Fostamatinib which will be administered orally, at a dose of 100 mg twice a day for 14 days and if tolerated, will be escalated to a dose of 150 mg, taken orally, twice a day for 28 days (total 42 days).

Primary Outcome Measure

The number of type, incidence, severity and relationship to study treatment of adverse events and serious adverse events [ Time Frame: Baseline (Day 0) to End of Study (Day 70) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
NIH Clinical Center Office of Patient Recruitment (OPR)
[email protected]
800-411-1222

Find similar trials in Bethesda, MD

By condition
Sickle cell disease
By research site
National Institutes of Health Clinical Center· Bethesda, MD

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