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

NCT05904093 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2026-05-05

No results posted yet for this study

Summary

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

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

Interventions

DRUG

Fostamatinib

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).

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

  • Swee Lay Thein, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2024-12-18
Primary Completion
2026-05-14
Completion
2026-05-14
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05904093 on ClinicalTrials.gov