Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects Wit… (NCT05904093) | Clinical Trial Compass
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
United States25 participantsStarted 2024-12-18
Plain-language 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.
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion criteria
✓. Have provided signed written informed consent prior to performing any study procedure, including screening procedures.
✓. Age between 18-65 years
✓. Unequivocal diagnosis of SCA (HbSS or HbSBeta\^0) confirmed by hemoglobin electrophoresis performed on patients at least 60 days after a blood transfusion if previously transfused.
✓. No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC)
✓. Have adequate organ function, as defined by:
✓. Serum aspartate aminotransferase (AST) \<=1.5 x Upper Limit of Normal (ULN) (unless the increased AST is assessed by the Investigator as due to hemolysis) and alanine aminotransferase (ALT) \<=1.5 x ULN.
✓. Absolute neutrophil count \>=1.5 x 10\^9/L.
✓. Hemoglobin \>= 7 g/dL
Exclusion criteria
✕
What they're measuring
1
The number of type, incidence, severity and relationship to study treatment of adverse events and serious adverse events
Timeframe: Baseline (Day 0) to End of Study (Day 70)
Trial details
NCT IDNCT05904093
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
. Pain crisis requiring parenteral treatment within 14 days of signing consent.
✕. Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:
✕. History of neutropenia (benign ethnic neutropenia and/or acquired neutropenia related to drug suppression by hydroxyurea and/or cyclic hematopoiesis are permitted).
✕. History of posterior reversible encephalopathy syndrome (PRES)
✕. History of poorly controlled hypertension (defined as systolic blood pressure \>=130 mmHg or average diastolic blood pressure \>=90 mmHg based on an average of 3 blood pressure readings despite adequate antihypertensive therapy) unless controlled for \>90 days prior to enrollment.
✕. Active viral infection as evidenced by testing positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody (ab) with signs of active hepatitis B or C virus infection. If the subject is positive for HCV Ab, a reverse transcriptase-polymerase chain reaction test will be conducted. Subjects with hepatitis C may be rescreened after receiving appropriate hepatitis C treatment.