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Tests treatment safety and results for Sickle Cell Disease
Official title Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
ClinicalTrials.gov ID: NCT05904093
What this study is testing
What is Fostamatinib?
Fostamatinib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- Time commitment: about 12 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65. Healthy volunteers may be eligible.
You may be able to join if
- people will enroll onto the study and undergo screening. people who do not meet any of the following criteria during screening will not receive the...
- 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...
- No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC)
You likely can't join if
- An individual who meets any of the following criteria will be excluded from participation in this study:
- 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...
- History of neutropenia (benign ethnic neutropenia and/or acquired neutropenia related to drug suppression by hydroxyurea and/or cyclic hematopoiesis...
- 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...
See the full eligibility criteria
- people will enroll onto the study and undergo screening. people 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...
- 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
- Platelet count \>=100 x 10\^9/L.
- If on hydroxyurea, participant must have been on stable dose of hydroxyurea (defined as a stable dose for at least 3 months and inclusive of dose modifications for hematological toxicity per PI discretion) prior to...
- For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy...
- For women of reproductive potential as well as men and their partners who are women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use 2 effective forms of contraception from the...
- Be willing to comply with all study procedures for the duration of the study.
- An individual who meets any of the following criteria will be excluded from participation in this study:
- 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...
- 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...
- 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...
- History of drug-induced cholestatic hepatitis.
- History of any primary malignancy.
- Testing positive for human immunodeficiency virus 1 or 2 Ab with evidence for ongoing active infection (i.e., CD 4 count \ 100,000 copies/ml) on antiretroviral therapy.
- Current or recent history of psychiatric disorder that, in the opinion of the Investigator or Medical Monitor, could compromise the ability of the subject to cooperate with study visits and procedures.
- Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo.
- Use of newly approved SCD therapy (L-glutamine, voxelotor or crizanlizumab) is NOT permitted on this study. people who have received newly approved SCD therapy in the 7 days prior to signing consent will be excluded.
- Having had a prior bone marrow or stem cell transplant.
- Currently pregnant or lactating.
- Currently receiving strong inhibitors of CYP3A4/5 that have not been stopped for \>=5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong inducers of CYP3A4 that have not been stopped for...
- Currently receiving erythropoiesis stimulating agents.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 12 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.