RecruitingPhase 1

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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 65 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Visits mentioned: 6 visits
  • Time periods mentioned: 12 weeks, 6 weeks, 2 weeks
  • Procedures mentioned: Blood draws, Physical exams, Pills

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Design: Participants will have 6 clinic visits over 12 weeks."
  • "They will have a physical exam with blood tests."
  • "Fostamatinib is a tablet taken by mouth."
  • "Participants will take the drug at home, twice a day, for up to 6 weeks."
  • "At each visit they will have a physical exam with blood tests."
  • "They will have a physical exam and blood tests; they will be checked for any side effects of the drug."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety of fostamatinib at increasing doses in adults ages 18 to 65 with stable sickle cell disease. It checks how well people handle the drug. Participants take tablets twice a day for up to 6 weeks and have 6 clinic visits over 12 weeks.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be 18 to 65 years old.
  • Have sickle cell anemia of the HbSS or HbS beta-zero type, confirmed by a blood test.
  • Have had no blood transfusion in the past 60 days, or have no hemoglobin A on a blood test. Testing to confirm the disease must be at least 60 days after any transfusion.
  • Meet the study’s liver test and blood count limits, including hemoglobin of at least 7 g/dL. Hemoglobin carries oxygen in the blood.
  • If taking hydroxyurea, have been on a stable dose for at least 3 months, with some doctor-approved dose changes allowed.
  • If able to become pregnant, have a negative pregnancy blood test.
  • If pregnancy is possible for you or your partner, use 2 effective birth control methods or continue your usual practice of not having sex. Follow this rule through 28 days after the last dose.
  • Sign a consent form before any study tests and agree to follow all study steps.

Who can't join

  • A sickle cell pain crisis needing medicine by injection or through a vein within 14 days before signing consent.
  • Certain health problems, including low infection-fighting white blood cells, a past brain condition called PRES, poorly controlled high blood pressure, drug-caused liver injury, or a history of cancer. Some exceptions apply.
  • Active hepatitis B or C, which are liver infections, or active HIV that meets the study’s blood test limits.
  • A mental health condition that could prevent you from following study steps, or another health problem that makes joining unsafe or affects study results.
  • Pregnancy, breastfeeding, or a past bone marrow or stem cell transplant.
  • Another ongoing treatment trial; use of L-glutamine or crizanlizumab during this study or within 7 days before consent; or medicines that boost red blood cell production.
  • Certain medicines that interact with fostamatinib, including some drugs such as rosuvastatin or digoxin. Some medicines must be stopped for a set time before consent.
  • The listing says people who have NOT received fostamatinib in the past 3 months are excluded. This wording is unclear; ask the study team to confirm.

Simplified for readability. The official criteria below are what the study team uses.

Official 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 be eligible to participate in this study, an individual must meet all of the following criteria: 1. Have provided signed written informed consent prior to performing any study procedure, including screening procedures. 2. Age between 18-65 years 3. 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. 4. No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC) 5. Have adequate organ function, as defined by: 1. 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. 2. Absolute neutrophil count \>=1.5 x 10\^9/L. 3. Hemoglobin \>= 7 g/dL 4. Platelet count \>=100 x 10\^9/L. 6. 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 signing consent. 7. 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, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception). 8. 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 time of giving informed consent, during the study, and for 28 days (both men and women) following the last dose of study treatment. An effective form of contraception is defined as hormonal oral contraceptives, injectables, patches, intrauterine or subdermal contraceptive implants, and barrier methods. 9. Be willing to comply with all study procedures for the duration of the study. EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: 1. Pain crisis requiring parenteral treatment within 14 days of signing consent. 2. 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: 1. History of neutropenia (benign ethnic neutropenia and/or acquired neutropenia related to drug suppression by hydroxyurea and/or cyclic hematopoiesis are permitted). 2. History of posterior reversible encephalopathy syndrome (PRES) 3. 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. 4. 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. 5. History of drug-induced cholestatic hepatitis. 6. History of any primary malignancy. 7. Testing positive for human immunodeficiency virus 1 or 2 Ab with evidence for ongoing active infection (i.e., CD 4 count \<400/microliter and viral load \>100,000 copies/ml) on antiretroviral therapy. 8. 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. 9. Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo. 10. Use of newly approved SCD therapy (L-glutamine or crizanlizumab) is NOT permitted on this study. Subjects who have received newly approved SCD therapy in the 7 days prior to signing consent will be excluded. 11. Having had a prior bone marrow or stem cell transplant. 12. Currently pregnant or lactating. 13. 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 \>=28 days or a time frame equivalent to 5 half-lives (whichever is longer), prior to signing consent. SCD patients that are receiving treatment with CYP3A4 substrate drugs, some BCRP substrate drugs (eg. rosuvastatin), and some P-glycoprotein substrate drugs (eg. Digoxin) are excluded from the study. 14. Currently receiving erythropoiesis stimulating agents. 3. Has not received fostamatinib in 3 months prior to signing consent

Study sites (1)

MD