Because living with sickle cell disease should be made easier.
This is a Phase 1 study evaluating the safety and effectiveness of an investigational medicine to prevent painful episodes caused by sickle cell disease (SCD) called vaso-occlusive crises (VOCs). Adults ages 18 to 65 years old with sickle cell disease who have been seen by a healthcare provider for 2 to 10 VOCs in the past year may be eligible. Learn more to see if you can participate.
Who may participate
Each clinical study has its own guidelines for who can participate, called eligibility criteria. However, only the research study staff can determine if you qualify to enroll in the study.
What to expect
About sickle cell disease
Sickle cell disease (SCD) is a genetic (inherited) disease that causes red blood cells to be shaped like a crescent or half-moon instead of round. These abnormally shaped cells tend to get stuck in small blood vessels, which blocks blood flow and causes severe pain. People with SCD may also have a chronic shortage of red blood cells (anemia) that causes tiredness, weakness, and difficulty breathing.
About vaso-occlusive crisis
Vaso-occlusive crisis (VOC), or sickle cell crisis, is a painful complication of SCD. VOCs occur when abnormally-shaped red blood cells get stuck and clog small blood vessels, blocking blood flow to certain parts of the body. These blockages cause severe pain and may lead to organ damage from lack of oxygen.
Although there are currently some treatments available, most people with SCD continue to experience VOCs. New treatment options are needed to improve the lives of people living with SCD by reducing the duration and severity of VOCs.
Frequently asked questions
How long will I be in the study?
Participation in this study will take approximately 5 months (including the time it takes to complete screening).
What if this study is not right for me?
You can choose to leave the study at any time, for any reason. Choosing to leave the study will not affect your SCD treatment or future medical care.
Am I guaranteed to receive the study medicine?
Yes, all participants will receive 3 doses of the study medicine. This study does not include a placebo (no active ingredient).
What are the possible side effects?
All medicines have the potential to cause unwanted side effects. The study doctor will discuss the possible risks and benefits of participating in this study with you before you choose to enroll. You can choose to leave the study at any time.
Does it cost anything to participate?
No. Participants will receive all study-related tests and procedures for free. Additionally, participants will receive reimbursement for attending study visits and completing daily entries in the electronic diary. Transportation and nearby lodging can also be arranged at no cost.