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Help Researchers
Expand Treatment
Options For Sickle
Cell Disease Patients

About the Study

People with Sickle cell disease (SCD) have a genetic disorder that causes their red blood cells to be crescent-shaped instead of round. Because of their abnormal shape, these cells frequently become stuck in small blood vessels, leading to anemia and painful episodes called vaso-occlusive crises. In this study, researchers are determining if an investigational medicine is safe and effective for treating SCD in adults. The study medicine is considered investigational because it has not yet been approved for use. 


All participants will take the study medicine orally once a day for one or two treatment periods. The first treatment period (Part A) is 4 weeks long with 6 office visits and 3 overnight stays required during this time. Participants may choose to continue treatment in Part B for an additional 8 weeks, with 4 more office visits during this time. Participants’ total time in the study may be up to 15-16 weeks, including a 3 week follow-up period after completing treatment. 

Medical Care

Taking part in this study is voluntary and there is no penalty or change to your regular medical care if you decide not to participate. 

How to Participate

If you choose to participate, you will undergo a series of screening assessments including:

  • Review of medical history and SCD symptoms
  • Review of medications 
  • Physical examination
  • Vital signs 
  • Blood draws 
  • Urine collection 
  • Electrocardiogram (ECG)

Taking part in this study is voluntary and there is no penalty or change to your regular medical care if you decide not to participate. We encourage you have conversations with family, caregivers, doctors and the study team about taking part in this study and whether it is right for you.

Why Should I Participate?

By choosing to participate, you will be helping to advance science by contributing to our understanding of SCD and potentially improving future treatment options for others with SCD. There may be no direct benefit to you. Eligible participants will receive study-related tests and procedures at no cost. All research has some risks; The study team will discuss all risks with you and answer any additional questions you may have.

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About Clinical Trials

Before a new treatment or medicine is available to the public, it is first tested in a series of carefully regulated

clinical trials. All clinical trials must be reviewed and approved by committees responsible for ensuring that

studies are ethical, and that the rights and privacy of study participants are protected.

Why are clinical trials conducted?  

Without clinical trials, we would not have most of the modern medicines we use today. Clinical trials provide a framework for the medical community to discover ways to diagnose, prevent and treat disease. They are essential to understanding more about whether a potential medicine may be better than current standards of care or may offer alternative treatment for people not responding well to their current treatment. Clinical research studies also help us learn more about the side effects of a potential treatment. 

Is taking part in a clinical research study voluntary?

Yes, taking part in any clinical research study is completely voluntary. You can choose to leave a study at any time, for any reason, without any impact to your future medical care.

What is Informed Consent?

Before you can join any clinical research study you need to give your consent. You must be fully informed about the study before you give your consent. You must give your consent voluntarily; you should not feel pressured to do so. You should feel free to discuss the study with family and friends before you make your decision.

What is a study drug?

A study drug is a medicine or treatment that has not yet been approved by the U.S. Food and Drug Administration (FDA), The European Medicines Agency (EMA), or similar agencies in other countries for doctors to prescribe for a particular condition.

Am I Eligible?

Am I Eligible?

You may be eligible to participate in this clinical research study if you:

  • Are between 18 and 65 years of age 
  • Have been diagnosed with Sickle Cell Disease (SCD)

Note: These are not the only eligibility criteria for this clinical research study, and other criteria may exclude you. A clinical research team member will help determine if you meet all necessary criteria to participate.