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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 disease that causes their red blood cells to be crescent-shaped instead of round. These cells get stuck in small blood vessels and this causes pain, tiredness, and difficulty breathing. In this study, researchers are determining if an investigational medicine is safe and works well for treating SCD in adults. The study medicine is investigational because it has not been approved for use yet. 

All people that take part in the study will take medicine by mouth once a day for between 4-12 weeks. The first part of the study (Part A) is 4 weeks long with 6 office visits. Participants can continue the study in Part B for 8 more weeks and attend 4 additional office visits. The total time on the study may be up to 15-16 weeks, including follow-up with the doctor or study team for 3 weeks after finishing treatment. 

Medical Care

Taking part in this study is your choice. Joining this study does not change your regular medical care if you decide not to participate.

How to Participate

If you decide to take part in the study, a doctor will take a series of measurements early in the study. These measurements include:

  • Review of medical history and SCD symptoms
  • Review of medications 
  • Physical examination
  • Heart rate and blood pressure
  • Heart activity 
  • Blood sample 
  • Urine collection 

Taking part in this study is your choice. Joining this study does not change your regular medical care if you decide to not participate. We suggest you talk to family, caregivers, doctors, and the study team about taking part in this study and whether it is right for you.

Why Should I Participate?

If you decide to take part in the study, you help advance science, our understanding of SCD, and potentially improve future medicines for other people with SCD. There may be no direct benefit to you. People who are able to join the study will receive study-related tests and procedures for free. All research has some risks. The study team will talk with you about risks and answer any 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?

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)
  • Do not take hydroxyurea for your SCD, or have been on a stable dose of hydroxyurea for at least 3 months

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.