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Options For People with Sickle Cell Disease

Learn more to see if you may be eligible to
participate in this research study.
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About The Pioneer Study

People with Sickle Cell Disease (SCD) have a disease that causes their red blood cells to be crescent (sickle)-shaped instead of round. These cells have difficulty carrying oxygen and can get stuck. This can cause pain, tiredness, and create difficulty breathing. In this study, researchers are exploring if an investigational oral medicine (Pociredir) still being studied is safe and has the desired biologic effect for which it was designed to treat SCD. The study medicine is investigational because it has not been approved for use except in clinical trials.

All people that are eligible to take part in the study will take medicine by mouth once a day for 12 weeks. The total time on the study may be up to 4 months, including follow-up with the doctor or study team 4 weeks after finishing study treatment.

About The Study Drug (Pociredir)

Hemoglobin is a protein found in red blood cells that carries oxygen throughout the body.

There are many types of hemoglobin, including fetal and adult hemoglobin.

People normally stop making fetal hemoglobin after birth, and instead start making adult hemoglobin.
In Sickle Cell Disease, this adult hemoglobin is abnormal and causes red blood cells to be mis-shapen (sickle-shaped) and to burst open.
  • On the other hand, fetal hemoglobin doesn’t cause sickling of the red blood cells and works fine for oxygen transport.
  • In a person with Sickle Cell Disease, making more fetal hemoglobin can improve the symptoms of SCD, and can make the disease less severe.
  • Pociredir acts to increase the amount of fetal hemoglobin made in red blood cells.

Why Consider Participating in a Clinical Trial?

If you decide to see if you qualify to take part in the study, you will help to advance the scientific 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 at no cost. All research has some risks. The study team will talk with you about risks and answer any questions.

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)
  • Have previous experiences with hydroxyurea for at least 6 months at the highest dose possible, and it has not been effective, or you were unable to take hydroxyurea for reasons specified by your doctor

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

Medical Care During The Study

Taking part in this study is your choice. Whether or not you decide to participate will not change your regular medical care. 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.

What to expect at the start of the clinical trial

If you decide to take part in the study, the study 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

Participants will receive the study treatment, tests and procedures at no cost. Daily compensation will be provided.

Types of Support Available for Participants

Participants and caregivers may be eligible for support from Fulcrum

Support from Fulcrum may include

Transportation

Meals

Gas

Hotels

Childcare Reimbursements

Wage/Time Loss

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 your doctor, 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.