• GBT HOPE SCD Program

    The GBT HOPE Sickle Cell Disease (SCD) program currently consists of three interventional research studies and one open label extension study that evaluate whether an investigational medication will be effective in reducing the damage the Sickle Cell Disease causes to red blood cells and therefore, decrease the day-to-day symptoms of Sickle Cell Disease in adolescents and adults who have the disease. The studies will also examine the effects of different doses of the investigational medication to determine the best dose to give to adolescents and adults with Sickle Cell Disease.

  • GBT HOPE Study

    The GBT HOPE study will evaluate the effectiveness of the investigational medication compared to placebo in participants with Sickle Cell Disease.

    Who can participate in the GBT HOPE study?

  • What is the investigational medication in the GBT HOPE study?

    GBT440 binds to red blood cells, preventing sickling and allowing them to travel normally through blood vessels

    Voxelotor, previously referred to as GBT440, is the investigational medication in the GBT HOPE study, is a potential therapy for patients with Sickle Cell Disease that is designed to attack the cause of the disease. This investigational medicine is taken by mouth daily and is designed to work by keeping hemoglobin molecules in the red blood cells to keep their normal shape and stop them from sticking to each other.

  • GBT HOPE-Kids 1 Study

    Part A of the study evaluated the safety of one dose of the investigational medication in participants with Sickle Cell Disease.


    Part B of the study evaluated the safety and effectiveness of two doses of the investigational medication in participants with Sickle Cell Disease.

     

    Part C of this study is evaluating the safety and effectiveness of the investigational medication on patients with Sickle Cell Disease, including whether the investigational medication can improve blood flow in the brain as measured by Transcranial Doppler (TCD).

     

    Parts A and B of this study are no longer enrolling study participants. Only Part C of this study is open for enrollment.

    Who can participate in the GBT HOPE-Kids 1 study?

    To qualify for the GBT HOPE-Kids 1 study, you or your loved one must:

    • Be between 4 and 17 years of age
    • Be diagnosed with Sickle Cell Disease

    How long will GBT HOPE-Kids 1 study participation last?

    The study consists of three parts – Part A, Part B, and Part C. Parts A and B are no longer enrolling. Part C will last about 399 days (about 12 months)

    How many people will participate in the GBT HOPE-Kids 1 study?

    About 6 people (ages 6-11 years old) with Sickle Cell Disease are expected to participate in Part A of the study.
     
    Between 24 and 36 people (ages 12 to 17 years old) with Sickle Cell Disease are expected to participate in Part B of the study.

     

    Up to 70 participants (ages 4 to 17 years old) with Sickle Cell Disease are expected to participate in Part C of the study.

    Will I need to pay for participation in the GBT HOPE-Kids 1 study?

    Should you or your loved one participate in the GBT HOPE-Kids 1 study, the investigational medication and all study-related tests will be provided at no cost to you.

     

    Transportation assistance to and from the study site may be available

  • What will happen during Part C of the GBT HOPE-Kids 1 study

    Participation in Part C of the study will be divided into three periods:

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    Screening Period

    35 days

    Before you or your loved one are able to participate in the study, you will need to go through a screening process to determine if you meet all the medical requirements for participation. The study staff will review medical history and conduct tests to make the decision, as discussed with potential participants during the informed consent process.

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    Dosing Day

    48 weeks

    Participants will take the investigational medication daily

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    Follow-up Period

    4 weeks

    After participants take the investigational medication, follow-up visits are required to ensure that participants remain healthy and to observe any lasting effects of the investigational medication on their Sickle Cell Disease

  • What is the investigational medication in the GBT HOPE-Kids 1 study?

    GBT440 binds to red blood cells, preventing sickling and allowing them to travel normally through blood vessels

    voxelotor, previously referred to as GBT440, is the investigational medication in the GBT HOPE-Kids 1 study, is a potential therapy for patients with Sickle Cell Disease that is designed to attack the cause of the disease. This investigational medicine is taken by mouth daily and is designed to help hemoglobin molecules in the red blood cells to keep their normal shape and stop them from sticking to each other.