• GBT HOPE SCD Program

    The GBT HOPE Sickle Cell Disease (SCD) program currently consists of two clinical research studies that will 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?

    To qualify for the GBT HOPE study, one must:

    • Be 12 to 65 years of age
    • Be diagnosed with Sickle Cell Disease
    • Have had at least one pain crisis in the past 12 months

    How long will GBT HOPE study participation last?

    Participation in the study will last at least 12 weeks and may go up to 72 weeks (1.5 years). Participants who complete the study may be eligible to continue receiving the investigational medication in an extension study.

    How many people will participate in the GBT HOPE study?

    Between 340 and 400 adolescents and adults with Sickle Cell Disease are expected to participate.

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

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

     

    Transportation assistance to and from the study site may be available.

  • What will happen during the GBT HOPE study?

    Throughout the study, participants are required to keep an electronic diary to record events related to their Sickle Cell Disease. Participation in the study will be divided into three consecutive time 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. You will also be asked to complete a daily electronic diary to record the symptoms related to your Sickle Cell Disease.

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    Treatment Period

    12 to 72 weeks

    Participants will be divided into three groups. Members of each of the three groups will receive either the investigational medication or placebo. Placebo looks like the investigational medication but has no active ingredients. Placebo acts as a “control” in clinical trial data, meaning it allows scientists to account for any subjective opinion that participants may have on whether what they are taking is working or not.

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    Follow-up Period

    4 weeks

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

  • 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

    GBT440, 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 will evaluate the safety of one dose of the investigational medication in participants with Sickle Cell Disease.

     

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

    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 12 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 two parts – Part A and Part B. Part A will last about 44 days (about 1.5 months) and Part B will last about 226 days (about 7.5 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.

    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.

     

    Transportation assistance to and from the study site may be available.

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

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

    screening checklist icon

    Screening Period

    28 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

    1 day

    Participants will take the investigational medication on Day 1.

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    Follow-up Period

    15 days

    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 will happen during Part B of the GBT HOPE-Kids 1 study?

    Throughout Part B of the study, participants are required to keep an electronic diary to record events related to their Sickle Cell Disease. Participation in Part B of the study will be divided into three periods:

    screening checklist icon

    Screening Period

    28 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. You will also be asked to complete a daily electronic diary to record the symptoms related to your Sickle Cell Disease.

    medication icon

    Treatment Period

    24 weeks

    Participants will take the investigational medication daily.

    follow up checklist icon

    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

    GBT440, 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.