Global Blood Therapeutics (GBT), Inc. Community Q & A Document
Global Blood Therapeutics (GBT), Inc. is a biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat blood-based disorders with significant unmet need. Our most advanced program, GBT440, is in development as a once-daily therapy taken by mouth for sickle cell disease.
At GBT, we are committed to advancing the science and transforming the treatment of sickle cell disease. We maintain an open dialogue with the sickle cell community to strengthen our understanding of the needs of the community and share the latest information on GBT’s sickle cell research, including our ongoing clinical trials.
Today January 18, 2017, we were pleased to announce via a press release, the enrollment of the first patient in the GBT HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a pivotal Phase 3 clinical study of our investigational drug candidate, GBT440, in people living with sickle cell disease.
The HOPE study has an innovative design that will allow GBT to utilize a primary endpoint that is a clinically meaningful measure of sickle cell disease, and a novel patient reported outcome (PRO) tool that will allow us to capture patients’ daily experience of pain, fatigue and other symptoms of sickle cell disease. We are deeply grateful to the patients, families, healthcare providers, and advocacy organizations that contributed their insights during the design of our clinical program and collaborated with us in the development of the PRO tool, which is designed to measure the true burden of painful crises and other sickle cell symptoms.
As we move forward with our efforts, we are committed to working closely with you, our patient advocacy community partners, to increase awareness of GBT440 and the HOPE study.
Following is a brief list of Questions & Answers to assist you, or others in your organization, address questions or concerns that your constituents may have regarding GBT440 and the HOPE study. You can also direct the community to the GBT HOPE Clinical Research Program website gbtsicklecellstudy.com that will be launched later in January. The website will have general information about the HOPE study, a pre-screener to help patients (or caregivers) determine if they may be eligible for the study or other sickle cell related studies GBT is conducting over time, and contact information of study sites in the United States.
If you have additional questions, please contact Eydith Comenencia Ortiz, Associate Director of Community Engagement & Partnership, at patientadvocacy@globalbloodtx.com.
GBT HOPE Study Q & A
What is GBT440?
GBT440 is a potential therapy for patients with sickle cell disease that is designed to attack sickle hemoglobin, the cause of the disease. GBT440 works by helping hemoglobin, the molecules inside red blood cells, hold onto more oxygen as the red blood cells travel through the body. This may help keep red blood cells in their normal shape and stop sickling.
In clinical studies to date, GBT440 has been well-tolerated and has reduced the damage to red blood cells caused by sickle cell disease. A large, ongoing global study (Phase 3 clinical trial), called the GBT HOPE study, will evaluate the safety and efficacy of GBT440 in adolescents and adults with sickle cell disease.
What is the GBT HOPE Study?
The GBT HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) study is a global clinical research study that will evaluate whether GBT440 will be effective in reducing anemia, and severity of daily symptoms due to sickle cell disease in adolescents and adults with sickle cell disease. It will also study the effects of different doses of GBT440 to determine the best dose to give to adolescents and adults with sickle cell disease. The study will utilize an increase in hemoglobin as a primary measure to evaluate the effectiveness of GBT440, and an electronic patient diary that is designed to capture patients’ daily experience and overall severity of symptoms due to their sickle cell disease.
The HOPE study is a randomized placebo-controlled clinical study, which means that participants will be assigned at random (like flipping a coin) to receive either the investigational medication, GBT440, or placebo. Placebo is a “dummy pill” that looks like the investigational medication but does not contain any drug.
After participants complete the study, they may be able to take GBT440 in an extension of the HOPE study, regardless of whether they received GBT440 or placebo during the study.
Who can participate in the HOPE Study?
The HOPE study will include up to 400 adolescents and adults living with sickle cell disease (ages 12 to 65) that have had at least one sickle cell crisis or vaso-occlusive crisis in the previous year.
Potential HOPE study participants need to go through a screening process to determine if they meet all the requirements for participation. Study doctors and staff will evaluate potential participants and explain other requirements for enrollment. If you or a loved one is interested in participating in the GBT HOPE study, go to gbtsicklecellstudy.com or talk to your doctor.
How do I know if there is a clinical study site near me?
The HOPE study is a global clinical trial that will include sites throughout the United States. There will also be study sites in Canada, and in some countries in Europe, the Middle East, and Africa. GBT is committed to working with sickle cell patient advocacy and community based organizations to provide information about clinical trial sites opening in your area. To find information about study sites in your area you can go to gbtsicklecellstudy.com talk to your doctor, or contact your local community-based organization(s).
