The Northstar Studies are global clinical studies being conducted to determine if the one-time gene therapy can decrease or eliminate the need for continued blood transfusions in people living with transfusion-dependent beta-thalassemia, while also being safe and well-tolerated. The goal of this investigational gene therapy treatment is to help patients make more beta-globin, which will in turn create more hemoglobin and produce healthy red blood cells.
The Northstar Studies require a commitment, lasting a little more than two years. At the end of the study, participants are asked to participate in a separate, 13-year follow-up study. Northstar Study participants will undergo many tests and procedures, which will be monitored closely by a team of healthcare professionals.
The Northstar Study (HGB-204), an ongoing Phase 1/2 study, is fully enrolled and no longer enrolling new patients. Early data from the Northstar Study shows that all non-β0/β0 patients followed for 12 months or longer have so far been transfusion independent. The study is going and patients continue to be followed.
The Northstar-2 Study (HGB-207) is currently enrolling and seeking patients living with transfusion-dependent non beta zero-thalassemia (non-β0/β0) at sites around the world.
The Northstar-3 Study (HGB-212) is now enrolling patients living with transfusion-dependent beta-zero (β0/β0) beta-thalassemia at study sites around the world. Approximately one-third of people with transfusion-dependent beta-thalassemia living in the United States or United Kingdom have the β0/β0 genotype, which means they have gene mutations that produce no beta-globin. More information about this study can now be found on the NorthStar-3 Study page.
A clinical research study is a scientific evaluation used to determine if and how a new medication or treatment may work in people. Through clinical research studies, physicians may identify new and improved ways to prevent, detect, diagnose, manage and treat different diseases.
Before a physician can prescribe a medication or treatment, it must be proven to be safe and effective in people. Testing of a potential treatment is done in people who volunteer to participate in a clinical research study and understand that unknown risks and benefits are associated with the investigational treatment and participating in a clinical study.
People participate in clinical research studies for many reasons. These include the opportunity to take an active role in their healthcare and to help others with the same disease in the future by contributing to medical research. Additionally, clinical research studies may provide additional treatment options for people living with chronic conditions or other diseases.