The Northstar-2 Study (HGB-207) is a Phase 3 global clinical study seeking to enroll patients who cannot make enough of the beta-globin portion of hemoglobin and who you do not have a β0/β0 genotype. Based on the early data from the Northstar Study, it is believed that the current lentiviral vector technology may be more effective in people who do not have a β0/β0 genotype. The safety profile remains consistent with myeloablative conditioning using single agent busulfan (chemotherapy) with no ≥ Grade 3 drug product-related adverse events (AEs) reported.
The Northstar-2 Study is currently enrolling people living with transfusion-dependent beta-thalassemia who cannot make enough of the beta-globin portion of hemoglobin and who do not have a β0/β0 genotype.
To be considered for the Northstar-2 Study, you must meet the following initial criteria:
The Northstar-2 Study has many other requirements to help ensure the safety of all participants which will be reviewed with you by your physician or a study site. A full list of all the NorthStar-2 criteria and additional information about the study can be found on ClinicalTrials.gov. Review this information with your physician who can contact a Northstar Study site for addtitional information regarding the study criteria and status.
Those who are eligible for the study will also need to be able to undergo an autologous hematopoietic stem cell transplant, which is how the study drug is administered. If you are eligible and decide to participate, the study physician will discuss all potential benefits and risks in detail with you; however, it is not possible to know in advance if you will benefit from participating in the study. Since some of the potential risks are serious, you should talk to your physician about both the potential risks and benefits before deciding to participate.
If you are interested in learning more about the Northstar-2 Study, here are the steps to follow:
Northstar-2 Study participants will undergo many tests and procedures, which will be monitored closely by a team of healthcare professionals. The study medicine will be provided free of charge by the study sponsor and any costs incurred as a direct result of participation (e.g., travel expenses) will be reimbursed.
Study participants are encouraged to talk to their study team about gene therapy, chemotherapy and all the details associated with fertility and the options available to them at their study site. Fertility harvest, preservation and storage is available at no charge to people participating in any of the Northstar Studies. The options available may vary by study location.
There are four stages to the study: screening, blood stem cell collection, conditioning and infusion and follow-up. Full conditioning is needed to fully clear out the bone marrow for new cells to be infused and multiply. Below is an overview of the study and what may be expected.
The Northstar-3 Study (HGB-212) is a Phase 3 global clinical study seeking to enroll patients who do not make the beta-globin portion of hemoglobin and who have a β0/β0 genotype. Based on the early data from the Northstar Study, it is believed that the current lentiviral vector technology may be more effective in decreasing the need of transfusions in people who have a β0/β0 genotype.