Gene Therapy for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a gene therapy treatment, called bb1111, for sickle cell disease. The goal is to determine if a one-time stem cell transplant can help individuals with sickle cell disease by modifying their genes to produce healthier blood cells. It targets those who have experienced at least four painful episodes in the past two years, despite receiving care. As a Phase 3 trial, it represents the final step before FDA approval, offering participants an opportunity to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are on hydroxyurea, your neutrophil count requirements are different, which might imply some consideration for this medication.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Research shows that the gene therapy bb1111, also known as LentiGlobin BB305, has been studied for its safety and effectiveness in treating sickle cell disease (SCD). The treatment helps produce a stable form of hemoglobin, called HbA T87Q, which reduces the breakdown of red blood cells—a common issue for people with SCD.
In earlier studies, participants generally tolerated the treatment well. Although all treatments can have side effects, these studies reported no major safety concerns. This suggests the treatment is likely safe, but discussing any potential risks with the research team before joining a trial is important.12345Why do researchers think this study treatment might be promising for sickle cell disease?
Unlike the standard treatments for sickle cell disease, which often involve regular blood transfusions and pain management medications, bb1111 is a gene therapy that targets the root cause of the condition. This treatment is unique because it uses a lentiviral vector to introduce a modified beta-globin gene into the patient's own stem cells. By doing so, bb1111 has the potential to produce normal hemoglobin and significantly reduce the symptoms of sickle cell disease. Researchers are excited about bb1111 because it offers a one-time treatment that could provide a long-term solution, moving beyond managing symptoms to potentially curing the disease.
What evidence suggests that this gene therapy might be an effective treatment for sickle cell disease?
Research shows that the gene therapy bb1111, also known as LentiGlobin, may help treat sickle cell disease (SCD). In this trial, participants will receive a single dose of bb1111, which uses a special tool to add a modified gene into the patient's stem cells. This new gene helps produce healthier red blood cells, reducing the symptoms of sickle cell disease. In earlier studies, patients who received this treatment experienced fewer painful sickle cell crises. Early results indicate that many patients continue to have better blood counts and fewer symptoms over time. Overall, these findings suggest that bb1111 could be a promising option for people with SCD.12467
Who Is on the Research Team?
Anjulika Chawla, MD, FAAP
Principal Investigator
bluebird bio, Inc.
Are You a Good Fit for This Trial?
This trial is for individuals aged 2 to 50 with sickle cell disease (SCD), who have had at least four pain episodes in the last two years and either failed hydroxyurea treatment or can't tolerate it. Participants must weigh over 6 kg, have a certain level of physical ability, and agree to use contraception if applicable. Those with inadequate bone marrow function, prior malignancies, genetic risks for other blood disorders, or severe cerebral vasculopathy cannot join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Mobilization and Apheresis
Plerixafor mobilization and apheresis are used for collection of autologous CD34+ hematopoietic stem cells
Transplantation
Subjects receive a single dose of Drug Product manufactured with autologous CD34+ hematopoietic stem cells transduced with BB305 lentiviral vector
Follow-up
Participants are monitored for safety and effectiveness after treatment, including resolution of VOEs and globin response
What Are the Treatments Tested in This Trial?
Interventions
- bb1111
Find a Clinic Near You
Who Is Running the Clinical Trial?
bluebird bio
Lead Sponsor
Genetix Biotherapeutics Inc.
Lead Sponsor