Gene Therapy for Sickle Cell Disease

Palo Alto (17 mi)

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: bluebird bio

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

Eligibility Criteria

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

I am mostly able to care for myself and carry out daily activities.

I have been treated for sickle cell disease and followed at a medical center with detailed records for the past 2 years.

I have had a bad reaction to or did not respond to hydroxyurea.

I have sickle cell disease with a specific genetic makeup.

I am between 2 and 50 years old.

Exclusion Criteria

I am currently pregnant, breastfeeding, or planning to become pregnant.

I cannot receive red blood cell transfusions.

My bone marrow is not working properly.

I need blood-thinning medication from the start of my treatment until my platelet count recovers.

I do not have any serious ongoing infections.

A close family member has or might have a hereditary cancer syndrome.

I have genetic mutations that affect my α-globin genes.

My liver disease is in an advanced stage.

I have severe blood vessel problems in my brain, including a stroke or significant narrowing/blockage.

I have had an allogeneic transplant or gene therapy before.

I am not allergic or unable to take plerixafor, busulfan, or similar medications.

Treatment Details

The study tests a gene therapy called bb1111 in about 35 people with SCD. It's an open-label Phase 3 trial where participants receive one dose of the therapy through hematopoietic stem cell transplantation (HSCT). The goal is to see how well this new treatment works compared to traditional treatments.

1Treatment groups

Experimental Treatment

Group I: bb1111Experimental Treatment1 Intervention

Subjects will receive treatment with a single dose of Drug Product manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and apheresis, transduced with BB305 lentiviral vector (LVV) encoding the human beta-A-T87Q globin gene. Plerixafor mobilization and apheresis will also be used for collection of rescue cells.