A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Recruiting at 8 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: bluebird bio

Must not be taking: Investigational drugs

Disqualifiers: HIV, HBV, HCV, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 25

Duration: 1 day

19 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

What is the purpose of this trial?

This study is evaluating whether a drug can be used to treat a blood disorder.

Research Team

Himal L Thakar, MD

Principal Investigator

bluebird bio

Eligibility Criteria

Inclusion Criteria

Clinically stable and eligible to undergo HSCT.

• Participants less than or equal to (<=) 50 years of age at the time of consent or assent (as applicable), and able to provide written consent (adults, or legal guardians, as applicable) or assent (adolescents or children). Provided that the data monitoring committee (DMC) has approved enrolling participants younger than 5 years of age, participants younger than 5 years of age may be enrolled if they weigh a minimum of 6 kilograms (kg) and are reasonably anticipated to be able to provide at least the minimum number of cells required to initiate the manufacturing process.

Diagnosis of TDT with a history of at least 100 milliliter per kilogram per year (mL/kg/year) of pRBCs in the 2 years preceding enrollment (all participants), or be managed under standard thalassemia guidelines with >= 8 transfusions of pRBCs per year in the 2 years preceding enrollment (participants >=12 years).

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of LentiGlobin BB305 Drug Product through autologous hematopoietic stem cell transplantation

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of transfusion independence and quality of life

24 months

Regular visits (in-person and virtual) up to Month 24

Long-term Follow-up

Participants continue to be monitored for long-term safety and efficacy outcomes

Beyond 24 months

Treatment Details

Interventions

• LentiGlobin BB305 Drug Product

Participant Groups

Group I: LentiGlobin BB305 Drug Product Experimental Treatment 1 Intervention

LentiGlobin BB305 Drug Product (autologous CD34+ cell-enriched population that contains cells transduced with LentiGlobin BB305 lentiviral vector encoding human βA-T87Q-globin)

Who Is Running the Clinical Trial?

bluebird bio

Lead Sponsor

Trials

21

Recruited

2,000+