Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Bluebird Bio Inc.1 site in 1 country3 target enrollmentJune 25, 2024

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Not specified
Sponsor: Bluebird Bio Inc.
Enrollment: 3
Locations: 1
Primary Endpoint: The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)
Status: Recruiting
Last Updated: last year

Evaluate long-term safety of treatment with lovocel (lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD)
Evaluate long-term efficacy of treatment with bb1111 in subjects with SCD

Registry

euclinicaltrials.eu

Start Date

June 25, 2024

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sponsor

Responsible Party

Principal Investigator

Clinical Trial Information

Scientific

Bluebird Bio Inc.

•Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)
•Treated with drug product for therapy of SCD in a bluebird bio-sponsored clinical study

The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)

The number of subjects with new or worsening hematologic disorders

The number of subjects with new or worsening neurologic disorders

The number of subjects with malignancies

Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
Annualized number of severe VOEs over time through Year 15
Annualized number of VOEs over time through Year 15
Change from parent study baseline in annualized number of severe VOEs over time through Year 15
Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb
Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase
Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content