Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

Bluebird Bio

Status: Invitation-only

Conditions: Sickle Cell Disease

Treatments: Other: Safety and efficacy assessments

Study type: Observational

Funder types: Industry

Identifiers

NCT04628585

LTF-307

2024-513901-30-00 (EU Trial (CTIS) Number)

2019-004266-18 (EudraCT Number)

Details and patient eligibility

About

This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

Enrollment: 85 estimated patients

Sex: All

Ages: 2 to 53 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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 sickle cell disease in a bluebird bio-sponsored clinical study

Exclusion criteria

• There are no exclusion criteria for this study

Trial design

85 participants in 1 patient group

Subjects with sickle-cell disease

Description:

Subjects treated with ex vivo gene therapy drug product for sickle cell disease in a bluebird bio-sponsored study who agree to participate in this long-term follow-up study

Treatment:

Other: Safety and efficacy assessments