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

Plain-language summary

This study follows, over the long term, people who were treated with an ex vivo gene therapy for sickle cell disease, assessing safety and effectiveness. It can enroll people who received the drug product for sickle cell disease in a bluebird bio-sponsored clinical study. It is a study without an assigned phase and enrolls by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Enrolling by invitation

Sponsor

Genetix Biotherapeutics Inc.

Intervention

Safety and efficacy assessments

Start date

10/21/2020

Estimated completion

1/1/2038

Countries

United States, France

Related genes

Locations

• Birmingham, United States
• Oakland, United States
• Atlanta, United States
• Chicago, United States
• Bethesda, United States
• Minneapolis, United States
• Hackensack, United States
• New Hyde Park, United States
• New York, United States
• Chapel Hill, United States
• Durham, United States
• Philadelphia, United States
• Charleston, United States
• Houston, United States
• Richmond, United States
• Paris, France

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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