NCT04628585

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

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

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 85 (estimated)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: All
• Age: 2 Years – 53 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
OTHER	Safety and efficacy assessments	Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Timeline

• Start date: 2020-10-21
• Primary completion: 2038-01-01
• Completion: 2038-01-01
• First posted: 2020-11-13
• Last updated: 2025-03-20

Locations

16 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study