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

NCT04628585 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 85

Last updated 2025-03-20

No results posted yet for this study

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

Interventions

OTHER

Safety and efficacy assessments

Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Sponsors & Collaborators

  • Genetix Biotherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Anjulika Chawla, MD · bluebird bio, Inc.

Eligibility

Min Age
2 Years
Max Age
53 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-21
Primary Completion
2038-01-31
Completion
2038-01-31
FDA Drug
Yes

Countries

  • United States
  • France

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04628585 on ClinicalTrials.gov