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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Genetix Biotherapeutics Inc.
Study ID
NCT04628585
Status
Enrolling By Invitation

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 53 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Oct 21, 2020
Status verified
Mar 2025
Primary completion
Jan 31, 2038
Completion
Jan 31, 2038

Study Design

Enrollment
85 participants (estimated)

Arms

  • Arm: Subjects with sickle-cell disease
    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

Primary Outcome Measure

Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) [ Time Frame: Through 15 years post-drug product infusion ]

Locations (15)

FacilityCityStateZIPSite coordinators
University of AlabamaBirminghamAlabama35294-
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609-
Children's Healthcare of AtlantaAtlantaGeorgia30322-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-2991-
Warren Grant Magnuson Clinical CenterBethesdaMaryland20892-
University of Minnesota Masonic Children's HospitalMinneapolisMinnesota55454-
Hackensack University Medical CenterHackensackNew Jersey07601-
Cohen Children's Medical CenterNew Hyde ParkNew York11040-
Columbia University Medical CenterNew YorkNew York10032-
The University of North CarolinaChapel HillNorth Carolina27514-
Duke University Medical CenterDurhamNorth Carolina27705-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
Medical University of South CarolinaCharlestonSouth Carolina29425-
Baylor College of MedicineHoustonTexas77030-
Virginia Commonwealth UniversityRichmondVirginia23298-

Find similar trials in Birmingham, AL

By condition
Sickle cell disease
By research site
University of Alabama· Birmingham, ALUCSF Benioff Children's Hospital Oakland· Oakland, CAChildren's Healthcare of Atlanta· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILWarren Grant Magnuson Clinical Center· Bethesda, MDUniversity of Minnesota Masonic Children's Hospital· Minneapolis, MN

Related Studies