This study is accepting new patients by invitation only

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

a study on Sickle Cell Anemia Anemia

Summary

Eligibility
for people ages 2-53 (full criteria)
Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Mark Walters (ucsf)
Headshot of Mark Walters
Mark Walters

Description

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.

Official Title

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

Keywords

Sickle Cell Disease, Hematopoietic Stem Cells, Sickle Cell Anemia, Safety, Safety and efficacy assessments

Eligibility

You can join if…

Open to people ages 2-53

  • 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

You CAN'T join if...

  • There are no exclusion criteria for this study

Locations

  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States
  • Baylor College of Medicine
    Houston Texas 77030 United States

Lead Scientist at University of California Health

  • Mark Walters (ucsf)
    The over-arching goal of my research career has been to develop and expand curative therapies for hemoglobin disorders in particular and non-malignant hematopoietic disorders more broadly. I have focused on genome editing approaches most recently, which rely upon modification of autologous hematopoietic cells to elicit a curative effect.

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Genetix Biotherapeutics Inc.
ID
NCT04628585
Study Type
Observational
Participants
Expecting 85 study participants
Last Updated