Clinical trialNCT04628585Enrolling, by, invitationObservational

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

SponsorGenetix Biotherapeutics Inc.Enrollment85Sickle Cell Disease

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Anjulika Chawla, MD
bluebird bio, Inc.
Study Director
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

16 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

France

Hospital Necker
Paris, France

United States

University of Alabama
Birmingham, Alabama, United States
Ucsf Benioff Children'S Hospital Oakland
Oakland, California, United States
Children'S Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
Warren Grant Magnuson Clinical Center
Bethesda, Maryland, United States
University of Minnesota Masonic Children'S Hospital
Minneapolis, Minnesota, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Cohen Children'S Medical Center
New Hyde Park, New York, United States
Columbia University Medical Center
New York, New York, United States
the University of North Carolina
Chapel Hill, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Baylor College of Medicine
Houston, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 21 Oct 2020
Primary completion: 31 Jan 2038
Completion: 31 Jan 2038
Last update posted: 20 Mar 2025
Registered enrollment: 85
Registry id: NCT04628585

Cross-registrations

EU-CTR / CTIS (EudraCT)2024-513901-30-00EU-CTR / CTIS (EudraCT)2019-004266-18

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 4 Nov 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.