NCT02633943Active, not recruitingPhase 3
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
Source: ClinicalTrials.gov · Last updated 9 Apr 2025
Brief summary (registry, in English)
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical studies (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 this study.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Observational
- Enrollment
- 66 (actual)
- First submitted
- 30 Nov 2015
- Start date
- 1 Jan 2014
- Primary completion date
- 30 Nov 2035
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Diseases
- Thalassaemia
- NCT number
- NCT02633943
- EU CT number
- 2024-513905-29-00
- EudraCT number
- 2013-002245-11
What you commit to
- Study type: Observational · Phase 3
- Participants enrolled: 66
- From 1 Jan 2014 to the primary completion date, 30 Nov 2035
- 15 sites in 8 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- There are no exclusion criteria for this study
Who the study is looking for
- Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s)
- Treated with drug product for therapy of transfusion-dependent β-thalassemia in a bluebird bio-sponsored clinical study
Age in the registry: 0 years to 50 years · Sex: All · Does not accept healthy volunteers
Sites (15)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Sydney | Australia | Unknown status |
| Medizinische Hochschule Hannover Coordinator of this site? Confirm its status | Hanover | Germany | Active, not recruiting |
| Universitaetsklinikum Heidelberg AöR Coordinator of this site? Confirm its status | Heidelberg | Germany | Active, not recruiting |
| Hopital de la Timone Enfants Coordinator of this site? Confirm its status | Marseille | France | Active, not recruiting |
| Hopital Necker Enfants Malades Coordinator of this site? Confirm its status | Paris | France | Active, not recruiting |
| - Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Geniko Nosokomeio Thessalonikis George Papanikolaou Coordinator of this site? Confirm its status | Thessaloniki | Greece | Active, not recruiting |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Rome | Italy | Active, not recruiting |
| - Coordinator of this site? Confirm its status | Bangkok | Thailand | Unknown status |
| - Coordinator of this site? Confirm its status | Bethesda | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Charleston | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| - Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Oakland | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
Related publications
This page is information, not medical advice. Discuss any trial with your doctor.