A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
Public ClinicalTrials.gov record NCT03207009. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age
Study identification
- NCT ID
- NCT03207009
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Enrollment
- 19 participants
Conditions and interventions
Conditions
Interventions
- LentiGlobin BB305 Drug Product Genetic
Genetic
Eligibility (public fields only)
- Age range
- 0 Years to 50 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 7, 2017
- Primary completion
- Nov 14, 2022
- Completion
- Nov 14, 2022
- Last update posted
- Mar 6, 2024
2017 – 2022
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 6 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03207009, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 6, 2024 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03207009 live on ClinicalTrials.gov.