A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
Public ClinicalTrials.gov record NCT04293185. 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 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the BB305 Lentiviral Vector in Subjects With Sickle Cell Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).
Study identification
- NCT ID
- NCT04293185
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 35 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 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
- Feb 14, 2020 (Actual)
- Primary completion
- May 2027 (Estimated)
- Completion
- Nov 2027 (Estimated)
- Last update posted
- Dec 10, 2024
2020 – 2027
United States locations
- U.S. sites
- 9
- U.S. states
- 9
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35233 | — |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | — |
| Tufts Medical Center | Boston | Massachusetts | 02111 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Montefiore Medical Center | The Bronx | New York | 10467 | — |
| Duke University | Durham | North Carolina | 27705 | — |
| Baylor College of Medicine/Texas Children's Hospital | Houston | Texas | 77030 | — |
| Virginia Commonwealth University (VCU) | Richmond | Virginia | 23219 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04293185, 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 Dec 10, 2024 · Synced Oct 19, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04293185 live on ClinicalTrials.gov.