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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
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).
Details
| Lead sponsor | Genetix Biotherapeutics Inc. |
|---|---|
| Phase | Phase 3 |
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 35 |
| Start date | 2020-02-14 |
| Completion | 2027-11 |
Conditions
- Sickle Cell Disease
Interventions
- bb1111
Primary outcomes
- VOE-CR — 6-18 months post-transplant
Proportion of subjects achieving complete resolution of VOEs between 6 months and 18 months after drug product infusion
Countries
United States