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NCT03207009 · ClinicalTrials.gov registry record · Phase 3
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
A Phase 3 study, sponsored by Genetix Biotherapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 19
- Enrollment target
NCT03207009: Completed Phase 3 study, sponsored by Genetix Biotherapeutics.
NCT03207009 is a Phase 3 study that has completed, run by Genetix Biotherapeutics. The registered enrollment target is 19 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03207009, a Phase 3 study, has completed, sponsored by Genetix Biotherapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 19 participants
- Enrollment target
Study Summary
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (\<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
Primary Outcome
TI was defined as a weighted average hemoglobin (Hb) \>= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of \>= 12 months at any time during the study after drug product infusion.
Interventions
- GENETIC LentiGlobin BB305 Drug Product
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2017-06-08 |
| Est. Completion | 2022-11-15 |
| Phase | Phase 3 |
What the finished NCT03207009 record still lists
NCT03207009 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 19 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which LentiGlobin BB305 Drug Product is the first listed.
NCT03207009 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03207009 about?
NCT03207009 is a clinical study titled "A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia". This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (\<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety o...
What is the current status of trial NCT03207009?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 19 participants. The study started on 2017-06-08. Estimated completion is 2022-11-15.
What interventions are being tested in trial NCT03207009?
The interventions under investigation include: LentiGlobin BB305 Drug Product (GENETIC).
Who is sponsoring clinical trial NCT03207009?
This trial is sponsored by Genetix Biotherapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03207009's enrollment target sits among peer trials
19 1969th of 2000 higher than 31 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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