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NCT01745120 · ClinicalTrials.gov registry record · Phase 1
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
A Phase 1 study, sponsored by Genetix Biotherapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 19
- Enrollment target
NCT01745120: Completed Phase 1 study, sponsored by Genetix Biotherapeutics.
NCT01745120 is a Phase 1 study that has completed, run by Genetix Biotherapeutics. The registered enrollment target is 19 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (68% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01745120, a Phase 1 study, has completed, sponsored by Genetix Biotherapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 19 participants
- Enrollment target
Study Summary
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product \[autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene\].
Primary Outcome
Percentage of participants with sustained production of \>=2.0 grams per deciliter (g/dL) of hemoglobin A (HbA) containing βA-T87Q-globin (HbAT87Q) for 6 months (Month 18 to Month 24) was reported.
Interventions
- GENETIC LentiGlobin BB305 Drug Product
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2013-08 |
| Est. Completion | 2018-02-21 |
| Phase | Phase 1 |
What the finished NCT01745120 record still lists
NCT01745120 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 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (68% lower).
The record links to 0 conditions, and to 1 intervention - of which LentiGlobin BB305 Drug Product is the first listed.
NCT01745120 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01745120 about?
NCT01745120 is a clinical study titled "A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants". This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transpl...
What is the current status of trial NCT01745120?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 19 participants. The study started on 2013-08. Estimated completion is 2018-02-21.
What interventions are being tested in trial NCT01745120?
The interventions under investigation include: LentiGlobin BB305 Drug Product (GENETIC).
Who is sponsoring clinical trial NCT01745120?
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 NCT01745120's enrollment target sits among peer trials
19 1716th of 2000 higher than 281 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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