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NCT04293185 · ClinicalTrials.gov registry record · Phase 3
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
A Phase 3 study of Sickle Cell Disease, sponsored by Genetix Biotherapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 35
- Enrollment target
- 9
- Study locations
NCT04293185: Active Phase 3 study of Sickle Cell Disease, sponsored by Genetix Biotherapeutics.
NCT04293185 is a Phase 3 study of Sickle Cell Disease that is active but no longer recruiting, run by Genetix Biotherapeutics. The registered enrollment target is 35 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (88% lower). The trial reports 9 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04293185, a Phase 3 study of Sickle Cell Disease, is active but no longer recruiting, sponsored by Genetix Biotherapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 35 participants
- Enrollment target
- 9
- Study locations
Study Summary
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).
Primary Outcome
Proportion of subjects achieving complete resolution of VOEs between 6 months and 18 months after drug product infusion
Conditions Studied
Interventions
- GENETIC bb1111
Study Locations (9)
Alabama
- University of Alabama - Birmingham
District of Columbia
- Children's National Hospital - Washington D.C.
Massachusetts
- Tufts Medical Center - Boston
Minnesota
- University of Minnesota - Minneapolis
New Jersey
- Hackensack University Medical Center - Hackensack
New York
- Montefiore Medical Center - The Bronx
North Carolina
- Duke University - Durham
Texas
- Baylor College of Medicine/Texas Children's Hospital - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2020-02-14 |
| Est. Completion | 2027-11 |
| Phase | Phase 3 |
What the registry record for NCT04293185 still lists
NCT04293185 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (88% lower).
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which bb1111 is the first listed.
NCT04293185 lists 9 locations in 9 states (Alabama, District of Columbia, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT04293185 about?
NCT04293185 is a clinical study titled "A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in 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 ...
What is the current status of trial NCT04293185?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 35 participants. The study started on 2020-02-14. Estimated completion is 2027-11.
What conditions does trial NCT04293185 study?
This clinical trial studies the following conditions: Sickle Cell Disease.
What interventions are being tested in trial NCT04293185?
The interventions under investigation include: bb1111 (GENETIC).
Who is sponsoring clinical trial NCT04293185?
This trial is sponsored by Genetix Biotherapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04293185 being conducted?
This trial has 9 study locations across Alabama, District of Columbia, Massachusetts, Minnesota, New Jersey. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04293185's enrollment target sits among peer trials
35 134th of 213 higher than 80 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease 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.
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