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Clinical trialNCT04293185Active, not recruitingPhase 3Interventional

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

SponsorGenetix Biotherapeutics Inc.Enrollment35Sickle Cell Disease
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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).
clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

9 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • University of Alabama

    Birmingham, Alabama, United States

  • Children'S National Hospital

    Washington D.C., District of Columbia, United States

  • Tufts Medical Center

    Boston, Massachusetts, United States

  • University of Minnesota

    Minneapolis, Minnesota, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, United States

  • Montefiore Medical Center

    The Bronx, New York, United States

  • Duke University

    Durham, North Carolina, United States

  • Baylor College of Medicine/texas Children'S Hospital

    Houston, Texas, United States

  • Virginia Commonwealth University (vcu)

    Richmond, Virginia, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
14 Feb 2020
Primary completion
31 May 2027
Completion
30 Nov 2027
Last update posted
10 Dec 2024
Registered enrollment
35
Registry id
NCT04293185
clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 4 Nov 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.