Who is eligible for clinical trial NCT04293185?

Participants must meet protocol criteria for Sickle Cell Disease. Confirm exact inclusion and exclusion requirements with the study coordinator.

Does clinical trial NCT04293185 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2024-12-05

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease (NCT04293185)

🔵 Active, Not Recruiting Phase3 📋 Clinical Research Protocol Sponsor: Genetix Biotherapeutics Inc.

🩺 Plain-English Summary (8th-Grade Level)

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).

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (9 items) and avoid the listed exclusions (21 items).

Location: University of Alabama — Birmingham, Alabama

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype.
Be ≥2 and ≤50 years of age at time of consent.
Weigh a minimum of 6 kg.
Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (\<16 years of age).
Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history.
In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined VOEs in the 24 months prior to informed consent.
Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment).
Female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion.
Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).

Usually NOT eligible if any of the following apply:

Exclusion Criteria:
Subjects for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available.
Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) for subjects ≤ 16 years of age (e.g. TCD velocity \>200 cm/sec) requiring ongoing chronic transfusions, a Screening TCD or TCDI velocity \> 200 cm/sec (central read), a Screening MRA showing \> 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read).
Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1), active syphilis.
Clinically significant, active bacterial, viral, fungal, or parasitic infection
Advanced liver disease, such as
clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
Inadequate bone marrow function, as defined by an absolute neutrophil count of \<1×10\^9/L (\<0.5×10\^9/L for subjects on hydroxyurea treatment) or a platelet count \<100×10\^9/L.
Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
Unable to receive pRBC transfusion.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (9 Sites)

University of Alabama — Birmingham, Alabama
Children's National Hospital — Washington D.C., District of Columbia
Tufts Medical Center — Boston, Massachusetts
University of Minnesota — Minneapolis, Minnesota
Hackensack University Medical Center — Hackensack, New Jersey
Montefiore Medical Center — The Bronx, New York
Duke University — Durham, North Carolina
Baylor College of Medicine/Texas Children's Hospital — Houston, Texas
Virginia Commonwealth University (VCU) — Richmond, Virginia

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

🏛️ View Official NIH Record ↗