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A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

Sickle cell anemia
All genders
person-wave 2-50
Recruiting now
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Principal Investigator: Andreas Klein, MD

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 ages with SCD. The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using LentiGlobin BB305 Drug Product, an autologous CD34+ cell-enriched population that contains hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector (LVV) encoding beta A-T87Q-globin gene, suspended in cryopreservation solution in the final immediate container for the intended medical use.

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Robin Kallfelz

Study details

Inclusion Criteria
  • Have a diagnosis of SCD, with either betaS/betaS, beta S/beta-0 or beta S/beta + genotype
  • Be > 2 and <50 years of age at time of consent.
  • Weigh a minimum of 6 kg and reasonably be anticipated to be able to provide at least the minimum number of cells require to initiate the manufacturing process.
Exclusion Criteria
  • Applicable to subjects <18 years of age only: availability of a willing, matched human leukocyte antigen (HLA)-identical sibling hematopoietic cell donor
  • Severe cerebral vasculopathy, defined by any history of: overt ischemic or hemorrhagic stroke, abnormal transcranial Doppler (>200 cm/sec based on central read) requiring chronic transfusion, occlusion, or stenosis in the circle of Willis, or presence of Moyamoya disease (central read of brain imaging MRI [or computed tomography (CT) scan if MRI not feasible to perform]). Subjects with radiologic evidence of silent infarction in the absence of any of the above criteria would still be eligible.
  • 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) or -2 (HTLV-2), active syphilis. Not that subjects who have been vaccinated against hepatitis B [hepatitis B surface B antibody-positive] are eligible. Subjects with past exposure to HBV [HBc Ab positive and/or HBe Ab positive] are also eligible for the study provided they are negative for HBV DNA. Subjects who are positive for anti-hepatitis C antibody are eligible as long as they have an undetectable hepatitis C viral load. Where clinically and/or regionally indicated, other tests may be performed, in which case relevant positive results suggesting active infection would exclude the subject from participating, depending on regional guidelines: for example, malaria, tuberculosis, active toxoplasmosis, Trypanosoma cruzi, or West Nile Virus.
Study Requirements

Time between Screening and drug product infusion will be variable and is estimated generally to be between 3 to 5 month (e.g., up to 3 months between Screening and Mobilization, followed by approximately 2 months before drug product infusion). Thereafter the subject is planned to remain on study for approximately 24 months. Eligible subject will then be asked to enroll in a separate long-term follow-up study until approximately 15 years post drug product infusion.

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