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MT2019-06: 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.
Status: Recruiting
Evaluate the efficacy of treatment with bb1111 (also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD).
Age: Not specified
Healthy Volunteers:
Inclusion Criteria:
• must be 2 to 50 years old
• diagnosis of Sickle Cell Disease
• weigh a minimum of 6 kg (13.2 pounds)
• treated and followed for at least the past 24 months
• experienced at least 4 protocol-defined VOEs in the past 24 months
• experienced HU failure at any point in the past or must have intolerance to HU
• female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from starting the study to at least 6 months after drug product infusion.
Exclusion Criteria:
• if allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available
• unable to receive a transfusion
• prior allogeneic transplant or gene therapy
• prior or current malignancy or immunodeficiency disorder, except cured tumors such as squamous cell carcinoma of the skin
• women who are pregnant or breast feeding
• additional exclusion criteria (study staff will review)
Conditions:
Blood Disorders
Keywords:
SCD, Sickle Cell Disease
Contact(s): Danielle Jin - zhuxx003@umn.edu
Principal Investigator: Ashish Gupta
Phase: PHASE3
IRB Number: STUDY00006923
System ID: 25197
See this study on ClinicalTrials.gov