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Report for Clinical Trial NCT03207009
Developed by Shray Alag, 2020.
SNP Clinical Trial Gene
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 subjects ≤50
years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110,
or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of
autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug
Product.
1 Interventions
Name: LentiGlobin BB305 Drug Product
Description: LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.
Type: Genetic
Group Labels: 1 LentiGlobin BB305 Drug Product
Primary Outcomes
Description: TR is defined as demonstration of reduction in volume of RBC transfusion requirements (in mL/kg) in the post-treatment time period of Months 12 to 24 compared to the average annual transfusion requirement in the 24 months prior to enrollment.
Measure: The proportion of subjects who meet the definition of "transfusion reduction" (TR).
Time: 12-24 months post-transplant
Secondary Outcomes
Measure: The proportion of treated subjects who meet the definition of "transfusion independence" (TI). TI is defined as Hb ≥9g/dL without any RBC transfusions for a continuous period of ≥12 months at any time during the study after drug product infusion. Time: 12-24 months post-transplant
Measure: Percentage of subjects with a reduction in the mL/kg RBC transfused from Month 12 through Month 24 after drug product infusion of at least 50% compared to the average annual RBC transfusion requirement during the 2 years prior to enrollment. Time: 12-24 months post-transplant
Measure: Engraftment defined as an absolute neutrophil count ≥500 cells/µL for 3 consecutive days Time: 24 months post-transplant
Measure: Detection of vector-derived replication competent lentivirus (RCL) using a RCL screening assay Time: 24 months post-transplant
Measure: Frequency of events of insertional mutagenesis leading to clonal dominance or leukemia. Time: 24 months post-transplant
Measure: Frequency of clinical adverse events Time: 24 months post-transplant
Purpose: Treatment
Allocation: N/A
Single Group Assignment
There is one SNP
SNPs
1 T87Q
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age. --- T87Q ---