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Report for Clinical Trial NCT02906202

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 23 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

NCT02906202 Beta-Thalassemia
MeSH: Thalassemia beta-Thalassemia

1 Interventions

Name: LentiGlobin BB305 Drug Product

Description: LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.

Type: Genetic

LentiGlobin BB305 Drug Product


Primary Outcomes

Description: TI is defined as a weighted average Hb ≥9g/dL without any RBC transfusions for a continuous period of ≥12 months at any time during the study after drug product infusion.

Measure: The proportion of treated subjects who meet the definition of "transfusion independence" (TI).

Time: 12 - 24 months post-transplant

Secondary Outcomes

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

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: 24 months post-transplant

Purpose: Treatment

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, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age. --- T87Q ---



HPO Nodes