NCT01621724

WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Cell Medica Ltd · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

WT1 TCR gene therapy is a new treatment for acute myeloid leukaemia and chronic myeloid leukaemia. Patient's white blood cells (T cells) are modified to specifically fight the leukaemia cells by transferring a gene into the T cells, which allows them to recognize fragments of a protein called WT1. This protein is present on the surface of leukaemia cells at very high levels. The gene transferred to the T cells enables them to make a new T cell receptor (TCR), which will allow them to attack leukaemia cells with high levels of WT1 on their surface. Using this form of gene therapy the investigators can convert some of the patient's immune system's own T cells into T cells that the investigators hope will be much more effective at recognizing and killing leukaemia cells.

Detailed description

This trial concerns a novel approach to generating leukaemia antigen-specific T cells for adoptive cellular therapy in HLA-A\*0201 patients with acute myeloid leukaemia (AML) and chronic myeloid leukaemia (CML) In this study, patient T cells will be gene-modified using a GMP grade retroviral vector containing the genes for a WT1-specific, HLA-A2-restricted T cell receptor. This ex vivo gene therapy will generate T cells expressing the WT1-specific TCR and thus able to recognise WT1-expressing target cells. The autologous Cys1 WT1 TCR-transduced T cells will be re-infused back into adult leukaemia patients following lymphodepleting conditioning.

Conditions

• Acute Myeloid Leukaemia
• Chronic Myeloid Leukaemia

Interventions

Type	Name	Description
GENETIC	WT1 TCR-transduced T cells	Two patient cohorts: Cohort 1 (up to 6 patients) = ≤ 2 x 107/kg WT1 TCR-transduced T cells Cohort 2 (12 patients)= ≤ 108/kg WT1 TCR-transduced T cells

Timeline

• Start date: 2012-04-01
• Primary completion: 2018-05-01
• Completion: 2018-05-01
• First posted: 2012-06-18
• Last updated: 2018-10-02

Locations

2 sites across 1 country: United Kingdom