WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study
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|ClinicalTrials.gov Identifier: NCT01621724|
Recruitment Status : Recruiting
First Posted : June 18, 2012
Last Update Posted : April 19, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukaemia Chronic Myeloid Leukaemia||Genetic: WT1 TCR-transduced T cells||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||18 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study|
|Study Start Date :||April 2012|
|Estimated Primary Completion Date :||May 2019|
|Estimated Study Completion Date :||October 2019|
Experimental: Single arm cohort study
WT1 TCR-transduced T cells
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
- Identify organ toxicities and other side effects [ Time Frame: Up to 12 months per patient ]
- Transduction efficiency and TCR expression on TCR-transduced cells [ Time Frame: Up to 12 months per patient ]
- WT1-specific immune responses of TCR-transduced T cells [ Time Frame: Up to 12 months per patient ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01621724
|University Hospitals Bristol NHS Foundation Trust||Recruiting|
|Bristol, United Kingdom, BS38 3AP|
|Contact: Stephen Robinson, Dr 0117 342 8523 Stephen.Robinson@ubht.nhs.uk|
|Principal Investigator: Stephen Robinson, Dr|
|University College London Hospitals NHS Trust||Recruiting|
|London, United Kingdom, NW1 2PG|
|Contact: Emma Morris, Dr 0845 155 5000 ext 9712 email@example.com|
|Principal Investigator: Emma Morris, Dr|
|Principal Investigator:||Emma Morris, Dr||University College, London|