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WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study (WT1 TCR-001)

Phase 1/Phase 2
18 Years
75 Years
Open (Enrolling)
Acute Myeloid Leukaemia, Chronic Myeloid Leukaemia

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Trial Information

WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study (WT1 TCR-001)

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.


Inclusion Criteria:

- Age ≥ 18 years and ≤ 75 years.

- Life expectancy ≥ 16 weeks (4 months).

- World Health Organisation (WHO) performance status of 0-2

- HLA A*0201 positive

- Completed previous course of chemotherapy ≥ 4 weeks prior to commencing the initial
phase of the trial (leucapheresis for collection of patient PBMC).

- Peripheral blood total lymphocyte count > 0.5x109/L.

- Informed consent in writing and ability to co-operate with treatment and follow up.

- Willing, able and available for collection of PBMC/ T cells by leucapheresis.

- Hepatitis B and C, HTLV-1, Syphilis, HIV negative.

- Free from serious concurrent illness.

- Female patients of child-bearing age must have a negative pregnancy test and agree to
use reliable contraceptive methods for the duration of the therapy and for 6 months

- Male patients must agree to use appropriate medically approved contraception during
the trial and for six months afterwards.

- Haematological and Biochemical Indices:

- Haemoglobin (Hb) ≥ 7.0 g/dl; neutrophils ≥ 0.2 x 109/L; total lymphocytes > 0.5 x
109/L; platelets (Plts) ≥ 40 x 109/L

- serum bilirubin, Alanine amino-transferase (ALT) and/or aspartate amino transferase
(AST) < 3 x upper normal limit

- calculated creatinine clearance ≥ 30 ml/min (uncorrected value) or isotope clearance
measurement ≥ 30ml/min

Further disease specific inclusion criteria are detailed in Protocol

Exclusion Criteria:

- Age < 18 years or > 75 years.

- Patients should not receive concurrent systemic corticosteroids whilst on the study.

- Within three months of having received fludarabine (at time of leucapheresis).

- Major thoracic and/or abdominal surgery in the preceding three to four weeks from
which the patient has not yet recovered.

- Patients who are high medical risks because of non-malignant systemic disease, as
well as those with active uncontrolled infection.

- Patients with any other condition, which in the Investigator's opinion would not make
the patient a good candidate for the clinical trial.

- Patients known to be serologically positive for Hepatitis B, C, HTLV-1 Syphilis or

- Concurrent congestive heart failure or prior history of New York Heart Association
(NYHA) class III/ IV cardiac disease

- Positive pregnancy test or reluctance to use contraception.

- Pregnant and lactating women are excluded.

- History of Severe Allergy.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Identify organ toxicities and other side effects

Outcome Time Frame:

Up to 12 months per patient

Safety Issue:


Principal Investigator

Emma Morris, Dr

Investigator Role:

Principal Investigator

Investigator Affiliation:

Royal Free and University College London


United Kingdom: Medicines and Healthcare Products Regulatory Agency

Study ID:

UCL 06/154



Start Date:

April 2012

Completion Date:

April 2016

Related Keywords:

  • Acute Myeloid Leukaemia
  • Chronic Myeloid Leukaemia
  • Gene therapy
  • WT1 TCR
  • AML
  • CML
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive