- Investigational drugs: Gene modified WT1 (Wilms’ Tumor 1) TCR therapy - Patient's white blood cells (T cells) will be modified by transferring a gene which enables them to make a new T cell receptor (TCR) that can recognize fragments of a protein called WT1 which is present at abnormally high levels on the surface of myelodysplastic and leukaemic cells.
- For what level of MDS severity risk: Intermediate – 2 or High Risk
- What subtype of MDS: High (> 10% bone marrow blasts); also Acute Myeloid Leukaemia
- Aims and benefits:
- Safety and signs of efficacy of the WT1 TCR therapy in a carefully defined group of patients (those who have failed to respond to Azacitidine but do not have rapid disease progression).
- Aims to assess blood count improvement, overall remission rate, marrow remission, cytogenetic response, reduction in size of molecular clone, AML transformation, progression free, event free and overall survival.
- Basic inclusion criteria:
- Aged ≥18 yrs
- ECOG performance status 0, 1 or 2
- Diagnosis of MDS (Intermediate – 2 or High Risk) or AML
- Basic exclusion criteria:
- Patients who are in complete remission, partial remission, marrow complete remission or have cytogenetic response, or haematological improvement or molecular response following Azacitidine treatment
- CMML patients who have a white blood cell count > 13 x 109/L
Acute promyelocytic leukaemia (FAB M3 Classification)
- Active malignancy, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast
- Known history of Human Immunodeficiency Virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV) or positive for Human T-Lymphocyte Virus (HTLV) or Syphilis.
- Active auto-immune disease
- Kidney impairment requiring dialysis
- Trial sites/locations and name of physician in charge of trial:
Aberdeen Royal Infirmary, Aberdeen; University College London Hospitals NHS Trust, London.
University Hospitals Bristol NHS Foundation Trust, Bristol;
Western General Hospital, Edinburgh;
The Leeds Teaching Hospitals NHS Trust, Leeds;
University College London Hospitals NHS Trust, London
- More information:
Please read information and always discuss trial information with your own physician.