Ignite Creation Date:
2024-05-06 @ 7:32 AM
Last Modification Date:
2024-10-26 @ 11:49 AM
Study NCT ID:
NCT02550535
Status:
COMPLETED
Last Update Posted:
2018-10-02
First Post:
2015-04-28
Brief Title:
A Phase III Study of Gene-modified WT1 TCR Therapy in MDS AML Patients
Sponsor:
Cell Medica Ltd
Organization:
Cell Medica Ltd
Study Overview
Official Title:
Single Arm Phase III Study of the Safety and Efficacy of Gene-modified WT1 TCR Therapy in Patients With Myelodysplastic Syndrome MDS or Acute Myeloid Leukaemia AML Who Have Failed to Achieve or Maintain an IWG Response Following Hypomethylating Agent Therapy
Status:
COMPLETED
Status Verified Date:
2018-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase III trial to determine safety clinical efficacy and feasibility of a gene-modified WT1 TCR therapy in patients with myelodysplastic syndrome MDS and acute myeloid leukaemia AML
Patients 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 Wilms tumour 1 which is present at abnormally high levels on the surface of myelodysplastic and leukaemic cells
In this trial approximately 25 participants with an Human Leukocyte Antigen A2 HLA-A0201 tissue type who have failed to achieve or maintain an IWG defined response following hypomethylating agent therapy will be recruited
Patients 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 Wilms tumour 1 which is present at abnormally high levels on the surface of myelodysplastic and leukaemic cells
In this trial approximately 25 participants with an Human Leukocyte Antigen A2 HLA-A0201 tissue type who have failed to achieve or maintain an IWG defined response following hypomethylating agent therapy will be recruited
Detailed Description:
This is a Phase III trial to determine safety clinical efficacy and feasibility of a gene-modified WT1 TCR therapy Following provision of informed consent each subject will undergo screening assessments including HLA-A0201 screening if not already documented and a bone marrow aspiratebiopsy BMA to determine subject eligibility for the trial
Subjects will undergo leukapheresis within 14 days of screening
Once successful manufacture of the WT1 TCR-transduced T cells has been confirmed by the Sponsor each subject will be administered a lymphodepletive conditioning regimen for five days consisting of fludarabine x 5 days 30mgm2 intravenous iv and methylprednisolone x 1 day 500 mg iv Upon completion of the conditioning regimen subjects will receive an infusion of WT1 TCR-transduced T cells of 2 x 107kg followed by daily IL-2 subcutaneous injections 1 x 106 unitsm2 subcutaneous sc od for 5 days
If an IWG response has not been reported one or more criteria met at 3 months post-infusion then if agreed by both the Investigator and Sponsor the subject will be offered to have a repeat infusion of WT1 TCR-transduced T cells
Following infusion subjects will enter an intensive period of out-patient follow-up to observe them for any acute complications and toxicities Subjects will then be followed monthly in the clinic for the first 6 months for routine safety and clinical evaluations including disease response evaluations All subjects will be followed-up for a minimum of 12 months
Subjects will undergo leukapheresis within 14 days of screening
Once successful manufacture of the WT1 TCR-transduced T cells has been confirmed by the Sponsor each subject will be administered a lymphodepletive conditioning regimen for five days consisting of fludarabine x 5 days 30mgm2 intravenous iv and methylprednisolone x 1 day 500 mg iv Upon completion of the conditioning regimen subjects will receive an infusion of WT1 TCR-transduced T cells of 2 x 107kg followed by daily IL-2 subcutaneous injections 1 x 106 unitsm2 subcutaneous sc od for 5 days
If an IWG response has not been reported one or more criteria met at 3 months post-infusion then if agreed by both the Investigator and Sponsor the subject will be offered to have a repeat infusion of WT1 TCR-transduced T cells
Following infusion subjects will enter an intensive period of out-patient follow-up to observe them for any acute complications and toxicities Subjects will then be followed monthly in the clinic for the first 6 months for routine safety and clinical evaluations including disease response evaluations All subjects will be followed-up for a minimum of 12 months
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None