Acute Myeloid LeukaemiaPhase 1/2NCT01621724
WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study
A Phase 1/2 study testing WT1 TCR-transduced T cells in people with Acute Myeloid Leukaemia and Chronic Myeloid Leukaemia, run by Cell Medica Ltd at 2 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukaemia, Chronic Myeloid Leukaemia
- What is being tested?
- WT1 TCR-transduced T cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- Apr 2012to May 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- 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.
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- Patients known to be serologically positive for Hepatitis B, C, HTLV-1 Syphilis or HIV.
- 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.
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
University Hospitals Bristol NHS Foundation Trust
Bristol, BS38 3AP
University College London Hospitals NHS Trust
London, NW1 2PG
About this study
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.
Read the 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
- Being tested
- WT1 TCR-transduced T cells
- Keywords
- Gene therapy, WT1 TCR, AML, CML
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WT1 TCR-transduced T cellsGenetic
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
| Study group | Role | What they receive |
|---|---|---|
| Single arm cohort study | Experimental | WT1 TCR-transduced T cells Receives: Genetic: WT1 TCR-transduced T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify organ toxicities and other side effects
Measured: Up to 12 months per patient
Transduction efficiency and TCR expression on TCR-transduced cells
Measured: Up to 12 months per patient
Secondary outcomes (1)
WT1-specific immune responses of TCR-transduced T cells
Measured: Up to 12 months per patient
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2012 (estimated)
- Study startApr 2012
- Primary completionMay 2018
- Study completionMay 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukaemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cell Medica Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University College, London, Cell Therapy Catapult
- Responsible party
- Sponsor
- Investigators
- Emma Morris, Dr, Principal investigator, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01621724
- Sponsor's protocol ID
- D-00272-CT2014001
- Status last verified
- Oct 2018
- First submitted
- 22 Mar 2012
- Last updated
- 2 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study
- ClinicalTrials.gov ID
- NCT01621724
- Acronym / sponsor ID
- D-00272-CT2014001
- Conditions
- Acute Myeloid Leukaemia, Chronic Myeloid Leukaemia
- Keywords
- Gene therapy, WT1 TCR, AML, CML
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Cell Medica LtdIndustry (pharma, biotech, devices)
- Collaborators
- University College, London, Cell Therapy Catapult
- Study start
- Apr 2012
- Primary completion
- May 2018 (actual)
- Study completion
- May 2018 (actual)
- First posted
- 18 Jun 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm cohort study | Experimental | Genetic: WT1 TCR-transduced T cellsWT1 TCR-transduced T cells |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WT1 TCR-transduced T cellsTwo 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 | Genetic | Single arm cohort study |
Primary outcome
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
1 secondary outcome
WT1-specific immune responses of TCR-transduced T cells
Time frame: Up to 12 months per patient
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Emma Morris, Dr principal investigator
University College, London
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing WT1 TCR-transduced T cells in people with Acute Myeloid Leukaemia and Chronic Myeloid Leukaemia, run by Cell Medica Ltd at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identify organ toxicities and other side effects (time frame: Up to 12 months per patient). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2018.