Acute Myeloid Leukemia (AML)Phase 1CCRG 05-001NCT00834002
Dendritic Cell Vaccination for Patients With Acute Myeloid Leukemia in Remission
A Phase 1 study testing injection of antigen-loaded cultured dendritic cells in people with Acute Myeloid Leukemia (AML), run by University Hospital, Antwerp at 1 site in Belgium.
Trial summary
RATIONALE: Vaccines made from a patient's white blood cells (dendritic cells) and a specific leukemia antigen (Wilms tumor antigen-1) may induce an effective immune response to kill residual leukemic cells and/or prevent leukemia relapse. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- injection of antigen-loaded cultured dendritic cells
- Type of treatment
- Vaccines, Injection or infusion
- How many people
- 10 participants
- Where
- 1 siteBelgium
- Study dates
- Mar 2005to Dec 2008
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Tumor type: Acute Myeloid Leukemia (AML) according to the WHO criteria (ea at least 20% blasts in the marrow). All FAB subtypes except M3. Patients with Myelodysplastic Syndrome, category of Refractory Anemia with Excess Blasts (RAEB): RAEB I (WHO: medullary blast count ≤ 10% and a peripheral blast count ≤ 5%) and RAEB II (WHO: medullary blast count > 10% and/or > 5% peripheral blasts) can be included in the study in absence of other non-experimental treatment modalities.
- Extent of disease: remission (partial or complete) or smouldering course. Complete remission (CR) is defined as no blasts in the peripheral blood and no more than 5% blasts in the bone marrow. This definition is related to the hematological remission if it is not specified. Partial remission (PR) is defined as a decrease of at least 50% in the percentage of blasts to 5 to 25% in the bone marrow aspirate. Smouldering course is defined as a relatively low marrow blast count and slowly progressive disease.
- Overexpression of WT1 RNA (>50 copies of WT1 per 1000 copies ABL in bone marrow or >2 copy/1000 copies ABL in peripheral blood) as assessed by quantitative RT-PCR at the time of presentation.
- Prior treatments : Patients must have received at least one prior antileukemic chemotherapeutic regimen and must be more than 1 month past the last treatment and/or 6 months past allogeneic/autologous stem cell transplantation.
- Age: ≥ 18 years
- High risk of relapse because of (and/or)
Show all 17Show fewer
- Age > 60 years (if <60 y, no sibling allotransplant donor available)
- Poor risk cytogenetic or molecular markers at presentation
- Hyperleukocytosis at presentation
- Second complete remission after relapse
- Performance status: WHO PS grade 0-1 (Appendix B)
- Objectively assessable parameters of life expectancy: more than 3 months
- Prior and concomitant associated diseases allowed with the exception of underlying autoimmune disease and positive serology for HIV/HBV/HCV
- No concomitant use of immunosuppressive drugs
- Adequate renal and liver function, i.e. creatinin and bilirubin = 1.2 times the upper limit of normal
- Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial
- Women of child-bearing potential should use adequate contraception prior to study entry and for the duration of study participation
Who cannot join
- Subjects with concurrent additional malignancy (with exception of Non-melanoma skin cancers and carcinoma in situ of the cervix)
- Subjects who are pregnant
- Subjects who have sensitivity to drugs that provide local anesthesia
- Age < 18 years
Trial locations and contacts
1 site in 1 country.
Belgium1 site
Antwerp University Hospital/Center for Cellular Therapy and Regenerative Medicine
Edegem, 2650
About this study
Official title: Wilms Tumor Gene (WT1) mRNA-transfected Autologous Dendritic Cell Vaccination for Patients With Acute Myeloid Leukemia (AML): a Phase I Trial
RATIONALE: Vaccines made from a patient's white blood cells (dendritic cells) and a specific leukemia antigen (Wilms tumor antigen-1) may induce an effective immune response to kill residual leukemic cells and/or prevent leukemia relapse.
PURPOSE: This phase I/II trial is studying the feasibility, safety and efficacy of intradermal mRNA-transfected dendritic cell vaccination therapy in patients with acute myeloid leukemia.
Read the detailed description
Autologous dendritic cell (DC) vaccination is a promising strategy for adjuvant cancer therapy in the setting of minimal residual disease (MRD). We performed a phase I/II trial in patients with acute myeloid leukemia (AML) where patients received intradermal injections of autologous DC loaded with mRNA coding for the Wilms' tumor protein (WT1). WT1 is highly overexpressed in leukemia and the level of WT1 RNA in peripheral blood is a useful biomarker for molecular diagnosis en follow-up in the MRD setting. We want to prospectively monitor WT1 RNA expression in the peripheral blood of vaccinated and non-vaccinated AML patients in order to evaluate its predictive value as a biomarker for relapse and to assess the clinical efficacy of DC vaccination in acute myeloid leukemia patients. We believe, on the basis of already available evidence, that the use of WT1 both as a target for immunotherapy as well as a biomarker not holds promise to assess the efficacy of new experimental therapeutic interventions such as DC vaccination.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- injection of antigen-loaded cultured dendritic cells
- Keywords
- AML in remission
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
injection of antigen-loaded cultured dendritic cellsBiological
intradermal injection of WT1-RNA-electroporated autologous dendritic cell vaccine (therapeutic cell vaccine)
What is being measured
The primary outcome is the main question the study is designed to answer.
acute toxicity of intradermal injections of WT1 mRNA-electroporated autologous dendritic cells
feasibility to generate functional DC vaccines from leukapheresis material from AML patient in remission
Secondary outcomes (1)
T cell immunogenicity of WT1 mRNA-loaded dendritic cell vaccines in AML patients in remission
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2009 (estimated)
- Study startMar 2005
- Primary completionNov 2007
- Study completionDec 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by University Hospital, AntwerpThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Antwerp
- Sponsor type
- University, hospital or other
- Investigators
- Zwi Berneman, MD, PHD, Study director, University Hospital, AntwerpAnn Van de Velde, MD, Principal investigator, University Hospital, AntwerpViggo FI Van Tendeloo, PhD, Principal investigator, University Hospital, Antwerp
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00834002
- Sponsor's protocol ID
- EC 5/6/29
- Acronym
- CCRG 05-001
- Status last verified
- Aug 2009
- First submitted
- 30 Jan 2009
- Last updated
- 9 Feb 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Wilms Tumor Gene (WT1) mRNA-transfected Autologous Dendritic Cell Vaccination for Patients With Acute Myeloid Leukemia (AML): a Phase I Trial
- ClinicalTrials.gov ID
- NCT00834002
- Acronym / sponsor ID
- CCRG 05-001 / EC 5/6/29
- Conditions
- Acute Myeloid Leukemia (AML)
- Keywords
- AML in remission
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University Hospital, AntwerpUniversity, hospital or other
- Study start
- Mar 2005
- Primary completion
- Nov 2007 (actual)
- Study completion
- Dec 2008 (actual)
- First posted
- 2 Feb 2009 (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.
RATIONALE: Vaccines made from a patient's white blood cells (dendritic cells) and a specific leukemia antigen (Wilms tumor antigen-1) may induce an effective immune response to kill residual leukemic cells and/or prevent leukemia relapse.
PURPOSE: This phase I/II trial is studying the feasibility, safety and efficacy of intradermal mRNA-transfected dendritic cell vaccination therapy in patients with acute myeloid leukemia.
Detailed description
Autologous dendritic cell (DC) vaccination is a promising strategy for adjuvant cancer therapy in the setting of minimal residual disease (MRD). We performed a phase I/II trial in patients with acute myeloid leukemia (AML) where patients received intradermal injections of autologous DC loaded with mRNA coding for the Wilms' tumor protein (WT1). WT1 is highly overexpressed in leukemia and the level of WT1 RNA in peripheral blood is a useful biomarker for molecular diagnosis en follow-up in the MRD setting. We want to prospectively monitor WT1 RNA expression in the peripheral blood of vaccinated and non-vaccinated AML patients in order to evaluate its predictive value as a biomarker for relapse and to assess the clinical efficacy of DC vaccination in acute myeloid leukemia patients. We believe, on the basis of already available evidence, that the use of WT1 both as a target for immunotherapy as well as a biomarker not holds promise to assess the efficacy of new experimental therapeutic interventions such as DC vaccination.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Tumor type: Acute Myeloid Leukemia (AML) according to the WHO criteria (ea at least 20% blasts in the marrow). All FAB subtypes except M3. Patients with Myelodysplastic Syndrome, category of Refractory Anemia with Excess Blasts (RAEB): RAEB I (WHO: medullary blast count ≤ 10% and a peripheral blast count ≤ 5%) and RAEB II (WHO: medullary blast count > 10% and/or > 5% peripheral blasts) can be included in the study in absence of other non-experimental treatment modalities.
- Extent of disease: remission (partial or complete) or smouldering course. Complete remission (CR) is defined as no blasts in the peripheral blood and no more than 5% blasts in the bone marrow. This definition is related to the hematological remission if it is not specified. Partial remission (PR) is defined as a decrease of at least 50% in the percentage of blasts to 5 to 25% in the bone marrow aspirate. Smouldering course is defined as a relatively low marrow blast count and slowly progressive disease.
- Overexpression of WT1 RNA (>50 copies of WT1 per 1000 copies ABL in bone marrow or >2 copy/1000 copies ABL in peripheral blood) as assessed by quantitative RT-PCR at the time of presentation.
- Prior treatments : Patients must have received at least one prior antileukemic chemotherapeutic regimen and must be more than 1 month past the last treatment and/or 6 months past allogeneic/autologous stem cell transplantation.
- Age: ≥ 18 years
Key exclusion criteria (4 listed)
- Subjects with concurrent additional malignancy (with exception of Non-melanoma skin cancers and carcinoma in situ of the cervix)
- Subjects who are pregnant
- Subjects who have sensitivity to drugs that provide local anesthesia
- Age < 18 years
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 |
|---|---|---|
| injection of antigen-loaded cultured dendritic cellsintradermal injection of WT1-RNA-electroporated autologous dendritic cell vaccine (therapeutic cell vaccine) | Biological | – |
Primary outcome
acute toxicity of intradermal injections of WT1 mRNA-electroporated autologous dendritic cells
feasibility to generate functional DC vaccines from leukapheresis material from AML patient in remission
1 secondary outcome
T cell immunogenicity of WT1 mRNA-loaded dendritic cell vaccines in AML patients in remission
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 9 Feb 2010.
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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.
Zwi Berneman, MD, PHD study director
University Hospital, Antwerp
Ann Van de Velde, MD principal investigator
University Hospital, Antwerp
Viggo FI Van Tendeloo, PhD principal investigator
University Hospital, Antwerp
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing injection of antigen-loaded cultured dendritic cells in people with Acute Myeloid Leukemia (AML), run by University Hospital, Antwerp at 1 site in Belgium.
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 17 inclusion and 4 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 9 Feb 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. 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 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.
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?
acute toxicity of intradermal injections of WT1 mRNA-electroporated autologous dendritic cells. 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 9 Feb 2010.