Cellular Immunotherapy for Patients With High Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia
Primary Purpose
Myelodysplastic Syndromes, Acute Myeloid Leukemia
Status
Unknown status
Phase
Phase 1
Locations
Brazil
Study Type
Interventional
Intervention
Autologous dendritic cells electroporated with WT1 mRNA
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndromes
Eligibility Criteria
Inclusion Criteria:
- Individuals between the ages of 18 and 70
- High-risk myelodysplasia (AREB 1 or AREB 2 subtypes) and Acute Myeloid Leukemia
- Minimum interval of 30 days between the last cycle of chemotherapy (when it occurs) and start of immunotherapy
- Performance status between 0 and 3 on the WHO (World Health Organization)-ECOG (Eastern Cooperative Oncology Group) scale
- Calculated creatinine clearance> 30 ml / min using the Cockcroft-Gault formula
- Total bilirubin less than or equal to twice the lower limit of the normal range in the institution and aspartate aminotransferase (AST) less than or equal to twice the upper limit of normal
- Absence of blasts in peripheral blood
- Leukocyte count greater than 3000 cells / mm3, hemoglobin greater than 9.0 g / dl and platelets greater than 70,000 platelets / mm3, if possible. (If the patient does not meet these criteria for apheresis, the possibility of transfusion of blood components after leukapheresis will be proposed and the patient should sign a specific term of science on the possibility of transfusion)
- Normal cardiac evaluation
- Negative serologies for hepatitis B and C viruses and HIV
- Written informed consent form signed before entering the study
Exclusion Criteria:
- Does not meet any of the requirements of the inclusion criteria
- Low risk myelodysplasia by IPSS (International Prognostic Scoring System) or WPSS (WHO adapted Prognostic Scoring System) scores
- Individuals with a history of any previous neoplasia, except those with prolonged clinical remission (more than 5 years) of non-melanoma skin cancers and cervical cancer in situ
- Pregnant or lactating women
- Previous immunotherapy or biological therapy
Sites / Locations
- Hematology and Transfusion Medicine Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Patients
Arm Description
High Risk Myelodysplastic Syndromes or Acute Myeloid Leukemia
Outcomes
Primary Outcome Measures
Disease free survival
Secondary Outcome Measures
Full Information
NCT ID
NCT03083054
First Posted
March 12, 2017
Last Updated
October 9, 2020
Sponsor
University of Campinas, Brazil
1. Study Identification
Unique Protocol Identification Number
NCT03083054
Brief Title
Cellular Immunotherapy for Patients With High Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia
Official Title
Cellular Immunotherapy as a Treatment Option for Patients With High Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Unknown status
Study Start Date
August 2016 (Actual)
Primary Completion Date
July 12, 2020 (Actual)
Study Completion Date
July 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Campinas, Brazil
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The main objective of this work is to conduct a clinical study for the development and application of a vaccine with autologous dendritic cells submitted to electroporation with Wilm's tumor 1 (WT1) messenger ribonucleic acid (mRNA), as an adjuvant treatment of high-risk Myelodysplastic Syndromes and Acute Myeloid Leukemia, aiming to delay the progression of the disease or its relapse and increase overall and event-free survival.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes, Acute Myeloid Leukemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Patients
Arm Type
Experimental
Arm Description
High Risk Myelodysplastic Syndromes or Acute Myeloid Leukemia
Intervention Type
Biological
Intervention Name(s)
Autologous dendritic cells electroporated with WT1 mRNA
Intervention Description
Production and application of autologous dendritic cells vaccines, 4 doses, biweekly
Primary Outcome Measure Information:
Title
Disease free survival
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Individuals between the ages of 18 and 70
High-risk myelodysplasia (AREB 1 or AREB 2 subtypes) and Acute Myeloid Leukemia
Minimum interval of 30 days between the last cycle of chemotherapy (when it occurs) and start of immunotherapy
Performance status between 0 and 3 on the WHO (World Health Organization)-ECOG (Eastern Cooperative Oncology Group) scale
Calculated creatinine clearance> 30 ml / min using the Cockcroft-Gault formula
Total bilirubin less than or equal to twice the lower limit of the normal range in the institution and aspartate aminotransferase (AST) less than or equal to twice the upper limit of normal
Absence of blasts in peripheral blood
Leukocyte count greater than 3000 cells / mm3, hemoglobin greater than 9.0 g / dl and platelets greater than 70,000 platelets / mm3, if possible. (If the patient does not meet these criteria for apheresis, the possibility of transfusion of blood components after leukapheresis will be proposed and the patient should sign a specific term of science on the possibility of transfusion)
Normal cardiac evaluation
Negative serologies for hepatitis B and C viruses and HIV
Written informed consent form signed before entering the study
Exclusion Criteria:
Does not meet any of the requirements of the inclusion criteria
Low risk myelodysplasia by IPSS (International Prognostic Scoring System) or WPSS (WHO adapted Prognostic Scoring System) scores
Individuals with a history of any previous neoplasia, except those with prolonged clinical remission (more than 5 years) of non-melanoma skin cancers and cervical cancer in situ
Pregnant or lactating women
Previous immunotherapy or biological therapy
Facility Information:
Facility Name
Hematology and Transfusion Medicine Center
City
Campinas
State/Province
São Paulo
ZIP/Postal Code
13083-870
Country
Brazil
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Cellular Immunotherapy for Patients With High Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia
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