A Multi-Centre Observational Prospective Cohort Study Involving the Collection of Clinical Information and Biological Specimens for the Evaluation of Novel Prognostic Tests for Myelodysplasia and Acute Myeloid Leukemia
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Acute Myeloid Leukemia (AML)
- Sponsor
- Nova Scotia Health Authority
- Enrollment
- 11
- Locations
- 6
- Primary Endpoint
- Prognostic capacity of the candidate tests (alone and in combination) to predict response to treatment, time to relapse, time to death.
- Status
- Completed
- Last Updated
- 3 years ago
Overview
Brief Summary
This clinical study will provide the study specimens (samples of bone marrow and blood) and the clinical data for a pan-Canadian collaborative research project developed by the MDS/AML Research Consortium. The goal of this project involves the evaluation and potential validation of five novel prognostic tests for myelodysplasia (MDS) and/or acute myeloid leukemia (AML), as well as an analysis of health economic and socio-ethical implications related to the potential introduction of these tests into the clinical setting. The over-arching goal is to improve the outcomes of patients with MDS and AML. The primary hypothesis is that one or more of the laboratory tests being evaluated in conjunction with this study, either alone or in combination with other laboratory tests (either established or under investigation in this project), will have statistically significant prognostic value either alone or in combination with established clinical risk factors.
The clinical study will involve the enrollment of 200 adults with AML and 200 adults with MDS over a 2.5 year period. Participants will be followed on study for two years. Bone marrow and blood specimens will be collected at diagnosis and at other time points as required for the development of the five laboratory tests.
Participants will be assigned to treatment according to local institutional practice and will be followed for up to 2 years. Health economic and quality of life questionnaires will be administered at key time points. Data will be collected regarding participant characteristics, diagnosis, disease features, treatment and clinical outcome.
Detailed Description
Two of the tests involve a technology called flow cytometry. Both of the flow cytometry tests are used to predict whether a person is likely to have a good response to chemotherapy or not. Three of the tests involve new genetic-based technology. One of these tests is called comparative genomic profiling. This test can detect genetic abnormalities that current testing methods are not able to detect. Another test involves micro-RNA profiling. The final test involves RNA sequencing. The researchers think these tests might be useful in predicting how well a person will respond to treatment. The novel laboratory tests being evaluated as part of this study are still in the early phases of development and cannot be used for clinical decision making. Participants enrolled in this study will not be informed regarding their individual results with respect to the study tests that are conducted using their biospecimens. The following information (data) will be collected regarding study participants: diagnosis, results of relevant clinical tests, age, gender, treatment and outcome during the 2 year study follow-up period. The study also involves the completion of study questionnaires at six different time points over the course of the two year study follow-up.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Prognostic capacity of the candidate tests (alone and in combination) to predict response to treatment, time to relapse, time to death.
Time Frame: Two years following the completion of enrollment.
For the AML cohort, the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, white blood cell count at presentation, antecedent hematologic disorder, FLT-3 status, Karnofsky Performance Status (KPS), cytogenetic sub-group (using WHO 2008). For the MDS cohort the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, KPS, karyotype, bone marrow blast count and number of cytopenias at diagnosis.
Secondary Outcomes
- Cost impact of candidate tests.(Two years following the completion of enrollment.)
- Societal risks and benefits related to the candidate tests.(Two years following the completion of enrollment.)