AML-MDS Novel Prognostic Tests Clinical Study

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2013 by Capital District Health Authority, Canada
Sponsor:
Collaborator:
Terry Fox Research Institute
Information provided by (Responsible Party):
Stephen Couban, Capital District Health Authority, Canada
ClinicalTrials.gov Identifier:
NCT01685619
First received: September 12, 2012
Last updated: February 4, 2013
Last verified: February 2013
  Purpose

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.


Condition
Acute Myeloid Leukemia (AML)
Myelodysplastic Syndrome (MDS)

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Capital District Health Authority, Canada:

Primary Outcome Measures:
  • 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. ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Cost impact of candidate tests. [ Time Frame: Two years following the completion of enrollment. ] [ Designated as safety issue: No ]
    The cost-effectiveness of the candidate tests for AML and MDS treatment will be projected with the aid of economic simulation models.

  • Societal risks and benefits related to the candidate tests. [ Time Frame: Two years following the completion of enrollment. ] [ Designated as safety issue: No ]
    A comparative analysis of policies and regulations in Canada governing prognostic tests will be undertaken (including tests that utilize RNA and DNA based technologies). In addition, input will be obtained from key stakeholders. This information will be used to develop a set of guidelines and best practices for this type of research.


Biospecimen Retention:   Samples With DNA

Some of the laboratory tests that will be evaluated as part of this study will utilize RNA and/or DNA. There may be biospecimens left over after the work related to this study is complete. At the time of consent for the study, participants will be asked to provide consent (or decline their consent) regarding the use of leftover specimens (including RNA and DNA) for other research. The details regarding this other research are provided in the protocol and consent template.


Estimated Enrollment: 400
Study Start Date: September 2012
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)
Groups/Cohorts
AML Cohort
This cohort is comprised of participants who have acute myelogenous leukemia (AML).
MDS Cohort
This cohort is comprised of participants who have myelodysplastic syndrome (MDS).

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Prospective participants will include patients with known or supsected AML or MDS who are being treated and/or assessed at a participating site.

Criteria

There are two parts to the study: Part One (Collection of Biospecimens) and Part Two (Two year follow-up that includes data collection regarding treatment, outcome and questionnaire completion).

INCLUSION CRITERIA (PART ONE):

Prospective participants can be included in the study if:

  • The participant is 18 years of age or older
  • The participant is suspected to have a new diagnosis of MDS (including CMML) , OR suspected to have a new diagnosis of AML excluding acute promyelocytic leukemia (APL), OR known to have a diagnosis of MDS (including CMML) confirmed by bone marrow aspirate and biopsy no more than one year prior to the date of enrollment AND without commencement of definitive therapy prior to enrollment
  • The participant is scheduled to have a diagnostic or confirmatory bone marrow aspirate and biopsy at a participating site, or in the case of prospective participants with an established diagnosis of MDS (including CMML), must be able to undergo a bone marrow aspirate for the study at the participating site
  • The participant must be able to read and/or understand spoken English or French so that they will be eligible for Part Two of the study
  • The participant must be able to understand and sign the informed consent form applicable to their situation

EXCLUSION CRITERIA (PART ONE):

Prospective participants should be excluded from the study if:

  • The participant has already received definitive therapy for AML or MDS
  • The participant has a diagnosis of MDS that was confirmed more than one year prior to the date of enrollment

INCLUSION CRITERIA (PART TWO):

Participants who have been enrolled in Part One will be eligible to participate in the full two year study follow-up component if they meet the following criteria:

  • Confirmed diagnosis of either MDS, CMML or AML (excluding APL)
  • Sufficient cell count for the MDS/AML Clinical Study requirements as follows:

For participants with suspected (or known) AML:

The blast count of the peripheral blood taken at diagnosis must be greater than 1 x10^6 blast count/mL

  • It must be possible to earmark for the MDS/AML Study:
  • 3 vials 1.0 x 10^7/mL mononuclear peripheral blood cells
  • 1 vial 0.5 x 10^7/mL mononuclear bone marrow cells

Cells are to be prepared according to the site's local cell bank procedures so that they can be stored and transported to study labs as needed.

At sites participating in the Hogge Assay:

In addition to the specimens described above, it must be possible to provide 2 mL of fresh bone marrow or 5 mL of fresh peripheral blood with > 1 x 10^6 blast count/mL

For participants with suspected (or known) MDS:

  • It must be possible to earmark for the MDS/AML Study:
  • 2 vials 1.0 x 10^7/mL mononuclear peripheral blood cells
  • 2 vials 1.0 x 10^7/mL mononuclear bone marrow cells

Cells are to be prepared according to the site's local cell bank procedures so that they can be stored and transported to study labs as needed.

EXCLUSION CRITERIA (PART TWO):

Participants who have been enrolled in Part One will not be eligible to participate in the full two year study follow-up component if they:

  • Do not have sufficient cell count for the MDS/AML Clinical Study requirements as set out in Section 11.3
  • It is confirmed after enrollment that they do not have a diagnosis of MDS, CMML or AML
  • A diagnosis of APL is confirmed
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01685619

Contacts
Contact: Stephen Couban, M.D. 902-473-8562 Stephen.Couban@cdha.nshealth.ca
Contact: Holly Kerr, B.A. 604-875-4111 ext 63196 hkerr@bccancer.bc.ca

Locations
Canada, Alberta
Tom Baker Cancer Centre Not yet recruiting
Calgary, Alberta, Canada, T2N 4N2
Contact: Lynn Savoie, M.D.    403-944-1880    Lynn.Savoie@albertahealthservices.ca   
Principal Investigator: Lynn Savoie, M.D.         
Canada, British Columbia
Vancouver General Hospital Recruiting
Vancouver, British Columbia, Canada, V5Z 4E3
Contact: Donna Hogge, M.D., Ph.D.    604-875-4863    dhogge@bccancer.bc.ca   
Contact: Holly Kerr, B.A.    604-875-4111 ext 63196    hkerr@bccancer.bc.ca   
Principal Investigator: Donna Hogge, M.D., Ph.D.         
Canada, Manitoba
CancerCare Manitoba Recruiting
Winnipeg, Manitoba, Canada, R3M 1A5
Contact: Matthew Seftel, M.D.    204-787-2108    Matthew.Seftel@cancercare.mb.ca   
Contact: Kathryn Dyck, B.A.    204-787-2127    Kathryn.Dyck@cancercare.mb.ca   
Principal Investigator: Matthew Seftel, M.D.         
Canada, Nova Scotia
Queen Elizabeth II Health Sciences Centre Recruiting
Halifax, Nova Scotia, Canada, B3H 2Y9
Contact: Stephen Couban, M.D.    902-473-8562    Stephen.Couban@cdha.nshealth.ca   
Contact: Susan Pleasance, BScN    902-473-7585    Susan.Pleasance@cdha.nshealth.ca   
Principal Investigator: Stephen Couban, M.D.         
Canada, Ontario
Princess Margaret Cancer Centre (formerly Princess Margaret Hospital) Recruiting
Toronto, Ontario, Canada, M5G 2M9
Contact: Mark Minden, M.D.    416-946-4501 ext 5106    Mark.Minden@uhn.ca   
Contact: Andrea Arruda, M.Sc.    416-946-4501 ext 2648    andrea.arruda@uhnresearch.ca   
Principal Investigator: Mark Minden, M.D., Ph.D.         
Canada, Quebec
Hôpital Maisonneuve-Rosemont Recruiting
Montreal, Quebec, Canada, H1T 2M4
Contact: Julie Bergeron, M.D.    514-252-3404    juliebergeron.hmr@ssss.gouv.qc.ca   
Contact: Julie Lu, B.Sc.    514-254-7455 ext 3336    jtlu.hmr@ssss.gouv.qc.ca   
Principal Investigator: Julie Bergeron, M.D.         
Sponsors and Collaborators
Stephen Couban
Terry Fox Research Institute
Investigators
Principal Investigator: Stephen Couban, M.D. Capital District Health Authority, Canada
  More Information

No publications provided

Responsible Party: Stephen Couban, Interim Head, Division of Hematology, Director, Blood and Marrow Transplant Program, Capital District Health Authority, Canada
ClinicalTrials.gov Identifier: NCT01685619     History of Changes
Other Study ID Numbers: AML-MDS 01-2011
Study First Received: September 12, 2012
Last Updated: February 4, 2013
Health Authority: Canada: Submission of this trial to Health Canada is not required. (This has been confirmed with Health Canada.)

Keywords provided by Capital District Health Authority, Canada:
AML
Acute Myeloid Leukemia
MDS
Myelodysplastic Syndrome
Prognostic
Economic

Additional relevant MeSH terms:
Leukemia
Leukemia, Myeloid, Acute
Leukemia, Myeloid
Myelodysplastic Syndromes
Preleukemia
Neoplasms by Histologic Type
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions

ClinicalTrials.gov processed this record on July 20, 2014