AML-MDS Novel Prognostic Tests Clinical Study

NCT01685619 | Completed DMC

• 1 other identifier: AML-MDS 01-2011
• Study Type: observational
• Target: 11
• Locations: 1 country
• Sites: 6

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.

Conditions

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

Keywords

AML; Acute Myeloid Leukemia; MDS; Myelodysplastic Syndrome; Prognostic; Economic

Outcome Measures

Primary Outcomes (1)

• Prognostic capacity of the candidate tests (alone and in combination) to predict response to treatment, time to relapse, time to death.

  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.

  Two years following the completion of enrollment.

Secondary Outcomes (2)

• 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.

Study Arms (2)

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).

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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
• 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:
• vials 1.0 x 10\^7/mL mononuclear peripheral blood cells
• 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:

Sponsors & Collaborators

• Nova Scotia Health Authority: lead
• Terry Fox Research Institute: collaborator

Study Sites (6)

Tom Baker Cancer Centre

Calgary, Alberta, T2N 4N2, Canada

Location

Vancouver General Hospital

Vancouver, British Columbia, V5Z 4E3, Canada

Location

CancerCare Manitoba

Winnipeg, Manitoba, R3M 1A5, Canada

Location

Queen Elizabeth II Health Sciences Centre

Halifax, Nova Scotia, B3H 2Y9, Canada

Location

Princess Margaret Cancer Centre (formerly Princess Margaret Hospital)

Toronto, Ontario, M5G 2M9, Canada

Location

Hôpital Maisonneuve-Rosemont

Montreal, Quebec, H1T 2M4, Canada

Location

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.

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Study Officials

• Stephen Couban, M.D.

  Nova Scotia Health Authority

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 12, 2012
• First Posted: September 14, 2012
• Study Start: October 2, 2012
• Primary Completion: January 24, 2018
• Study Completion: January 24, 2018
• Last Updated: November 8, 2022

Record last verified: 2022-11

Locations

CA (6)