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Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02958462
Recruitment Status : Recruiting
First Posted : November 8, 2016
Last Update Posted : January 18, 2020
Information provided by (Responsible Party):
Mrinal S. Patnaik, M.B.B.S., Mayo Clinic

Brief Summary:
The use of genomics and functional translational studies is to identify and diagnose patients with precursor features of myeloid neoplasms and inherited bone marrow failure syndromes.

Condition or disease Intervention/treatment Phase
Myeloid Malignancy Inherited Bone Marrow Failure Syndrome Clonal Expansion Cytopenia Other: follow up Not Applicable

Detailed Description:

The pre-myeloid cancer and bone marrow failure clinic is an individualized medicine clinic conceptualized for the early detection of myeloid malignancies such as, myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN), MDS/MPN overlap syndromes and acute myeloid leukemia (AML). Using next-generation sequencing technologies, clonal hematopoietic stem cell changes can be detected early on in myeloid malignancies. These changes are currently classified as follows:

  1. Idiopathic cytopenias of unclear significance (ICUS).
  2. Clonal cytopenias of unclear significance (CCUS).
  3. Clonal hematopoiesis of indeterminate prognosis (CHIP).
  4. Bone marrow failure syndromes with risk for clonal myeloid transformation.
  5. Familial/Germline syndromes with increased risk for myeloid malignancies.

This clinic blends next-generation sequencing technologies with functional studies to help identify, prognosticate and define management plans; including early intervention strategies, for affected patients.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Screening
Official Title: Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
Study Start Date : November 2016
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : January 2023

Arm Intervention/treatment
Potential Premyeloid Cancer or Bone Marrow Failure Patients
Follow up provided for patients tested using Next Generation Sequencing (NGS) and other relevant functional studies
Other: follow up

Primary Outcome Measures :
  1. Number of subjects whose cytopenias are persistent or progressive over the course of the 5 year study [ Time Frame: Through study completion, an average of 5 years ]

Secondary Outcome Measures :
  1. Number of subjects who have evolved to myelodspalstic syndrome (MDS) or acute myeloid leukemia (aml) over the course of the five year study [ Time Frame: Through study completion, an average of 5 years ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Patients with:

  • Idiopathic cytopenias of unclear significance (ICUS)
  • Clonal hematopoiesis of indeterminate significance (CHIP)
  • Age related clonal hematopoiesis (ARCH)
  • Clonal cytopenias of unclear significance (CCUS)
  • Marrow failure syndromes with myeloid malignancy predisposition- telomere dysfunction, chromosomal breakage disorders, etc.
  • Germ line inherited syndromes with risk for malignant transformation
  • Low Risk Myelodysplastic Syndromes (MDS)

Exclusion Criteria:

  • Patients who are unable to understand and sign the Informed Consent Form

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02958462

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Contact: Gillian Currie, BSc 507-293-0792

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United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
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Principal Investigator: Mrinal S Patnaik, MBBS Mayo Clinic
Additional Information:
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Responsible Party: Mrinal S. Patnaik, M.B.B.S., Assistant Professor of Medicine and Oncology, Mayo Clinic Identifier: NCT02958462    
Other Study ID Numbers: 16-004173
First Posted: November 8, 2016    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Hematologic Diseases