Back to resultsPre-myeloid Cancer and Bone Marrow Failure Clinic Study
Primary Purpose
Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion
Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
follow up
Sponsored by
About this trial
This is an interventional screening trial for Myeloid Malignancy
Eligibility Criteria
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
Sites / Locations
- Mayo ClinicRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Potential Premyeloid Cancer or Bone Marrow Failure Patients
Arm Description
Follow up provided for patients tested using Next Generation Sequencing (NGS) and other relevant functional studies
Outcomes
Primary Outcome Measures
Number of subjects whose cytopenias are persistent or progressive over the course of the 5 year study
Secondary Outcome Measures
Number of subjects who have evolved to myelodspalstic syndrome (MDS) or acute myeloid leukemia (aml) over the course of the five year study
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02958462
Brief Title
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
Official Title
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 16, 2017 (Actual)
Primary Completion Date
September 15, 2030 (Anticipated)
Study Completion Date
September 15, 2035 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Mayo Clinic
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
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:
Idiopathic cytopenias of unclear significance (ICUS).
Clonal cytopenias of unclear significance (CCUS).
Clonal hematopoiesis of indeterminate prognosis (CHIP).
Bone marrow failure syndromes with risk for clonal myeloid transformation.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion, Cytopenia
7. Study Design
Primary Purpose
Screening
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2000 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Potential Premyeloid Cancer or Bone Marrow Failure Patients
Arm Type
Other
Arm Description
Follow up provided for patients tested using Next Generation Sequencing (NGS) and other relevant functional studies
Intervention Type
Other
Intervention Name(s)
follow up
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Brittany Burnap, BS
Phone
507-293-1764
Email
cimpmlresearch@mayo.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mrinal S Patnaik, MBBS
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
https://www.mayo.edu/research/clinical-trials
Description
Mayo Clinic Clinical Trials
Learn more about this trial
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
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