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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT02958462
Other study ID # 16-004173
Secondary ID NCI-2022-07824
Status Recruiting
Phase N/A
First received
Last updated
Start date January 16, 2017
Est. completion date September 15, 2035

Study information

Verified date March 2024
Source Mayo Clinic
Contact Brittany Burnap
Phone 507-293-1764
Email cimpmlresearch@mayo.edu
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes. NGS is a procedure that looks at relevant cancer associated genes and what they do. Finding genetic markers for pre-malignant conditions may help identify patients who are at risk of pre-myeloid cancers and bone marrow failure syndromes and lead to earlier intervention.


Description:

PRIMARY OBJECTIVES: I. To use genomics and functional translational studies to diagnose, prognosticate and potentially offer therapeutic directives for patients with precursor features of myeloid neoplasms (myelodysplastic syndrome [MDS], myeloproliferative neoplasms [MPN], MDS/MPN overlap syndrome) and germline predisposition/bone marrow failure states, who do not meet the criteria for the diagnosis of these cancers as of yet. II. To identify patients with precursor myeloid malignancies and bone marrow failure syndromes. III. To examine the utility of NGS methods for discovery of targets or pathways involved in precursor features of myeloid cancer and bone marrow failure. IV. To use clinomics/genomics to better understand pathobiology and risk of disease progression. V. To help better understand the implications of variants of unknown significance using computational biology and functional studies. VI. To utilize normal, age and sex matched controls to validate genetic and epigenetic testing carried out under this protocol (essential for accurate data analysis). VII. To assess frailty in patients with clonal hematopoiesis in order to validate genetic and epigenetic testing completed under this protocol as objective assessments of frailty and aging in comparison to standard of care frailty and geriatric assessments. OUTLINE: Participants may undergo blood sample collection, a bone marrow biopsy, a skin punch biopsy, hair follicle collection, a buccal swab, and/or saliva collection for NGS analysis on study. Patients may additionally undergo clinical assessment and may receive genetic counseling on study.


Recruitment information / eligibility

Status Recruiting
Enrollment 2000
Est. completion date September 15, 2035
Est. primary completion date September 15, 2030
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Patients with idiopathic cytopenias of unclear significance (ICUS) - Patients with clonal hematopoiesis of indeterminate significance (clonal hematopoiesis of indeterminate potential [CHIP]), including the recently described CHIP syndrome called VEXAS (vacuoles, E1 ubiquitin ligase, X chromosomal, autoimmune and somatic) - Patients with clonal cytopenias of undetermined significance (CCUS) - Marrow failure syndromes with myeloid malignancy predisposition - telomere dysfunction, chromosomal breakage disorders - Germ line inherited syndromes with risk for malignant transformation - GATA2, CEBPA, ETV-6, RUNX1, JAK2, PF6, etc. - Low risk MDS (idiopathic dysplasia of unclear significance) - Family member of a patient with one of the above conditions - Patient at high risk or suspected of developing one of the above conditions Exclusion Criteria: - Patients under 18 years of age

Study Design


Related Conditions & MeSH terms

  • Anemia
  • Anemia, Aplastic
  • Bone Marrow Failure Disorders
  • Bone Marrow Failure Syndrome
  • Clonal Cytopenia of Undetermined Significance
  • Clonal Expansion
  • Clonal Hematopoiesis of Indeterminate Potential
  • Congenital Bone Marrow Failure Syndromes
  • Cytopenia
  • Hematologic Neoplasms
  • Hemoglobinuria, Paroxysmal
  • Hereditary Neoplastic Syndrome
  • Inherited Bone Marrow Failure Syndrome
  • Leukopenia
  • Low Risk Myelodysplastic Syndrome
  • Myelodysplastic Syndromes
  • Myeloid Malignancy
  • Neoplasms
  • Neoplastic Syndromes, Hereditary
  • Pancytopenia
  • Syndrome
  • Thrombocytopenia

Intervention

Procedure:
Biospecimen Collection
Undergo blood sample, hair follicle, and saliva collection
Bone Marrow Biopsy
Undergo a bone marrow biopsy
Punch Biopsy
Undergo a skin punch biopsy
Buccal Swab
Undergo a saliva or buccal swab
Other:
Clinical Evaluation
Undergo clinical assessment
Genetic Counseling
Receive genetic counseling
Quality-of-Life Assessment
Ancillary studies
Electronic Health Record Review
Ancillary studies

Locations

Country Name City State
United States Mayo Clinic in Florida Jacksonville Florida
United States Mayo Clinic in Rochester Rochester Minnesota
United States Mayo Clinic in Arizona Scottsdale Arizona

Sponsors (1)

Lead Sponsor Collaborator
Mayo Clinic

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Occurrence of cytopenias Assessed by the number of subjects whose cytopenias are persistent or progressive over the course of the study Up tof 5 years
Secondary Occurrence of myelodysplastic syndrome (MDS) Assessed by the number of subjects who have evolved to MDS over the course of the study Up to 5 years
Secondary Occurrence of acute myeloid leukemia (AML) Assessed by the number of subjects who have evolved to AML over the course of the study Up to 5 years
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