Clinical trial NCT05361694: Two Biologically and Clinically Distinct Entities: Progressive Versus Stable Multiple Myeloma (MM) Precursor Conditions
Official title: Two Biologically and Clinically Distinct Entities: Progressive Versus Stable Multiple Myeloma (MM) Precursor Conditions (TRANSFORMM)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Multiple Myeloma
Smoldering Multiple Myeloma
Monoclonal Gammopathy of Undetermined Significance
Lead sponsor: University of Miami
Sponsor class: OTHER
First posted: 2022-05-05
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult participants diagnosed with MGUS or SMM.

Brief summary: The key aim of the study is to define the two biologically and clinically distinct entities: progressive versus stable myeloma precursor conditions.


Eligibility criteria: Inclusion Criteria:

1. Diagnosis of MGUS and SMM will be made in accordance with the clinical diagnostic criteria set forth by the 2014 International Myeloma Working Group (IMWG) Revised Criteria.2
2. The diagnoses will be confirmed by either serum/urine protein electrophoresis, immunofixation and light-chain assays; or immunohistochemistry analyses of the bone marrow biopsy, or a combination of these tests.
3. Age greater than or equal to 18 years.
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0-3.
5. The patient must be competent to sign an informed consent form.

Exclusion Criteria:

1. A diagnosis of MM as defined as any patient with detectable M-protein in blood and/or urine, monoclonal plasma cells in the bone marrow, and evidence of end-organ damage based on the Calcium Elevation, Renal Failure, Anemia, and Bone Disease (CRAB) criteria and/or myeloma-defining events.

   * Patients who have received previous therapy for MM.
   * Patients with known plasma cell or related lymphoid (e.g. lymphoplasmacytic lymphoma, Amyloid Light chain (AL) amyloidosis)
2. Confirmation of pathological diagnosis is required either from the initial pathology review report or review from the UM/SCCC Hematopathologist in accordance with the clinical diagnostic criteria set forth by the International Myeloma Working Group (IMWG) or World Health Organization (WHO). Tumor tissue that has been previously collected and is available for study or that can be collected with minimal additional risk to the patient during sampling required for routine patient care or required testing on a University of Miami (UM) /Sylvester Comprehensive Cancer Center (SCCC) research protocol will be used for diagnosis.
3. Active symptomatic major organ disorder that would increase the risk of biopsy or other procedure, including but not limited to ischemic heart disease, recent myocardial infarction, active congestive heart failure, pulmonary dysfunction.

   * Active concomitant medical or psychological illnesses that may increase the risk to the patient or inability to obtain informed consent, at the discretion of the Principal Investigator.
   * Pregnant or breast-feeding women will not be eligible for any aspect of this protocol.
   * Prisoners will be excluded.

Primary outcomes:
measure: Rate of progression to active Multiple Myeloma (MM)
timeFrame: Up to 5 years
Secondary outcomes:
measure: Frequency of participant conversion from MGUS/SMM to Myeloma defining genomic events
timeFrame: Up to 5 years
measure: Frequency of participant conversion from MGUS/SMM to associated progressive phenotype
timeFrame: Up to 5 years
measure: Rate of participant conversion from MGUS/SMM to Myeloma defining genomic events
timeFrame: Up to 5 years
measure: Rate of participant conversion from MGUS/SMM to associated progressive phenotype
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami Hospitals
state: Florida
Study officials:
affiliation: University of Miami
name: Carl Landgren, MD
role: PRINCIPAL_INVESTIGATOR
References: