Clinical trial NCT01958528: Study Of Circulating Multiple Myeloma Cells As A Biomarker of MGUS And SMM
Official title: A Prospective Study Of Circulating Multiple Myeloma Cells As A Biomarker Of Progression In Myeloma Precursor States (MGUS And SMM)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: a Diagnosis of MGUS or SMM by IMWG Criteria
Lead sponsor: Abramson Cancer Center at Penn Medicine
Sponsor class: OTHER
First posted: 2013-10-09
Enrollment: 52
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: Male and Female subjects with diagnosis of MGUS or SMM by IMWG Criteria

Brief summary: This is a prospective study to collect blood and bone marrow samples from patients with SM and MGUS. About 100 subjects will be enrolled at the University of Pennsylvania and followed for 2 years. The primary objective of this study is to evaluate CMMCs as a biomarker to detect patients at high risk of progression to multiple myeloma in patients with MGUS and SMM.

Detailed description: The primary objective is to evaluate CMMCs as a biomarker to detect patients at high risk of progression to multiple myeloma in patients with MGUS and SMM. The secondary objective are to correlate CMMCs with the Mayo Clinic prognostic models in MGUS/SMM, to compare bone marrow genomic findings with genomic findings in CMMC (utilizing multiple myeloma FISH panel), to perform immunophenotyping of bone marrow plasma cells, to perform gene expression profiling on CD138+bone marrow plasma cells and correlate with progression, to perform serum microRNA profiling and correlate with progression, to correlate bone marrow immunohistochemical studies evaluating the microenvironment with CMMC, to perform proteomic profiling on peripheral blood to evaluate serum biomarkers of progression and to establish a biorepository of samples of peripheral blood for future studies.

Eligibility criteria: Inclusion Criteria:

* Age 18 or greater
* Capable of informed consent
* A monoclonal gammopathy detected in the serum, including intact immunoglobulin (IgG and IgA) and light chain only gammopathies. Light chain only gammopathy is defined as an abnormal light chain ratio and increased involved light chain).44, 45
* Absence of myeloma related organ or tissue impairment ("CRAB") as defined by:

  * Hypercalcemia (calcium greater than or equal to 11)
  * Renal failure (creatinine \>2.0)
  * Anemia hemoglobin \<10 gm/dl)
  * Bone disease (Osteolytic lesions, fractures)

Exclusion Criteria:

* A secondary B-cell neoplasm or other active malignancy aside from non-melanoma skin cancer or localized prostate cancer. An active malignancy is any malignancy requiring therapy within the past 3 years. Patients with monoclonal B-cell lymphocytosis are not excluded.
* IgM monoclonal gammopathy
* Inability to comply with follow-up

Primary outcomes:
measure: Time to Progression
timeFrame: 4 years
Secondary outcomes:
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Abramson Cancer Center of the University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: Abramson Cancer Center at Penn Medicine
name: Alfred Garfall, MD
role: PRINCIPAL_INVESTIGATOR
References: