Natural History Study of Monoclonal Gammopathy of Undetermined Significance (MGUS) and Smoldering Myeloma (SMM)

March 4, 2021 updated by: National Cancer Institute (NCI)

Background:

- Multiple myeloma is a type of cancer that affects white blood cells and has a poor long-term survival rate. Two other types of cancer, monoclonal gammopathy of undetermined significance (MGUS) and smoldering myeloma (SMM), may eventually progress and develop into multiple myeloma. Researchers are interested in collecting samples from individuals who have been diagnosed with MGUS and SMM to study possible risk factors for developing multiple myeloma.

Objectives:

- To study risk factors that may cause MGUS and SMM to progress to multiple myeloma.

Eligibility:

- Individuals at least 18 years of age who have been diagnosed with either MGUS or SMM but do not have multiple myeloma.

Design:

  • Participants will be examined by study researchers at the initial visit, at 6 months following enrollment, and every 12 months for a maximum of 5 years.
  • The following tests may be performed: (1) blood and urine tests, (2) bone marrow aspiration and biopsy, (3) imaging studies, and (4) a skeletal survey (a series of skeletal X-rays of the skull, spine, pelvis, ribs, shoulders, upper arm, and thigh bones).
  • Treatment will not be provided as part of this protocol. - Participants will remain on the study for 5 years, or until their MGUS or SMM progresses to multiple myeloma requiring treatment.

Study Overview

Status

Terminated

Conditions

Detailed Description

Background:

  • Multiple Myeloma (MM) is an incurable plasma cell neoplasm with a median survival of 3-4 years.
  • Monoclonal gammopathy of undetermined significance (MGUS) and smoldering myeloma (SMM) are premalignant plasma cell proliferative disorders characterized by elevated monoclonal protein and bone marrow plasma cells. MGUS affects 3.2% of Caucasians over the age of 50 and has a 1% annual risk of progression to MM. approximately 3000 cases of SMM are diagnosed annually with a 10% annual risk of progression to MM.
  • Current risk stratification schemes rely on serum protein markers and phenotyping by flow cytometry. While they can differentiate high and low risk patients, they cannot predict outcome for individual patients, are not integrated with one another, and have limited direct correlation to biology.
  • Paired samples linked to clinical information can advance research into improved risk stratification, the pathogenesis of MGUS, SMM, and MM, and the potential for an early treatment window for these incurable diseases.

Objectives:

  • To characterize the natural history and prognosis of MGUS and SMM
  • To integrate protein markers (including immunoglobulin free light-chains) and immunophenotyping by flow cytometry with molecular profiles (including gene expression profiles) and clinical outcomes
  • To apply expertise and diagnostic technology to provide improved evaluation, monitoring, and risk-stratification for patients on this protocol
  • To provide paired samples of blood and tissue linked to clinical and molecular information for pilot translational studies

Eligibility:

  • A confirmed diagnosis of MGUS or SMM (based on IMWG diagnostic criteria)
  • Age greater than or equal to 18 years
  • ECOG performance status in the range of 0-2
  • Patients who have a diagnosis of MM are not eligible for this study.

Design:

  • This is a prospective cohort study of patients with either MGUS or SMM.
  • Following initial evaluation and confirmation of diagnosis, patients will be followed as clinically indicated, usually at 12 month intervals.
  • The primary endpoint is progression to MM requiring treatment.

  • Patients may donate cellular products or tissues as appropriate for research purposes.

    154 patients with MGUS and 154 patients with SMM will be enrolled on this protocol.

Study Type

Observational

Enrollment (Actual)

225

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Institutes of Health Clinical Center, 9000 Rockville Pike

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with MGUS or SMM will enroll on the study for the purpose of expert monitoring of their disease (MGUS or SMM), improved risk-stratification, and for the purpose of donating tissue and cellular products for research.@@@

Description

  • INCLUSION CRITERIA:
  • Diagnosis of MGUS and SMM will be made in accordance with the clinical diagnostic criteria set forth by the International Myeloma Working Group (Table 1).
  • 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.
  • Age greater than or equal to 18 years.
  • ECOG performance status of 0-2.
  • The patient must be competent to sign an informed consent form.

EXCLUSION CRITERIA:

- 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 International Myeloma Working Group s diagnostic CRAB criteria:

hypercalcemia [serum calcium at least 1 mg/dL above the upper limit of normal], renal failure [creatinine greater than 1.95 mg/dL), anemia (hemoglobin less than 10 g/dL], or bone lesions [lytic lesions or osteoporosis with compression fractures]).

- Patients who have received previous therapy for MM.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
patients with MGUS or SMM
patients with either Monoclonal gammopathy of undetermined significance (MGUS) or smoldering myeloma (SMM)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Characterize the natural history and prognosis of MGUS and SMM
Time Frame: 6 months
determination of subtypes of MM based on GEP, and determination of prognostic indicators
6 months
Integrate protein markers with molecular profiles and clinical outcomes.
Time Frame: 6 months
comparison of molecular profiles and rate of disease progression per MM subtype
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Cancer Institute (NCI)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 22, 2010

Primary Completion (Actual)

May 16, 2014

Study Completion (Actual)

February 25, 2021

Study Registration Dates

First Submitted

April 22, 2010

First Submitted That Met QC Criteria

April 22, 2010

First Posted (Estimate)

April 23, 2010

Study Record Updates

Last Update Posted (Actual)

March 8, 2021

Last Update Submitted That Met QC Criteria

March 4, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 100096
  • 10-C-0096

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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