Smoldering MyelomaNCT01109407
Natural History Study of Monoclonal Gammopathy of Undetermined Significance (MGUS) and Smoldering Myeloma (SMM)
An observational study of Smoldering Myeloma and Monoclonal Gammopathy of Undetermined Significance, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Smoldering Myeloma, Monoclonal Gammopathy of Undetermined Significance
- How many people
- 225 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Apr 2010to 25 Feb 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.@@@
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
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.
Read more
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.
Read the 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.
- Keywords
- Plasma Cell Neoplasm, Natural History, Translational Studies, Gene Expression Profiling, Protein Markers, Smoldering Myeloma, SMM, Monoclonal Gammopathy of Undetermined Significance, MGUS
- Medical subject headings
- Smoldering Multiple Myeloma, Monoclonal Gammopathy of Undetermined Significance, Neoplasms, Plasma Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| patients with MGUS or SMM | patients with either Monoclonal gammopathy of undetermined significance (MGUS) or smoldering myeloma (SMM) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize the natural history and prognosis of MGUS and SMM
determination of subtypes of MM based on GEP, and determination of prognostic indicators
Measured: 6 months
Integrate protein markers with molecular profiles and clinical outcomes.
comparison of molecular profiles and rate of disease progression per MM subtype
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2010 (estimated)
- Study start22 Apr 2010
- Primary completion16 May 2014
- Study completion25 Feb 2021
Why it stopped: PI Left before primary outcome was established
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Smoldering MyelomaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 225 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Mark J Roschewski, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01109407
- Sponsor's protocol ID
- 100096
- Other IDs
- 10-C-0096
- Status last verified
- Mar 2021
- First submitted
- 22 Apr 2010
- Last updated
- 8 Mar 2021
- Results posted
- No
Publications
Kristinsson SY, Landgren O, Dickman PW, Derolf AR, Bjorkholm M. Patterns of survival in multiple myeloma: a population-based study of patients diagnosed in Sweden from 1973 to 2003. J Clin Oncol. 2007 May 20;25(15):1993-9. doi: 10.1200/JCO.2006.09.0100. Epub 2007 Apr 9.
PubMed 17420512Kumar SK, Rajkumar SV, Dispenzieri A, Lacy MQ, Hayman SR, Buadi FK, Zeldenrust SR, Dingli D, Russell SJ, Lust JA, Greipp PR, Kyle RA, Gertz MA. Improved survival in multiple myeloma and the impact of novel therapies. Blood. 2008 Mar 1;111(5):2516-20. doi: 10.1182/blood-2007-10-116129. Epub 2007 Nov 1.
PubMed 17975015Landgren O, Kyle RA, Pfeiffer RM, Katzmann JA, Caporaso NE, Hayes RB, Dispenzieri A, Kumar S, Clark RJ, Baris D, Hoover R, Rajkumar SV. Monoclonal gammopathy of undetermined significance (MGUS) consistently precedes multiple myeloma: a prospective study. Blood. 2009 May 28;113(22):5412-7. doi: 10.1182/blood-2008-12-194241. Epub 2009 Jan 29.
PubMed 19179464
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study of Monoclonal Gammopathy of Undetermined Significance (MGUS) and Smoldering Myeloma (SMM)
- ClinicalTrials.gov ID
- NCT01109407
- Acronym / sponsor ID
- 100096
- Keywords
- Plasma Cell Neoplasm, Natural History, Translational Studies, Gene Expression Profiling, Protein Markers, Smoldering Myeloma, SMM, Monoclonal Gammopathy of Undetermined Significance
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: PI Left before primary outcome was established
- Enrolment
- 225 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 22 Apr 2010 (actual)
- Primary completion
- 16 May 2014 (actual)
- Study completion
- 25 Feb 2021 (actual)
- First posted
- 23 Apr 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with MGUS or SMM | – | –patients with either Monoclonal gammopathy of undetermined significance (MGUS) or smoldering myeloma (SMM) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.@@@
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
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
Results and safety
The study was stopped early (reason given: PI Left before primary outcome was established). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2021.
- Background: PubMed 17420512Kristinsson SY, Landgren O, Dickman PW, Derolf AR, Bjorkholm M. Patterns of survival in multiple myeloma: a population-based study of patients diagnosed in Sweden from 1973 to 2003. J Clin Oncol. 2007 May 20;25(15):1993-9. doi: 10.1200/JCO.2006.09.0100. Epub 2007 Apr 9.
- Background: PubMed 17975015Kumar SK, Rajkumar SV, Dispenzieri A, Lacy MQ, Hayman SR, Buadi FK, Zeldenrust SR, Dingli D, Russell SJ, Lust JA, Greipp PR, Kyle RA, Gertz MA. Improved survival in multiple myeloma and the impact of novel therapies. Blood. 2008 Mar 1;111(5):2516-20. doi: 10.1182/blood-2007-10-116129. Epub 2007 Nov 1.
- Background: PubMed 19179464Landgren O, Kyle RA, Pfeiffer RM, Katzmann JA, Caporaso NE, Hayes RB, Dispenzieri A, Kumar S, Clark RJ, Baris D, Hoover R, Rajkumar SV. Monoclonal gammopathy of undetermined significance (MGUS) consistently precedes multiple myeloma: a prospective study. Blood. 2009 May 28;113(22):5412-7. doi: 10.1182/blood-2008-12-194241. Epub 2009 Jan 29.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark J Roschewski, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Smoldering Myeloma and Monoclonal Gammopathy of Undetermined Significance, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: PI Left before primary outcome was established). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterize the natural history and prognosis of MGUS and SMM (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01109407, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Mar 2021.