Clinical trial NCT04615572: Screening to Improve Survival in AL Amyloidosis
Official title: Screening to Improve Survival in AL Amyloidosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Smoldering Multiple Myeloma
Monoclonal Gammopathy of Undetermined Significance
Keywords: SMM
MGUS
AL Amyloidosis
Lead sponsor: Tufts Medical Center
Sponsor class: OTHER
Collaborators: name: National Institute on Aging (NIA)
First posted: 2020-11-04
Enrollment: 35
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Smoldering Multiple Myeloma or Monoclonal Gammopathy of Undetermined Significance patients.

Brief summary: The purpose of this study is to see whether certain genes may be linked with the development of AL amyloidosis in subjects 60 years of age or older with the blood disorders SMM and MGUS. A limited repertoire of immunoglobulin (Ig) variable region genes have been associated with AL amyloidosis. The clonal plasma cells of subjects with SMM and MGUS may express one of these Ig variable region genes indicating a risk of progression to AL amyloidosis and potentially enabling early diagnosis. We hope this study will help us begin to understand whether Ig variable region gene identification can be a useful tool for assessing a subject's risk of progression to AL amyloidosis.


Eligibility criteria: Inclusion Criteria:

* Patients 60 years of age and older
* Diagnosed with λ LC MGUS or λ LC SMM
* dFLC greater than 23 mg/L and κ::λ free LC ratio below normal
* If the patient has an eGFR less than 50 mL/min/1.73m2, the κ::λ free LC ratio is inconsequential. The patient only needs to meet the age, dFLC, and λ LC MGUS or λ LC SMM diagnosis criteria in this case

Exclusion Criteria:

* Patients with κ LC MGUS or κ LC SMM
* Amyloid in the bone marrow or in other biopsies will not be included
* Patients younger than 60 years of age are not eligible

Primary outcomes:
measure: To determine risk of AL in patients with λ SMM or λ MGUS
timeFrame: 2 years
measure: To develop a B-cell Gene Rearrangement (BCGR) test for λ IGVL germline gene identification
timeFrame: 2 years
measure: To determine if marrow mononuclear cells are adequate for λ IGVL germline gene identification
timeFrame: 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Orange
country: United States
facility: University of California, Irvine Health Chao Family Comprehensive Cancer Center
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Miami
country: United States
facility: University of Miami Sylvester Comprehensive Cancer Center
state: Florida
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Chapel Hill
country: United States
facility: UNC Lineberger Comprehensive Cancer Center
state: North Carolina
Study officials:
References:
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