Clinical trial NCT06365060: Screening for AL Amyloidosis in Smoldering Multiple Myeloma
Official title: Screening for AL Amyloidosis in Smoldering Multiple Myeloma
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Smoldering Multiple Myeloma
Keywords: SMM
AL Amyloidosis
Lead sponsor: Tufts Medical Center
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2024-04-15
Enrollment: 400
Sex: ALL
Minimum age (years): 40
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 patients.

Brief summary: In this multicenter study, we will recruit 400 patients 40 years of age or older at 15 centers with a diagnosis of smoldering multiple myeloma (SMM), a group of patients for whom standard of care is observation not treatment. The main goal of this study is to screen for the diagnosis of light-chain amyloidosis (AL) before the onset of symptomatic disease and to develop a training set for a likelihood algorithm.

Detailed description: This study is based on results from two prior studies in which 4 of 36 patients with SMM and none of 14 patients with MGUS were found to have AL. The hypothesis that we test with this protocol is that patients with (1) a pre-existing diagnosis of SMM, (2) free light chain (FLC) abnormalities, (3) IGLV genes associated with AL,(4) t(11;14) or gain 1q, and (5) NT-proBNP \> 332pg/mL will have undiagnosed AL or risk of progression to AL. We will study the potential for SMM, the FLC screen, AL-related IGLV gene use, t(11;14) or gain 1q cytogenetic abnormalities, and NT-proBNP \> 332pg/mL to be the variables in a likelihood algorithm for AL.

Eligibility criteria: Inclusion Criteria:

* Patients 40 years of age and older
* diagnosed with Smoldering Multiple Myeloma
* dFLC greater than 23 mg/L
* abnormal FLC ratio
* If the patient has an eGFR less than 50 mL/min/1.73m2, the FLC ratio is inconsequential. The patient only needs to meet the age and dFLC criterion.

Exclusion Criteria:

* Patients younger than 40 years of age are not eligible
* Patients with a previous finding of amyloid in other biopsies will not be included
* Adults unable to consent are not eligible, including the cognitively impaired Pregnant women, pregnant minors, minors (i.e., individuals who are not yet adults), wards of the state, non-viable neonates, neonates of uncertain viability, and prisoners are not eligible

Primary outcomes:
measure: Creating a network to enroll patients on a collaborative study requiring marrow and blood specimens, to collect data for a training set of likelihood statistics and to plan a future validation study.
timeFrame: 5 years
measure: Validating an NGS assay that identifies IGLV genes in clonal plasma cells
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama Hospital
state: Alabama
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Weston
country: United States
facility: Cleveland Clinic Florida, Weston Hospital
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: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Charlotte
country: United States
facility: Atrium Health Levine Cancer Institute
state: North Carolina
city: Durham
country: United States
facility: UNC Lineberger Comprehensive Cancer Center
state: North Carolina
city: Dallas
country: United States
facility: UT Southwestern, Harold C. Simmons Comprehensive Cancer Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah, Huntsman Cancer Hospital
state: Utah
city: Richmond
country: United States
facility: VCU Medical Center
state: Virginia
Study officials:
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