Clinical Trial: A Diagnostic Screening Trial Seeking AL Amyloidosis Very Early
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: A Diagnostic Screening Trial Seeking AL Amyloidosis Very Early
Brief Summary: This protocol seeks to enroll smoldering multiple myeloma (SMM) patients with λ light chain (LC) involvement, a group of patients for whom standard of care is observation not treatment. Patients with SMM have a precursor plasma cell disorder from which light chain amyloidosis (AL) often evolves. In this trial they will have blood and bone marrow cells evaluated by molecular testing to determine their clonal λ LC variable region (VL) germline gene. The repertoire of clonal germline genes employed in AL is restricted; 70% of cases of AL involve just 7 germline donors, 5 of which are λ germline donors. The hypothesis that will be tested with this protocol is that the presence of VL germline genes associated with AL in patients with a pre-existing diagnosis of λ SMM will be indicative of risk of progression to AL.
Detailed Summary:
In this trial up to 200 smoldering multiple myeloma (SMM) patients with λ light chain (LC) involvement whose κ::λ LC ratio are < 0.26 and whose clonal minus non-clonal LC difference (called the dFLC) are greater than 23mg/L will be recruited. Heavy chain type will not affect patient eligibility as long as the involved LC is λ type. The assay for free LC is a routine standard test for all patients with monoclonal gammopathies and will provide the basis for this screening.
Patients will learn about the trial through internet advertisements, contact the data manager, consent to participate in writing, and sign the HIPAA release forms allowing the PI to receive their medical data relevant to their λ LC SMM; standard physician-to-physician medical practice will apply here. Patient recruitment will be open to all eligible patients within the United States. Informed consent may be obtained in person or in a phone call with the PI. The patient's physician will be contacted and informed of the patient's consent and enrollment in this study and asked to provide medical records for screening. If the patient is found eligible, the patient and physician will be informed of the required samples needed for this protocol, which include a one-time donation of peripheral blood and a one-time donation of marrow aspirate, both taken during routine clinical procedures. Prepaid FedEx boxes will be provided by the study to ship research samples to Tufts Medical Center for remote patients.
Both the peripheral blood and marrow samples will be tested for the presence of variable region (VL) germline genes. Samples will be processed on the day of arrival and stored at -80 degrees Celsius. The results of these laboratory tests will be shared with the patient and their physician after both samples have been processed. No fu
Sponsor: Tufts Medical Center
Current Primary Outcome: Observation of disease progression in participants with AL-associated VL germline genes [ Time Frame: 5 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Tufts Medical Center
Dates:
Date Received: April 11, 2016
Date Started: April 2016
Date Completion: April 2023
Last Updated: January 9, 2017
Last Verified: January 2017
