A Diagnostic Screening Trial Seeking AL Amyloidosis Very Early
| Status: | Recruiting |
|---|---|
| Conditions: | Hematology, Hematology, Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 1/11/2017 |
| Start Date: | April 2016 |
| End Date: | April 2023 |
| Contact: | Raymond Comenzo, MD |
| Email: | rcomenzo@tuftsmedicalcenter.org |
| Phone: | 617-636-6454 |
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.
(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.
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 further samples will be
requested from the participant at any time as a part of this study.
The analysis of this trial will be based on the frequency with which λ LC SMM patients are
found to have genes associated with light chain amyloidosis (AL) and are diagnosed with AL,
or have AL-associated genes and progress to AL. The diagnosis of AL is a tissue diagnosis.
If amyloidosis is suspected at any time during a subject's participation, their physician
will be contacted and given recommendations regarding diagnostic tests and disease staging.
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 further samples will be
requested from the participant at any time as a part of this study.
The analysis of this trial will be based on the frequency with which λ LC SMM patients are
found to have genes associated with light chain amyloidosis (AL) and are diagnosed with AL,
or have AL-associated genes and progress to AL. The diagnosis of AL is a tissue diagnosis.
If amyloidosis is suspected at any time during a subject's participation, their physician
will be contacted and given recommendations regarding diagnostic tests and disease staging.
Inclusion Criteria:
- SMM patients with λ LC involvement with dFLC greater than 23mg/L and κ::λ free LC
ratios < 0.26. Subjects must be able to share their medical records and ship us bone
marrow and blood samples.
Exclusion Criteria:
- λ SMM patients with light chains that do not meet the inclusion criteria as well as
patients with monoclonal gammopathy of undetermined significance (MGUS), SMM with
kappa LC involvement or active myeloma will not be included. And patients who are
unable to send us blood and marrow for any reason will not be eligible.
We found this trial at
1
site
800 Washington St
Boston, Massachusetts 02111
Boston, Massachusetts 02111
(617) 636-5000
Phone: 617-636-5907
Tufts Medical Center Tufts Medical Center is an internationally-respected academic medical center – a teaching...
Click here to add this to my saved trials
