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Assessment of Minimal Residual Disease (MRD) After Antineoplastic Treatment in Patients With AL Amyloidosis

Completed
Conditions
Amyloidosis
Registration Number
NCT02716103
Lead Sponsor
Boston Medical Center
Brief Summary

In this study, the investigators seek to evaluate bone marrow and blood samples and treatment responses to see if Minimal Residual Disease (MRD) can be used as a predictive method of response to treatment in amyloidosis.

Detailed Description

In this study, the investigators seek to evaluate bone marrow and blood samples and treatment responses to see if Minimal Residual Disease (MRD) (as described below), can be used as a predictive method of response to treatment in amyloidosis.

Minimal residual disease (MRD) is a concept that has gained significant value as a prognostic predictor and has become an emerging constituent of complete response (CR) reassessment in multiple myeloma (MM) patients. Studies in MM have demonstrated that up to 30% of patients achieving a CR after high-dose therapy will still have detectable MRD in the bone marrow as measured by standard-sensitivity flow cytometry or by molecular assays. Virtually every study examining MRD in MM has reported that among patients achieving a CR, those who were MRD negative (MRD-) had a significantly superior progression-free survival, with some studies reporting superior overall survival.

As amyloidosis is a disease that is very similar to multiple myeloma, the investigators wish to evaluate the concept in this disease.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
45
Inclusion Criteria
  • Biopsy-proven systemic AL amyloidosis defined as
  • At least one + Congo Red stain
  • Proof of a clonal plasma cell dyscrasia by:
  • Immunofixation electrophoresis (IFE) of the urine or serum
  • Light chain restriction based on Immunohistochemistry (IHC) in bone marrow plasma cells or in the amyloid tissue
  • Must be scheduled to undergo antineoplastic therapy (this may include high dose melphalan and Autologous Stem Cell Transplantation) for AL Amyloidosis (Part II enrollments only)
Exclusion Criteria
  • Co-existing Multiple Myeloma
  • Prior antineoplastic treatment for AL amyloidosis at time of enrollment.
  • Prior negative bone marrow biopsy showing no identifiable clone

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Isolation of a plasma cell clone1 year

Number of samples that have a successful isolation of a plasma cell clone

Secondary Outcome Measures
NameTimeMethod
Minimal residual disease observed5 years

Number of cases in which minimal residual disease observed in specimens correlates

Trial Locations

Locations (1)

Boston Medical Center

🇺🇸

Boston, Massachusetts, United States

Boston Medical Center
🇺🇸Boston, Massachusetts, United States

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