Clinical trial NCT02716103: Assessment of Minimal Residual Disease (MRD) After Antineoplastic Treatment in Patients With AL Amyloidosis
Official title: Assessment of Minimal Residual Disease (MRD) After Antineoplastic Treatment (Which May Include High Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT)) in Patients With AL Amyloidosis: Feasibility and Prognostic Significance
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Amyloidosis
Keywords: AL amyloidosis, systemic primary amyloidosis
Lead sponsor: Boston Medical Center
Sponsor class: OTHER
First posted: 2016-03-23
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with previously untreated AL amyloidosis

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.

Eligibility criteria: 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

Primary outcomes:
measure: Isolation of a plasma cell clone
timeFrame: 1 year
Secondary outcomes:
measure: Minimal residual disease observed
timeFrame: 5 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
Study officials:
affiliation: Boston Medical Center
name: Shayna Sarosiek, MD
role: PRINCIPAL_INVESTIGATOR
References: