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

NCT ID: NCT02716103

Last Updated: 2021-09-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

45 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-11-21

Study Completion Date

2020-09-04

Brief Summary

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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

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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.

Conditions

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Amyloidosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Initial Cohort: feasibility

Bone marrow collection and peripheral blood collection from ten patients with untreated AL amyloidosis will be evaluated to determine feasibility of isolating a plasma cell clone. An additional three teaspoons of bone marrow and 4 teaspoons of blood will be collected at the time of standard blood draw and bone marrow biopsy before receiving any treatment. There will be no extra procedures or visits specifically for this research.

blood collection

Intervention Type OTHER

bone marrow collection

Intervention Type OTHER

2nd Cohort - pre-treatment

If feasibility is determined with initial cohort, bone marrow collection and peripheral blood collection from 20 patients with untreated AL amyloidosis who are scheduled to undergo antineoplastic therapy will be evaluated to isolate a plasma cell clone. An additional 3 teaspoons of bone marrow and 4 teaspoons of blood will be collected at the time of standard blood draw and bone marrow biopsy before receiving therapy. For those who complete therapy and achieve complete response or very good partial response, subsequent samples of bone marrow and peripheral blood will be sent for minimal residual disease detection (based on the previously identified cancer clone) at 6 to 12 months post treatment.

blood collection

Intervention Type OTHER

bone marrow collection

Intervention Type OTHER

Interventions

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blood collection

Intervention Type OTHER

bone marrow collection

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Boston Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Shayna Sarosiek, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Medical Center

Locations

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Boston Medical Center

Boston, Massachusetts, United States

Site Status

Countries

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United States

Other Identifiers

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H-34469

Identifier Type: -

Identifier Source: org_study_id

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