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

September 8, 2021 updated by: Boston Medical Center

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

45

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Boston Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Patients with previously untreated AL amyloidosis

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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.
Other: blood collection
Other: bone marrow collection
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.
Other: blood collection
Other: bone marrow collection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Isolation of a plasma cell clone
Time Frame: 1 year
Number of samples that have a successful isolation of a plasma cell clone
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Minimal residual disease observed
Time Frame: 5 years
Number of cases in which minimal residual disease observed in specimens correlates
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Boston Medical Center

Investigators

  • Principal Investigator: Shayna Sarosiek, MD, Boston Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 21, 2016

Primary Completion (Actual)

September 4, 2020

Study Completion (Actual)

September 4, 2020

Study Registration Dates

First Submitted

March 14, 2016

First Submitted That Met QC Criteria

March 22, 2016

First Posted (Estimate)

March 23, 2016

Study Record Updates

Last Update Posted (Actual)

September 10, 2021

Last Update Submitted That Met QC Criteria

September 8, 2021

Last Verified

September 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • H-34469

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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