NCT02716103

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
45

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Nov 2016

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 14, 2016

Completed
9 days until next milestone

First Posted

Study publicly available on registry

March 23, 2016

Completed
8 months until next milestone

Study Start

First participant enrolled

November 21, 2016

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 4, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 4, 2020

Completed
Last Updated

September 10, 2021

Status Verified

September 1, 2021

Enrollment Period

3.8 years

First QC Date

March 14, 2016

Last Update Submit

September 8, 2021

Conditions

Amyloidosis

Keywords

AL amyloidosis, systemic primary amyloidosis

Outcome Measures

Primary Outcomes (1)

  • Isolation of a plasma cell clone

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

    1 year

Secondary Outcomes (1)

  • Minimal residual disease observed

    5 years

Study Arms (2)

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 collectionOther: 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 collectionOther: bone marrow collection

Interventions

blood collectionOTHER
2nd Cohort - pre-treatmentInitial Cohort: feasibility
bone marrow collectionOTHER
2nd Cohort - pre-treatmentInitial Cohort: feasibility

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Patients with previously untreated AL amyloidosis

You may qualify if:

  • 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)

You may not qualify if:

  • Co-existing Multiple Myeloma
  • Prior antineoplastic treatment for AL amyloidosis at time of enrollment.
  • Prior negative bone marrow biopsy showing no identifiable clone

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Bone marrow and peripheral blood

MeSH Terms

Conditions

AmyloidosisImmunoglobulin Light-chain Amyloidosis

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Proteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesNeoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemias

Intervention Hierarchy (Ancestors)

Specimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques

Study Officials

  • Shayna Sarosiek, MD

    Boston Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 14, 2016

First Posted

March 23, 2016

Study Start

November 21, 2016

Primary Completion

September 4, 2020

Study Completion

September 4, 2020

Last Updated

September 10, 2021

Record last verified: 2021-09

Data Sharing

IPD Sharing
Will not share

Locations

US(1)