AmyloidosisMRDNCT02716103
Assessment of Minimal Residual Disease (MRD) After Antineoplastic Treatment in Patients With AL Amyloidosis
An observational study of Amyloidosis, run by Boston Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis
- What is being tested?
- blood collection and bone marrow collection
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Nov 2016to 4 Sep 2020
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with previously untreated AL amyloidosis
Who may be able to join
- 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)
Who cannot join
- Co-existing Multiple Myeloma
- Prior antineoplastic treatment for AL amyloidosis at time of enrollment.
- Prior negative bone marrow biopsy showing no identifiable clone
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Amyloidosis
- Being tested
- blood collection, bone marrow collection
- Keywords
- AL amyloidosis, systemic primary amyloidosis
- Medical subject headings
- Amyloidosis, Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood collectionOther
bone marrow collectionOther
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: blood collection; Other: bone marrow collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Isolation of a plasma cell clone
Number of samples that have a successful isolation of a plasma cell clone
Measured: 1 year
Secondary outcomes (1)
Minimal residual disease observed
Number of cases in which minimal residual disease observed in specimens correlates
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2016 (estimated)
- Study start21 Nov 2016
- Primary completion4 Sep 2020
- Study completion4 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AmyloidosisStudies of the same condition, open and under way.
- More trials run by Boston Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 45 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Boston Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Shayna Sarosiek, MD, Principal investigator, Boston Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02716103
- Sponsor's protocol ID
- H-34469
- Acronym
- MRD
- Status last verified
- Sep 2021
- First submitted
- 14 Mar 2016
- Last updated
- 10 Sep 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02716103
- Acronym / sponsor ID
- MRD / H-34469
- Conditions
- Amyloidosis
- Keywords
- AL amyloidosis, systemic primary amyloidosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Boston Medical CenterUniversity, hospital or other
- Study start
- 21 Nov 2016 (actual)
- Primary completion
- 4 Sep 2020 (actual)
- Study completion
- 4 Sep 2020 (actual)
- First posted
- 23 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Initial Cohort: feasibility | – | Other: blood collection, Other: bone marrow collectionBone 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. |
| 2nd Cohort - pre-treatment | – | Other: blood collection, Other: bone marrow collectionIf 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with previously untreated AL amyloidosis
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- Co-existing Multiple Myeloma
- Prior antineoplastic treatment for AL amyloidosis at time of enrollment.
- Prior negative bone marrow biopsy showing no identifiable clone
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| blood collection | Other | 2nd Cohort - pre-treatment, Initial Cohort: feasibility |
| bone marrow collection | Other | 2nd Cohort - pre-treatment, Initial Cohort: feasibility |
Primary outcome
Isolation of a plasma cell clone
Time frame: 1 year
Number of samples that have a successful isolation of a plasma cell clone
1 secondary outcome
Minimal residual disease observed
Time frame: 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shayna Sarosiek, MD principal investigator
Boston Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyloidosis, run by Boston Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Isolation of a plasma cell clone (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2021.