Multiple MyelomaNCT03127761
Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma
An observational study of Multiple Myeloma, run by Center for International Blood and Marrow Transplant Research at 1 site in United States.
Trial summary
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- Allogeneic Hematopoietic Stem Cell Transplant
- How many people
- 544 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jul 2017to Apr 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: A prospective cohort study of approximately 544 patients receiving allogeneic HCT for multiple myeloma in CIBMTR centers in the US matched to a historical control patients treated with autoHCT between 2010 and 2016
Who may be able to join
- Medicare beneficiary
- Stage II or III multiple myeloma and/or primary plasma cell leukemia
- Eligible to receive an allogeneic HCT from any suitable allogeneic donor (as determined by the transplant center) including umbilical cord blood
- Will receive allogeneic HCT at a US transplant center
- Agree to submit comprehensive clinical data on their pre- and post-transplant clinical status and outcomes to the CIBMTR
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Center for International Blood and Marrow Transplant Research
Minneapolis, Minnesota, 55401
Michael Tierney, DatabaseIRB@NMDP.org
About this study
Official title: Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma: A Study to Develop Evidence of Effectiveness for the Centers for Medicare and Medicaid Services (CMS)
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients.
Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.
Read the detailed description
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. Overall survival (OS) in MM has improved significantly in the last 15 years with the emergence of novel therapies such as thalidomide, bortezomib and lenalidomide. The median life expectancy of patients with MM treated in the current era is more than 6 years, while SEER data from a slightly earlier time period (2008-12) estimated the 5 year survival at 48.5%. However, prognosis is not uniform and varies considerably based on a presenting features and response to therapy.
The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). There is controversy regarding the timing of autoHCT after initial novel therapy induction with randomized trials showing similar OS whether done early or delayed to time of relapse as salvage therapy. However, more recent trials comparing early versus delayed transplant support the benefit of early upfront autoHCT.
Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients. Current data from the Center for International Blood and Marrow Research (CIBMTR) show transplant-related mortality rates of 23 (20-26)% at 5 years with myeloablative conditioning.
Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.
- Conditions
- Multiple Myeloma
- Being tested
- Allogeneic Hematopoietic Stem Cell Transplant
- Keywords
- Multiple Myeloma
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Hematopoietic Stem Cell TransplantOther
This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of matched autoHCT controls.
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic HCT | Prospectively enrolled cohort of patients receiving allogeneic hematopoietic cell transplantation for multiple myeloma Receives: Other: Allogeneic Hematopoietic Stem Cell Transplant | |
| Historical autoHCT | Historical cohort of patients with autologous hematopoietic cell transplantation between 2010 and 2016 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare five-year survival
Compare five-year overall survival between the alloHCT cohort and an age and disease risk matched cohort of autoHCT patients
Measured: 5 years post transplant
Secondary outcomes (5)
Progression-free survival (PFS)
five-year PFS probabilities between the AlloHCT cohort and an age and disease risk matched cohort of autoHCT patients
Measured: 5 years post transplant
Relapse or progression
Myeloma recurrence or progression will be defined per International Myeloma Working Group (IMWG) guidelines
Measured: 5 years post transplant
Transplant related mortality
Death from any cause within 28 days after alloHCT or death in the absence of progression/relapse of MM after day 28 post transplant
Measured: 5 years post transplant
Incidence of acute GVHD
Occurrence of Grade I, II and III/IV skin, gastrointestinal, or liver abnormalities fulfilling the Consensus criteria of Grades II-IV acute GVHD
Measured: 5 years post transplant
Incidence of chronic GVHD
Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD
Measured: 5 years post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2017
- Study start25 Jul 2017
- Primary completionMay 2027 (estimated)
- Study completionApr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- 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
- 544 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Sponsor type
- Research network
- Collaborators
- National Marrow Donor Program
- Responsible party
- Sponsor
- Investigators
- Anita D'Souza, MD, MS, Study chair, CIBMTR, Medical College of WisconsinParameswaran Hari, MD, MS, Study chair, CIBMTR, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03127761
- Sponsor's protocol ID
- 17-CMS-MM
- Status last verified
- Aug 2023
- First submitted
- 18 Apr 2017
- Last updated
- 30 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma: A Study to Develop Evidence of Effectiveness for the Centers for Medicare and Medicaid Services (CMS)
- ClinicalTrials.gov ID
- NCT03127761
- Acronym / sponsor ID
- 17-CMS-MM
- Conditions
- Multiple Myeloma
- Keywords
- Multiple Myeloma
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 544 participants (estimated)
- Lead sponsor
- Center for International Blood and Marrow Transplant ResearchResearch network
- Collaborators
- National Marrow Donor Program
- Study start
- 25 Jul 2017 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- Apr 2028 (estimated)
- First posted
- 25 Apr 2017 (actual)
- 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.
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients.
Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.
Detailed description
Multiple myeloma (MM) is the second most common hematologic malignancy in adults. Overall survival (OS) in MM has improved significantly in the last 15 years with the emergence of novel therapies such as thalidomide, bortezomib and lenalidomide. The median life expectancy of patients with MM treated in the current era is more than 6 years, while SEER data from a slightly earlier time period (2008-12) estimated the 5 year survival at 48.5%. However, prognosis is not uniform and varies considerably based on a presenting features and response to therapy.
The current standard of care for MM patients fit to undergo high dose conditioning chemotherapy is an autologous HCT (autoHCT). There is controversy regarding the timing of autoHCT after initial novel therapy induction with randomized trials showing similar OS whether done early or delayed to time of relapse as salvage therapy. However, more recent trials comparing early versus delayed transplant support the benefit of early upfront autoHCT.
Allogeneic HCT (alloHCT) is the only potentially curative therapy available to patients with MM. However, the significant morbidity and mortality of this procedure historically limited its application in older patients. Current data from the Center for International Blood and Marrow Research (CIBMTR) show transplant-related mortality rates of 23 (20-26)% at 5 years with myeloablative conditioning.
Thus, although potentially curative, standard risk MM patients have excellent prognoses in the era of novel therapies which reduces the overall benefit of alloHCT. However, because the outcomes for high-risk MM remain poor despite the best available standard therapies (overall survival of 24-36 months), initial data suggest that alloHCT should be explored in this subset.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Allogeneic HCT | – | Other: Allogeneic Hematopoietic Stem Cell TransplantProspectively enrolled cohort of patients receiving allogeneic hematopoietic cell transplantation for multiple myeloma |
| Historical autoHCT | – | –Historical cohort of patients with autologous hematopoietic cell transplantation between 2010 and 2016 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- A prospective cohort study of approximately 544 patients receiving allogeneic HCT for multiple myeloma in CIBMTR centers in the US matched to a historical control patients treated with autoHCT between 2010 and 2016
Key inclusion criteria (5 listed)
- Medicare beneficiary
- Stage II or III multiple myeloma and/or primary plasma cell leukemia
- Eligible to receive an allogeneic HCT from any suitable allogeneic donor (as determined by the transplant center) including umbilical cord blood
- Will receive allogeneic HCT at a US transplant center
- Agree to submit comprehensive clinical data on their pre- and post-transplant clinical status and outcomes to the CIBMTR
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Allogeneic Hematopoietic Stem Cell TransplantThis observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of matched autoHCT controls. | Other | Allogeneic HCT |
Primary outcome
Compare five-year survival
Time frame: 5 years post transplant
Compare five-year overall survival between the alloHCT cohort and an age and disease risk matched cohort of autoHCT patients
5 secondary outcomes
Progression-free survival (PFS)
Time frame: 5 years post transplant
Relapse or progression
Time frame: 5 years post transplant
Transplant related mortality
Time frame: 5 years post transplant
Incidence of acute GVHD
Time frame: 5 years post transplant
Incidence of chronic GVHD
Time frame: 5 years post transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 2023.
- Individual participant dataSharing planned: Undecided
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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.
Anita D'Souza, MD, MS study chair
CIBMTR, Medical College of Wisconsin
Parameswaran Hari, MD, MS study chair
CIBMTR, Medical College of Wisconsin
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 Multiple Myeloma, run by Center for International Blood and Marrow Transplant Research 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 5 inclusion and 0 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: looking for participants now, as of the sponsor's last update on 30 Aug 2023. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mona Patel (414-805-0655, mopatel@mcw.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Compare five-year survival (time frame: 5 years post transplant). 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 30 Aug 2023.