MyelofibrosisNCT02934477
Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
An observational study of Myelofibrosis, run by Center for International Blood and Marrow Transplant Research at 1 site in United States.
Trial summary
This observational study will compare outcomes of a prospectively-enrolled cohort of Hematopoietic Stem Cell Transplant (HCT) recipients with outcomes of a cohort of age-matched historical non-HCT controls. Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- Hematopoietic Stem Cell Transplant
- How many people
- 650 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2016to Oct 2027
- 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 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with primary MF (PMF), post-essential thrombocythemia (ET) MF, or post-polycythemia vera (PV) MF, with intermediate-2 or high-risk disease as determined by the DIPSS, and aged ≥55 at the time of DIPSS assessment are eligible for this study. For the alloHCT arm of the HLA-Matched Donor HCT Study, donors must be either 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high resolution DNA-based typing and Class II (HLA-DRBI) at high resolution DNA-based typing (but not monozygotic twins), OR an 8/8 HLA-A, -B, -C, and -DRB1 at high resolution DNA-based typing matched unrelated donors; both peripheral blood stem cells and bone marrow grafts are allowed, and all conditioning regimen intensities and GVHD prophylaxis regimens are allowed. For the Haploidentical Donor Study, donors must be haploidentical.
Who may be able to join
- Patients fulfilling the following criteria will be eligible for inclusion in the study:
- PMF, post-ET MF, or post-PV MF.
- Int-2 or high-risk disease as determined by the DIPSS.
- Age ≥55 at the time of DIPSS assessment.
- For the alloHCT arm:
- Donors must be a 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high resolution DNA-based typing and Class II (HLA-DRBI) at high resolution DNA-based typing (but not monozygotic twins) OR an 8/8 HLA-A, -B, -C, and -DRB1 at high resolution DNA-based typing matched unrelated donor identified through the National Marrow Donor Program (NMDP)/Be The Match. Donors must meet institutional or NMDP/Be The Match selection criteria; there is no age restriction for sibling donors.
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- Both peripheral blood stem cells and bone marrow grafts are allowed.
- All conditioning regimen intensities are allowed.
- All GVHD prophylaxis regimens are allowed.
- Haploidentical donors are allowed in the Haploidentical Donor Study
Who cannot join
- Patients with the following criteria will be ineligible for entry into the study:
- AlloHCT using umbilical cord blood unit(s) or HLA-mismatched adult donors (< 6/6 HLA alleles for related and < 8/8 HLA alleles for unrelated).
- Overlap syndromes.
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
This observational study will compare outcomes of a prospectively-enrolled cohort of Hematopoietic Stem Cell Transplant (HCT) recipients with outcomes of a cohort of age-matched historical non-HCT controls. Patients undergoing alloHCT will receive HCT in a US transplant center and be reported to the Center for International Blood and Marrow Transplant Research (CIBMTR) using well-established CIBMTR report forms and data collection procedures as well as a study-specific supplemental form. Data on the historical non-HCT controls will be collected at 14 US academic centers. These centers will provide data on all consecutive patients with PMF, post-ET MF, or post-PV MF referred to their institutions between 2000 and 2012.
Read the detailed description
Patients with primary MF (PMF), post-essential thrombocythemia (ET) MF, or post-polycythemia vera (PV) MF, with intermediate-2 or high-risk disease as determined by the DIPSS, and aged ≥55 at the time of DIPSS assessment are eligible for this study. For the allogeneic HCT arm of the HLA-Matched Donor HCT Study, donors must be either 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high resolution DNA-based typing and Class II (HLA-DRBI) at high resolution DNA-based typing (but not monozygotic twins), OR an 8/8 HLA-A, -B, -C, and -DRB1 at high resolution DNA-based typing matched unrelated donors; both peripheral blood stem cells and bone marrow grafts are allowed, and all conditioning regimen intensities and graph versus host disease (GVHD) prophylaxis regimens are allowed. For the Haploidentical Donor Study, donors must be haploidentical.
This study will target accrual of 650 patients receiving alloHCT, including approximately 225 receiving myeloablative conditioning. Participating centers are expected to provide data for approximately 2,400 patients to form the non-HCT historical control cohort.
- Conditions
- Myelofibrosis
- Being tested
- Hematopoietic Stem Cell Transplant
- Keywords
- Myelofibrosis, Hematopoietic Stem Cell Transplant, Medicare
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic Stem Cell TransplantOther
This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of age-matched non-HCT controls.
| Study group | Role | What they receive |
|---|---|---|
| Hematopoietic Stem Cell Transplant (HCT) | Patients undergoing alloHCT in a US transplant center and reported to the CIBMTR Receives: Other: Hematopoietic Stem Cell Transplant | |
| Non-HCT | Historical non-transplant controls collected from 14 US academic centers. Centers will provide data on all consecutive patients with PMF, post-ET MF, or post-PV MF referred to their institutions between 2000 and 2012. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare five year survival
Compare the five-year survival probabilities from DIPSS assessment between the two study arms: alloHCT recipients (arm 1) and non-HCT therapies (ruxolitinib / best supportive care) recipients (arm 2).
Measured: Five years post transplant
Secondary outcomes (10)
Compare leukemia-free survival
Compare leukemia-free survival at five years from DIPSS assessment date to the date of progression to AML or death from any cause, whichever comes first. Two co-secondary analyses will be conducted, one for all alloHCT patients versus Arm 2 and one for the subset of patients receiving MAC prior to alloHCT versus Arm 2. Observation is censored at the date of last follow-up for patients known to be alive without leukemia. Progression to AML is defined as >20% leukemia blasts in bone marrow or in the peripheral blood.
Measured: Five years post transplant
Cumulative incidences of chronic GVHD
Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD. Patients are censored at last follow-up or second transplant.
Measured: Five years post transplant
Cumulative incidences of acute GVHD
Occurrence of grade II, III, and/or IV skin, gastrointestinal, or liver abnormalities fulfilling the Consensus criteria of acute GVHD. Patients are censored at last follow-up or second transplant.
Measured: Five years post transplant
Cumulative incidence of treatment related mortality
Death from any cause in the first 28 days post-transplantation, irrespective of relapse status. Death beyond day +28 will only be considered transplant-related if the disease is in remission. This event will be summarized as a cumulative incidence estimate with relapse/persistence as the competing risk.
Measured: Five years post transplant
The impact of certain patient, disease and HCT related factors on survival in the alloHCT arm.
Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 years vs. >= 65 years, disease duration and DIPSS on overall survival in the alloHCT arm. The time to event in the analyses will start at the time of transplant.
Measured: Five years post transplant
The impact of certain patient, disease and HCT related factors on leukemia free survival in the alloHCT arm.
Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs. >= 65 years), disease duration and DIPSS on leukemia free survival in the alloHCT arm. The time to event in the analyses will start at the time of transplant.
Measured: Five years post transplant
The impact of certain patient, disease and HCT related factors on hematopoietic recovery in the alloHCT arm.
Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs >=65 years), disease duration and DIPSS on hematopoietic recovery in the alloHCT arm. The time to event in the analyses will start at the time of transplant.
Measured: Five years post transplant
The impact of certain patient, disease and HCT related factors on acute and chronic GVHD in the alloHCT arm.
Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs >= 65 years), disease duration and DIPSS on acute and chronic GVHD in the alloHCT arm. The time to event in the analyses will start at the time of transplant.
Measured: Five years post transplant
The impact of certain patient, disease and HCT related factors on treatment related mortality in the alloHCT arm.
Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs >=65 years), disease duration, and DIPSS on treatment related mortality in the alloHCT arm. The time to event in the analyses will start at the time of transplant.
Measured: Five years post transplant.
The impact of certain patient, disease and HCT related factors on relapse.
Evaluation of the impact of response to ruxolitinib therapy, patient age (65 vs >=65 years), disease duration and DIPSS on relapse in the alloHCT arm. The time to event in the analyses will start at the time of transplant.
Measured: Five 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 posted17 Oct 2016 (estimated)
- Study startNov 2016
- Primary completionNov 2026 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies 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
- 650 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Sponsor type
- Research network
- Collaborators
- National Marrow Donor Program, National Institutes of Health (NIH), National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Wael Saber, MD, Study chair, Medical College of WisconsinLaura Michaelis, MD, Study chair, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02934477
- Sponsor's protocol ID
- 16-CMS-MF
- Other IDs
- U24CA076518
- Status last verified
- Aug 2023
- First submitted
- 30 Sep 2016
- Last updated
- 30 Aug 2023
- Results posted
- No
- Data monitoring committee
- 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
- Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
- ClinicalTrials.gov ID
- NCT02934477
- Acronym / sponsor ID
- 16-CMS-MF
- Conditions
- Myelofibrosis
- Keywords
- Myelofibrosis, Hematopoietic Stem Cell Transplant, Medicare
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 650 participants (estimated)
- Lead sponsor
- Center for International Blood and Marrow Transplant ResearchResearch network
- Collaborators
- National Marrow Donor Program, National Institutes of Health (NIH), National Cancer Institute (NCI)
- Study start
- Nov 2016
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 17 Oct 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.
This observational study will compare outcomes of a prospectively-enrolled cohort of Hematopoietic Stem Cell Transplant (HCT) recipients with outcomes of a cohort of age-matched historical non-HCT controls. Patients undergoing alloHCT will receive HCT in a US transplant center and be reported to the Center for International Blood and Marrow Transplant Research (CIBMTR) using well-established CIBMTR report forms and data collection procedures as well as a study-specific supplemental form. Data on the historical non-HCT controls will be collected at 14 US academic centers. These centers will provide data on all consecutive patients with PMF, post-ET MF, or post-PV MF referred to their institutions between 2000 and 2012.
Detailed description
Patients with primary MF (PMF), post-essential thrombocythemia (ET) MF, or post-polycythemia vera (PV) MF, with intermediate-2 or high-risk disease as determined by the DIPSS, and aged ≥55 at the time of DIPSS assessment are eligible for this study. For the allogeneic HCT arm of the HLA-Matched Donor HCT Study, donors must be either 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high resolution DNA-based typing and Class II (HLA-DRBI) at high resolution DNA-based typing (but not monozygotic twins), OR an 8/8 HLA-A, -B, -C, and -DRB1 at high resolution DNA-based typing matched unrelated donors; both peripheral blood stem cells and bone marrow grafts are allowed, and all conditioning regimen intensities and graph versus host disease (GVHD) prophylaxis regimens are allowed. For the Haploidentical Donor Study, donors must be haploidentical.
This study will target accrual of 650 patients receiving alloHCT, including approximately 225 receiving myeloablative conditioning. Participating centers are expected to provide data for approximately 2,400 patients to form the non-HCT historical control cohort.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hematopoietic Stem Cell Transplant (HCT) | – | Other: Hematopoietic Stem Cell TransplantPatients undergoing alloHCT in a US transplant center and reported to the CIBMTR |
| Non-HCT | – | –Historical non-transplant controls collected from 14 US academic centers. Centers will provide data on all consecutive patients with PMF, post-ET MF, or post-PV MF referred to their institutions between 2000 and 2012. |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with primary MF (PMF), post-essential thrombocythemia (ET) MF, or post-polycythemia vera (PV) MF, with intermediate-2 or high-risk disease as determined by the DIPSS, and aged ≥55 at the time of DIPSS assessment are eligible for this study. For the alloHCT arm of the HLA-Matched Donor HCT Study, donors must be either 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high resolution DNA-based typing and Class II (HLA-DRBI) at high resolution DNA-based typing (but not monozygotic twins), OR an 8/8 HLA-A, -B, -C, and -DRB1 at high resolution DNA-based typing matched unrelated donors; both peripheral blood stem cells and bone marrow grafts are allowed, and all conditioning regimen intensities and GVHD prophylaxis regimens are allowed. For the Haploidentical Donor Study, donors must be haploidentical.
Key inclusion criteria (10 listed)
- Patients fulfilling the following criteria will be eligible for inclusion in the study:
Key exclusion criteria (3 listed)
- Patients with the following criteria will be ineligible for entry into the study:
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 |
|---|---|---|
| Hematopoietic Stem Cell TransplantThis observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of age-matched non-HCT controls. | Other | Hematopoietic Stem Cell Transplant (HCT) |
Primary outcome
Compare five year survival
Time frame: Five years post transplant
Compare the five-year survival probabilities from DIPSS assessment between the two study arms: alloHCT recipients (arm 1) and non-HCT therapies (ruxolitinib / best supportive care) recipients (arm 2).
10 secondary outcomes
Compare leukemia-free survival
Time frame: Five years post transplant
Cumulative incidences of chronic GVHD
Time frame: Five years post transplant
Cumulative incidences of acute GVHD
Time frame: Five years post transplant
Cumulative incidence of treatment related mortality
Time frame: Five years post transplant
The impact of certain patient, disease and HCT related factors on survival in the alloHCT arm.
Time frame: Five years post transplant
The impact of certain patient, disease and HCT related factors on leukemia free survival in the alloHCT arm.
Time frame: Five years post transplant
The impact of certain patient, disease and HCT related factors on hematopoietic recovery in the alloHCT arm.
Time frame: Five years post transplant
The impact of certain patient, disease and HCT related factors on acute and chronic GVHD in the alloHCT arm.
Time frame: Five years post transplant
The impact of certain patient, disease and HCT related factors on treatment related mortality in the alloHCT arm.
Time frame: Five years post transplant.
The impact of certain patient, disease and HCT related factors on relapse.
Time frame: Five 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.
- CIBMTRLink provided by the sponsor.
- 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.
Wael Saber, MD study chair
Medical College of Wisconsin
Laura Michaelis, MD study chair
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 Myelofibrosis, 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 10 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: 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 Patricia Steinert, PhD (414-805-0700, psteinert@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: Five 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.