Myelodysplastic Syndromes (MDS)NCT02390414
The Myelodysplasia Transplantation-Associated Outcomes (MDS-TAO) Study
An observational study of Myelodysplastic Syndromes (MDS), run by Dana-Farber Cancer Institute at 3 sites in United States.
Trial summary
The purpose of this observational study is to compare overall survival in older adults with myelodysplastic syndromes (MDS) who receive reduced intensity conditioning hematopoietic stem cell transplant (RIC HSCT) versus those who do not receive HSCT. Read more about this study
- Who is it for?
- Ages 60 to 75 years
- Conditions the study is for
- Myelodysplastic Syndromes (MDS)
- How many people
- 290 participants
- Where
- 3 sitesUnited States
- Study dates
- May 2011to Sep 2020
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 60 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Eligible patients will be 60 to 75 years old with a diagnosis of MDS or related disorder (eg, MDS/MPD or CMML) who have disease that is advanced enough to warrant RIC HSCT (defined by high-risk cytogenetics OR int-2 or high-risk on IPSS OR transfusion dependence as defined by WPSS) and who are physically fit enough to undergo RIC HSCT as assessed by pre-determined measures of organ function. Patients whose baseline donor status is known will be excluded; however, knowledge of HLA status is allowed as long as a donor search has not been performed.
Who may be able to join
- Histologically-confirmed diagnosis of:
- Primary or secondary MDS using the World Health Organization (WHO) 2008 classification:
- Refractory cytopenia with unilineage dysplasia
- Refractory Anemia (RA)
- Refractory Neutropenia (RN)
- Refractory Thrombocytopenia (RT)
Show all 44Show fewer
- Refractory Anemia with Ring Sideroblasts (RARS)
- Refractory Cytopenia with Multilineage Dysplasia (RCMD)
- Refractory Anemia with Excess Blasts-1 (RAEB-1)
- Refractory Anemia with Excess Blasts-2 (RAEB-2)
- MDS with isolated del (5q)
- MDS-Unclassified (MDS-U)
- Another of the following related disorders:
- Chronic Myelomonocytic leukemia (CMML)
- Myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-U)
- Age 60 to 75 years
- Any of the following (high-risk characteristics):
- Intermediate-2 or High-Risk on International Prognostic Scoring System (IPSS)
- Secondary MDS (any karyotype)
- Documented non-IPSS intermediate- or poor-prognosis karyotype including:
- +8
- t(11q23)
- Rea 3q
- +19
- 3 or greater abnormalities
- del(7q)
- -5
- t(5q)
- Documented significant cytopenia for at least four months prior to enrollment, defined by the following criteria:
- Red Blood Cell (RBC) Transfusion Dependence: four or more units of RBC transfusions within an eight-week period for symptomatic anemia with hemoglobin of ≤ 9.0 g/dL; OR
- Severe Anemia: average of two or more hemoglobin values ≤ 8 g/dL within an eight-week period not influenced by RBC transfusions (i.e., must be seven days post transfusion); OR
- Severe Thrombocytopenia: average of two or more platelet counts ≤ 50
- × 109/L within an eight-week period not influenced by platelet transfusions (i.e., must be at least three days post- transfusion) or a clinically significant hemorrhage requiring platelet transfusions within the prior four months; OR
- Severe Neutropenia: average of two or more absolute neutrophil counts (ANC) ≤ 500 within an eight-week period, or a clinically significant infection requiring IV antibiotics in the setting of ANC ≤ 1000 within the prior four months.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
- Adequate organ function to permit RIC HSCT as indicated by the following:
- Serum bilirubin ≤ 2.5 mg/dL (except when Gilbert's syndrome or MDS-related hemolysis suspected).
- Aspartate aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 2.5 times the upper limit of normal (ULN).
- Serum creatinine ≤ 2.0 mg/dL.
- Seemingly sufficient baseline cardiac function to undergo HSCT (no echocardiogram required).
- Seemingly sufficient baseline pulmonary function to undergo HSCT (no pulmonary function tests required).
- Seemingly sufficient neuro-psychiatric function to undergo HSCT (no specific neuro-psychiatric evaluation required).
- Willingness to undergo human leukocyte antigen (HLA)-typing and consider subsequent HSCT.
- Willingness and ability to give informed consent.
Who cannot join
- Known baseline conversion to AML (eg, ≥ 20% peripheral or marrow blasts).
- Knowledge of potential donor status at study entry. Of note, knowledge of HLA status WITHOUT a related or unrelated search is allowed.
- History of prior malignancy within the past year, except for adequately-treated carcinoma in situ of uterine cervix, basal cell or squamous cell skin cancer.
- Any severe concurrent disease, infection, or comorbidity that, in the judgment of the principal investigator, would make the patient inappropriate for HSCT at the time of study entry.
- Psychiatric disorders including dementia that would preclude obtaining informed consent or the ability to participate in an ongoing research study.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
About this study
The purpose of this observational study is to compare overall survival in older adults with myelodysplastic syndromes (MDS) who receive reduced intensity conditioning hematopoietic stem cell transplant (RIC HSCT) versus those who do not receive HSCT.
Read the detailed description
MDS is a hematologic malignancy characterized by cytopenias, bone marrow failure, and a risk of transformation to acute myeloid leukemia (AML). HSCT is the only curative therapy for MDS. Despite its increasing use among older patients (age greater than 60), more data are needed to assess outcomes of HSCT in older adults compared to other therapies.
In this observational study, patients with MDS presenting at the study institutions are screened for disease characteristics that indicate that they are potentially appropriate for HSCT (both high-risk disease and fit for the procedure). Patients who meet inclusion and exclusion criteria and agree to participate in the study are entered into a clinical database and followed for overall survival. Patients also complete quality of life (QoL) assessments at enrollment and two years afterward, with the goal of investigating potential relationships between QoL and MDS treatment (HSCT vs. non-HSCT strategies).
- Conditions
- Myelodysplastic Syndromes (MDS)
- Keywords
- Myelodysplastic syndromes (MDS)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Gets HSCT | Patients with higher-risk myelodysplastic syndrome (MDS) aged 60-75 who are fit for HSCT and actually undergo HSCT. | |
| No HSCT | Patients with higher-risk myelodysplastic syndrome (MDS) aged 60-75 who are fit for HSCT but do not undergo HSCT. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
To prospectively compare the overall survival of patients in the HSCT group to that of patients in the non-HSCT group.
Measured: 3 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2015 (estimated)
- Study startMay 2011
- Primary completionSep 2020
- Study completionSep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic Syndromes (MDS)Studies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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
- 290 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gregory A. Abel, MD, Dana-Farber Cancer Institute
- Investigators
- Gregory A Abel, MD, MPH, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02390414
- Sponsor's protocol ID
- 11-056
- Status last verified
- Mar 2023
- First submitted
- 11 Mar 2015
- Last updated
- 3 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Myelodysplasia Transplantation-Associated Outcomes (MDS-TAO) Study
- ClinicalTrials.gov ID
- NCT02390414
- Acronym / sponsor ID
- 11-056
- Conditions
- Myelodysplastic Syndromes (MDS)
- Keywords
- Myelodysplastic syndromes (MDS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 290 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Study start
- May 2011 (actual)
- Primary completion
- Sep 2020 (actual)
- Study completion
- Sep 2020 (actual)
- First posted
- 17 Mar 2015 (estimated)
- Results
- Posted 3 Apr 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this observational study is to compare overall survival in older adults with myelodysplastic syndromes (MDS) who receive reduced intensity conditioning hematopoietic stem cell transplant (RIC HSCT) versus those who do not receive HSCT.
Detailed description
MDS is a hematologic malignancy characterized by cytopenias, bone marrow failure, and a risk of transformation to acute myeloid leukemia (AML). HSCT is the only curative therapy for MDS. Despite its increasing use among older patients (age greater than 60), more data are needed to assess outcomes of HSCT in older adults compared to other therapies.
In this observational study, patients with MDS presenting at the study institutions are screened for disease characteristics that indicate that they are potentially appropriate for HSCT (both high-risk disease and fit for the procedure). Patients who meet inclusion and exclusion criteria and agree to participate in the study are entered into a clinical database and followed for overall survival. Patients also complete quality of life (QoL) assessments at enrollment and two years afterward, with the goal of investigating potential relationships between QoL and MDS treatment (HSCT vs. non-HSCT strategies).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Gets HSCT | – | –Patients with higher-risk myelodysplastic syndrome (MDS) aged 60-75 who are fit for HSCT and actually undergo HSCT. |
| No HSCT | – | –Patients with higher-risk myelodysplastic syndrome (MDS) aged 60-75 who are fit for HSCT but do not undergo HSCT. |
Who may qualify
- Ages
- 60 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Eligible patients will be 60 to 75 years old with a diagnosis of MDS or related disorder (eg, MDS/MPD or CMML) who have disease that is advanced enough to warrant RIC HSCT (defined by high-risk cytogenetics OR int-2 or high-risk on IPSS OR transfusion dependence as defined by WPSS) and who are physically fit enough to undergo RIC HSCT as assessed by pre-determined measures of organ function. Patients whose baseline donor status is known will be excluded; however, knowledge of HLA status is allowed as long as a donor search has not been performed.
Key inclusion criteria (44 listed)
- Histologically-confirmed diagnosis of:
- Age 60 to 75 years
- Any of the following (high-risk characteristics):
- × 109/L within an eight-week period not influenced by platelet transfusions (i.e., must be at least three days post- transfusion) or a clinically significant hemorrhage requiring platelet transfusions within the prior four months; OR
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
Key exclusion criteria (5 listed)
- Known baseline conversion to AML (eg, ≥ 20% peripheral or marrow blasts).
- Knowledge of potential donor status at study entry. Of note, knowledge of HLA status WITHOUT a related or unrelated search is allowed.
- History of prior malignancy within the past year, except for adequately-treated carcinoma in situ of uterine cervix, basal cell or squamous cell skin cancer.
- Any severe concurrent disease, infection, or comorbidity that, in the judgment of the principal investigator, would make the patient inappropriate for HSCT at the time of study entry.
- Psychiatric disorders including dementia that would preclude obtaining informed consent or the ability to participate in an ongoing research study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Overall Survival
Time frame: 3 Years
To prospectively compare the overall survival of patients in the HSCT group to that of patients in the non-HSCT group.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Fit older adults with advanced myelodysplastic syndromes: who is most likely to benefit from transplant?Link provided by the sponsor.
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Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
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Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
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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.
Gregory A Abel, MD, MPH principal investigator
Dana-Farber Cancer Institute
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes (MDS), run by Dana-Farber Cancer Institute at 3 sites 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 44 inclusion and 5 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 3 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
Yes. Results are posted in the registry (first posted 3 Apr 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Survival (time frame: 3 Years). 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 3 Apr 2023.