CLINICAL TRIAL

Total-Body Irradiation for Myelodysplastic Syndromes

1 Prior Treatment
Relapsed
Waitlist Available · Any Age · All Sexes · Seattle, WA

This study is evaluating whether a combination of chemotherapy and total body irradiation before a stem cell transplant works better than chemotherapy and total body irradiation before a stem cell transplant.

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About the trial for Myelodysplastic Syndromes

Eligible Conditions
Myelodysplastic-Myeloproliferative Diseases · Chronic Myelomonocytic Leukemia (CMML) · Minimal Residual Disease · Myelodysplastic/Myeloproliferative Neoplasms · Syndrome · Myelodysplastic Syndromes (MDS) · Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable · Myeloproliferative Disorders · Acute myeloid leukaemia (in remission) · Leukemia · Leukemia, Myeloid · Preleukemia · Leukemia, Myelomonocytic, Juvenile · Neoplasm, Residual · Leukemia, Myeloid, Acute · Leukemia, Myelomonocytic, Chronic · Myelodysplastic Syndromes · Neoplasms

Treatment Groups

This trial involves 2 different treatments. Total-Body Irradiation is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Experimental Group 1
Total-Body Irradiation
RADIATION
+
Laboratory Biomarker Analysis
OTHER
+
Fludarabine Phosphate
DRUG
+
Allogeneic Bone Marrow Transplantation
PROCEDURE
+
Treosulfan
DRUG
+
Peripheral Blood Stem Cell Transplantation
PROCEDURE
Experimental Group 2
Laboratory Biomarker Analysis
OTHER
+
Fludarabine Phosphate
DRUG
+
Allogeneic Bone Marrow Transplantation
PROCEDURE
+
Treosulfan
DRUG
+
Peripheral Blood Stem Cell Transplantation
PROCEDURE

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Total-Body Irradiation
1997
Completed Phase 3
~1220
Fludarabine
FDA approved
Allogeneic Bone Marrow Transplantation
2009
Completed Phase 2
~440
Treosulfan
FDA approved
Peripheral Blood Stem Cell Transplantation
1997
Completed Phase 3
~1970

Eligibility

This trial is for patients born any sex of any age. You must have received 1 prior treatment for Myelodysplastic Syndromes or one of the other 17 conditions listed above. There are 9 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
(MRD) <0.1% by multiparameter flow cytometry (MFC) Leukemia other than APL that is in either first or second remission and has a minimal residual disease level of less than 0.1% by multiparameter flow cytometry. show original
A person who donates blood or bone marrow for another person is called a donor show original
DONOR: Donors able to undergo peripheral blood stem cell collection or bone marrow harvest
and without active cancer, are eligible for a kidney donation show original
The text explains that a donor is someone who is able to give informed consent, or with a legal guardian who can giveinformed consent, if the donor is younger than 18 years old. show original
MDS, myelodysplastic syndrome/myeloproliferative neoplasia overlap disorders (including chronic myelomonocytic leukemia [CMML], and MDS/myeloproliferative neoplasm [MPN] unclassifiable syndromes)
: If your Karnofsky index or Lansky Play-Performance scale is greater than 70% on your pre-transplant evaluation, you are likely to have a good outcome after transplant. show original
If you are 18 years old or older, you are able to give informed consent show original
in this study -People who have had a previous bone marrow transplant using their own cells (autologous) or someone else's cells (allogeneic) are allowed to enroll in this study. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 5 year
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 5 year
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 5 year.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Total-Body Irradiation will improve 1 primary outcome and 7 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of Baseline and at day 0 within 6 hours of conditioning prior to transplant.

Relapse Risk as Measured by Degree of Change in Gene Expression Profiles
BASELINE AND AT DAY 0 WITHIN 6 HOURS OF CONDITIONING PRIOR TO TRANSPLANT
Among genes identified whose expression is modified by conditioning, degree of change in expression will be evaluated to determine if it is correlated with relapse risk and offers improved prediction of relapse risk over that obtained with standard clinical parameters (cytogenetics, blast count, International Prognostic Scoring System score, minimal residual disease. To account for censoring and the competing risk of non-relapse mortality (NRM), the analysis will be a time-to-event analysis of relapse using Cox regression, with change in expression as a continuous covariate (on a log scale). 8
BASELINE AND AT DAY 0 WITHIN 6 HOURS OF CONDITIONING PRIOR TO TRANSPLANT
Change in Gene Expression Profiles
BASELINE AND AT DAY 0 WITHIN 6 HOURS OF CONDITIONING PRIOR TO TRANSPLANT
Differences between arms in the changes in gene expression will be compared. 80% power to detect mean differences of approximately 1.4 standard deviation units, at the 2-sided 0.05 level of significance (with Bonferroni correction for 50 genes).
BASELINE AND AT DAY 0 WITHIN 6 HOURS OF CONDITIONING PRIOR TO TRANSPLANT
Number of Participants With Acute GVHD, Graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
UP TO 84 DAYS
UP TO 84 DAYS
Number of Participants That Did Not Progress Within 6 Months
AT 6 MONTHS POST-TRANSPLANT
Progression is defined as relapse
AT 6 MONTHS POST-TRANSPLANT
Overall Survival (OS)
UP TO 2 YEAR
UP TO 2 YEAR
NRM
UP TO 5 YEARS
UP TO 5 YEARS
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Who is running the study

Principal Investigator
J. D.
Joachim Deeg, Principal Investigator
Fred Hutchinson Cancer Center

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is myelodysplastic syndromes?

The MDS diagnostic criteria for the WHO require the presence of one or more of the following 3 features: \n• ≤ 35% blasts in blood and/or bone marrow,\n• ≤ 35% platelet count,\n• > 100 x 10/l in peripheral blood blasts\n\nThis presentation consists of 5 criteria: \n• Cytological features,\n• Cytological features\n• Cytogenetic abnormalities,\n• Unfavourable haematological features\n• Cytogenetic abnormalities\n\nMDS may be associated with or caused by a number of identifiable genes, such as the 5q- and 7q- chromosome abnormalities.

Anonymous Patient Answer

Can myelodysplastic syndromes be cured?

MDS cannot be cured, but some have shown improvement and some have stopped disease progression with appropriate treatment. Patients with MDS are more likely to have improved symptoms with treatment.

Anonymous Patient Answer

How many people get myelodysplastic syndromes a year in the United States?

Around 10.4 million individuals are diagnosed with at least one myelodysplastic syndrome in the United States each a year. This makes up 2.7% of American adults.

Anonymous Patient Answer

What are the signs of myelodysplastic syndromes?

The presenting history is important and should include the date of onset, the frequency and duration of changes in the disease course, and whether any associated symptoms are present. A full blood count is helpful as well as further testing for erythropoietin resistance.

Anonymous Patient Answer

What are common treatments for myelodysplastic syndromes?

The use of conventional cytotoxic therapy and peripheral blood stem cell transplant as well as autologous stem cell transplant, are common treatments for MDS. Other options for treatment of refractory disease in selected patients, including immunomodulation, anti-angiogenic agents and the application of agents directed against JAK/STAT signalling have been described in the treatment of MDS.

Anonymous Patient Answer

What causes myelodysplastic syndromes?

The aetiology of MDS is not understood, but some factors may be modifiable. Dietary supplementation with folate and vitamin B12 might protect people with MDS against progression to acute myeloid leukemia.

Anonymous Patient Answer

What is the average age someone gets myelodysplastic syndromes?

The average age at diagnosis of MDS in Scotland is 67.2 years, with an increase in the proportion of patients diagnosed at more advanced ages from the 1990s to the current decade. The proportion of patients over the age of 70 years had increased over the period 1994-2014, irrespective of diagnosis criteria or method of referral, and increased by an average 4.7 years compared with a similar period in 2003-04. The age distribution of MDS is progressively changing in Scotland. The data have important implications for the management of patients with MDS, their prognosis and potential treatment.

Anonymous Patient Answer

Who should consider clinical trials for myelodysplastic syndromes?

Allogeneic stem cell and hematopoietic stem cell transplantation, as well as bone marrow and intraconventional procedures (allogeneic stem cell transplant, autologous stem cell transplantation, chemoablation, and nonmyeloablative allopurinol hyperbaric treatment are the therapeutic modalities used for treatment of MDS). However, all these modalities have significant toxicity in terms of hematopoietic or nonhematopoietic toxicity. The clinical trial for myelodysplastic syndromes should be tailored according to the MDS subtype.

Anonymous Patient Answer

How quickly does myelodysplastic syndromes spread?

The spread of MDS to other body sites is rare after the initial onset, probably because of the blood-lymphatic barrier, and because of the limited capacity of MDS for spreading to other places by way of the blood stream.

Anonymous Patient Answer

Does myelodysplastic syndromes run in families?

MDS run in families. Families with MDS should be offered genetic counselling when appropriate. Genetic counselling was provided to all members of one family. Genetic counselling is appropriate, for families with MDS, as many members have inherited at least one pathogenic MDS gene mutation.

Anonymous Patient Answer

What are the chances of developing myelodysplastic syndromes?

Myelodysplastic syndromes are relatively rare, but they can be a problem if they develop into acute myeloid leukemia. Those with a positive family history generally have a better prognosis than those without a family history.

Anonymous Patient Answer

What is the survival rate for myelodysplastic syndromes?

The median (or mid-term) survival rate for MDS in the general population is about 10 years. The 5-year survival rate is 40.2%, while the 10-year survival rate is 15.9%. The relative survival rate is 0.56 at 5 years and 0.50 at 7 years. These survival rates reflect patients with a diagnosis of MDS at a time when they are no longer at risk for developing myelodysplastic syndromes. Survival rates for patients diagnosed with MDS at an earlier time, as they approach the aging population, are probably similar to those for the general population.

Anonymous Patient Answer
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