Description:
This randomized clinical trial studies different chemotherapies in treating patients with
myelodysplastic syndrome before donor stem cell transplant. Giving chemotherapy before a
donor stem cell transplant helps stop the growth of cancer cells in the bone marrow,
including normal blood-forming cells (stem cells) and cancer cells, and may prevent the
myelodysplastic syndrome from coming back after the transplant. When the healthy stem cells
from a donor are infused into the patient they may help the patient's bone marrow make stem
cells, red blood cells, white blood cells, and platelets.
Title
- Brief Title: Chemotherapy in Treating Patients With Myelodysplastic Syndrome Before Donor Stem Cell Transplant
- Official Title: Initial Cytoreductive Therapy for Myelodysplastic Syndrome Prior to Allogeneic Hematopoietic Cell Transplantation (the ICT-HCT Study)
Clinical Trial IDs
- ORG STUDY ID:
2661.00
- SECONDARY ID:
NCI-2013-00538
- SECONDARY ID:
2661
- SECONDARY ID:
2661.00
- SECONDARY ID:
RG9215001
- NCT ID:
NCT01812252
Conditions
- Chronic Myelomonocytic Leukemia
- de Novo Myelodysplastic Syndrome
- Myelodysplastic Syndrome
- Secondary Myelodysplastic Syndrome
Interventions
Drug | Synonyms | Arms |
---|
Azacitidine | 5 AZC, 5-AC, 5-Azacytidine, 5-AZC, Azacytidine, Azacytidine, 5-, Ladakamycin, Mylosar, U-18496, Vidaza | Arm A (decitabine or azacitidine) |
Decitabine | 5-Aza-2'-deoxycytidine, Dacogen, Decitabine for Injection, Deoxyazacytidine, Dezocitidine | Arm A (decitabine or azacitidine) |
Purpose
This randomized clinical trial studies different chemotherapies in treating patients with
myelodysplastic syndrome before donor stem cell transplant. Giving chemotherapy before a
donor stem cell transplant helps stop the growth of cancer cells in the bone marrow,
including normal blood-forming cells (stem cells) and cancer cells, and may prevent the
myelodysplastic syndrome from coming back after the transplant. When the healthy stem cells
from a donor are infused into the patient they may help the patient's bone marrow make stem
cells, red blood cells, white blood cells, and platelets.
Detailed Description
PRIMARY OBJECTIVES:
I. To determine the effect of induction chemotherapy (IC) (intensive acute myeloid leukemia
[AML]-like therapy), versus less intensive hypomethylating agents (HMA) as initial therapy,
on failure-free survival.
SECONDARY OBJECTIVES:
I. Determine if IC (intensive AML-like therapy) in comparison to HMA as initial therapy, will
affect transplantation frequency and quality of life.
II. Conduct exploratory analysis of post-HCT outcomes (overall survival, and relapse).
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
ARM A: Patients receive decitabine or azacitidine intravenously (IV) or subcutaneously (SC)
for 7 days. Treatment repeats every 28 days for 4 cycles of decitabine or 6 cycles of
azacitidine in the absence of disease progression or unacceptable toxicity.
ARM B: Patients receive induction-like chemotherapy per standard of care or per experimental
protocol. This study does not require a specific chemotherapy regimen for Arm B.
After completion of study treatment, patients are followed up for 18 months.
Trial Arms
Name | Type | Description | Interventions |
---|
Arm A (decitabine or azacitidine) | Other | Patients receive decitabine or azacitidine IV or SC per standard of care. Treatment repeats per standard of care, every 28 days for 4 cycles of decitabine or 6 cycles of azacitidine in the absence of disease progression or unacceptable toxicity. | |
Arm B (induction-like chemotherapy regimen) | Other | Patients receive physician choice of standard of care or other experimental protocol using induction-like chemotherapy regimen. No one specific regimen is required. Several regimens are listed in the protocol for example only. | |
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of de novo or secondary myelodysplastic syndrome (MDS), including chronic
myelomonocytic leukemia, as defined by the 2008 World Health Organization
classification system
- Patients must have measurable disease requiring cytoreduction, defined as a bone
marrow myeloblast count >= 5% and < 20% on morphologic examination or by flow
cytometry in cases in which adequate morphologic examination is not possible
- Patients must be considered to have an acceptable risk of early mortality with
intensive chemotherapy as determined by the attending physician at the time of the
initial visit; since the specific therapy within each arm will be determined after
randomization, there is no threshold of organ dysfunction or performance status for
inclusion
- Considered a potential transplant candidate; the attending/treating physician will
determine transplant candidacy at the time of consent
- Capable of understanding the investigational nature, potential risks and benefits of
the study, and able to provide valid informed consent
Exclusion Criteria:
- A diagnosis of acute promyelocytic leukemia as defined by the 2008 World Health
Organization classification system
- Previous treatment for MDS or AML with intensive chemotherapy regimen (induction
chemotherapy) or hypomethylating agent
- Have any other severe concurrent disease, or have a history of serious organ
dysfunction or disease involving the heart, kidney, liver, or other organ system that
may place the patient at undue risk to undergo treatment
- Patients with a systemic fungal, bacterial, viral, or other infection not controlled
(defined as exhibiting ongoing signs/symptoms related to the infection and without
improvement, despite appropriate antibiotics or other treatment)
- Females who are pregnant or breastfeeding
- Fertile men and women unwilling to use contraceptive techniques during and for 12
months following treatment
- Any uncontrolled or significant concurrent disease, illness, or psychiatric disorder
that would compromise patient safety or compliance, interfere with consent, study
participation, follow up, or interpretation of study results
- Clinical evidence suggestive of central nervous system (CNS) involvement with MDS
unless a lumbar puncture confirms the absence of leukemic blasts in the cerebrospinal
fluid (CSF)
Maximum Eligible Age: | N/A |
Minimum Eligible Age: | 18 Years |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Failure-free survival (failure defined as death or relapse) |
Time Frame: | 18 months |
Safety Issue: | |
Description: | |
Secondary Outcome Measures
Measure: | Changes in quality of life scores using European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 |
Time Frame: | Baseline, pre-transplant, and up to 100 days post-transplant |
Safety Issue: | |
Description: | The quality-of-life questionnaires will be scored. Absolute scores will be reported. A distribution-based interpretation will be conducted using the standardized response mean to analyze changes in scores over time and differences between groups. |
Measure: | Frequency at which the patients undergo transplantation |
Time Frame: | Up to 18 months |
Safety Issue: | |
Description: | |
Measure: | Overall Survival |
Time Frame: | Up to 18 months |
Safety Issue: | |
Description: | |
Measure: | Relapse |
Time Frame: | Up to 18 months |
Safety Issue: | |
Description: | |
Details
Phase: | Phase 2 |
Primary Purpose: | Interventional |
Overall Status: | Recruiting |
Lead Sponsor: | Fred Hutchinson Cancer Research Center |
Trial Keywords
- myelodysplastic syndrome
- chronic myelomonocytic leukemia
- bone marrow transplant
- hypomethylating agent
- induction chemotherapy
Last Updated
July 20, 2021