Clinical trial NCT01812252: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Chronic Myelomonocytic Leukemia
de Novo Myelodysplastic Syndrome
Myelodysplastic Syndrome
Secondary Myelodysplastic Syndrome
Keywords: myelodysplastic syndrome
chronic myelomonocytic leukemia
bone marrow transplant
hypomethylating agent
induction chemotherapy
Lead sponsor: Fred Hutchinson Cancer Center
Sponsor class: OTHER
First posted: 2013-03-18
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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.

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 acute myeloblastic leukemia (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)

Primary outcomes:
measure: Failure-free Survival (Failure Defined as Death or Relapse)
timeFrame: 18 months
Secondary outcomes:
measure: Quality of Life Will be Assessed Using the European Organization for Research and Treatment of Cancer Quality of Life (QoL) Questionnaire (EORTC QLQ-C30) Questionnaire.
timeFrame: EORTC QLQ-C30 questionnaire will be collected at screening, after completion of therapy (HMA 4-6 month, and up to 6 months for induction-like chemotherapy) just prior to stem cell infusion and 100 (± 14) days after stem cell infusion (HSCT).
measure: Quality of Life Will be Assessed Using the EORTC QLQ-HDC29 a Supplementary Module Assessing the Quality of Life During and After High-dose Chemotherapy and Stem Cell Transplantation.
timeFrame: EORTC QLQ-HDC29 questionnaire will be collected after completion of therapy (HMA 4-6 month, and up to 6 months for induction-like chemotherapy) pre stem cell infusion (HSCT), and 100 (± 14) days post stem cell infusion (HSCT).
measure: Overall Survival
timeFrame: Up to 18 months
measure: Number of Patients Who Relapse Post-transplant
timeFrame: Up to 18 months
measure: Number of Participants Who Received a Hematopoietic Cell Transplantation (HCT).
timeFrame: Up to 18 months
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic in Arizona
state: Arizona
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Seattle
country: United States
facility: Fred Hutch/University of Washington Cancer Consortium
state: Washington
city: Seattle
country: United States
facility: Kaiser Permanente Washington
state: Washington
Study officials:
affiliation: Fred Hutch/University of Washington Cancer Consortium
name: Bart L. Scott
role: PRINCIPAL_INVESTIGATOR
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