Neoplasms

Blood and Lymph Conditions

All Conditions

Immune System Diseases

Rare Diseases

Leukemia

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Lymphoma

Neoplasms by Histologic Type

Hematologic Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Acute Graft Versus Host Disease

Lymphosarcoma

Antineoplastic Agents

All Drugs and Chemicals

Azacitidine

Decitabine

Antimetabolites

Enzyme Inhibitors

Decitabine administered intravenously (IV) over 1 hour at 10 mg/m2 daily x 5 days every other week.

Administered intravenously (IV) over 1 hour at 10 mg/m2 daily x 5 days every other week.

Guillermo Garcia-Manero, MD

The goal of this clinical research study is to find the safety of decitabine in patients with acute lymphocytic leukemia. Upon agreement of the patient, additional blood and bone marrow samples to be used to evaluate the effect of the treatment on leukemic cells. Also, with agreement of the patient, any leftover blood and bone marrow samples that are collected at the start of the study and during the regularly scheduled evaluations to be sent for research studies. The research studies will examine changes in the blood and bone marrow cells that might help explain the causes of leukemia.

Decitabine is a potent hypomethylating agent with clinical activity in myelodysplastic syndromes (MDS), and acute and chronic myelogenous leukemia (CML). In vitro, decitabine induces loss of cell viability and apoptosis in ALL derived cell lines with known DNA methylation alterations. Exposure of these cell lines to decitabine results in hypomethylation and reactivation of putative tumor suppressor genes, an effect that is thought to have a role in the antineoplastic activity of decitabine.

Aberrant DNA methylation of multiple promoter CpG islands is frequently observed in patients with ALL both at initial presentation and at the time of relapse. Indeed these methylation marks are stable in over 70% of patients with ALL at the time of relapse. Importantly, methylation of specific molecular pathways has been associated with an extremely poor prognosis in patients with ALL. For instance, data from our laboratory has identified methylation, and silencing, of a cell cycle pathway composed of p73 and the cyclin dependent kinase inhibitors p57KIP2 and p15, as a marker of poor prognosis in patients with Philadelphia chromosome (Ph) negative disease. These results have been corroborated at the protein level: expression of p57KIP2 and or p15/p73 has been associated with a better prognosis. Finally, although the global methylation patterns observed in children with ALL, that overall have an excellent prognosis, do not seem to differ with those of older patients with the same genetic characteristics, methylation of prognostically significant pathways, such as P73/P15/P57KIP2 are remarkably lower in the younger patients. Finally, introduction of p57KIP2 in methylated/silenced ALL cell lines results in cell cycle arrest and induction of apoptosis.

All these data indicates that aberrant methylation has a role in the clinical behavior of patients with ALL and that its reversal may result in clinical benefit.

A Study of Low-Dose Decitabine in Relapsed or Refractory Acute Lymphocytic Leukemia (ALL)

Phase I Study of Low Dose 5-Aza-2'-Deoxycytidine Administered Daily for 5 Days Every Other Week for Patients With Relapsed or Refractory Acute Lymphocytic Leukemia

Eisai Inc.

M.D. Anderson Cancer Center

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Leukemia, Myelomonocytic, Juvenile

Bone Marrow Diseases

Precancerous Conditions

Leukemia, Myeloid

Myeloproliferative Disorders

Myelodysplastic-Myeloproliferative Diseases

Chronic Myelomonocytic Leukemia

Juvenile Myelomonocytic Leukemia

Chronic Myeloproliferative Disorders

Myelodysplastic/myeloproliferative Disease

Azacitidine 75 mg/m2 by Intravenous (IV) or Subcutaneous (SC) administration once daily (QD) on Days 1 to 7 of a 28-day cycle for a minimum of 3 cycles and a maximum of 6 cycles.

Bouchra Benettaib, MD

Indication Treatment of pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT).

Objectives Primary Objective The primary objective is to assess the treatment effect on response rate (MDS: either complete remission \[CR\], partial remission \[PR\], or marrow CR; JMML: either clinical complete remission \[cCR\] or clinical partial remission \[cPR\]); at Cycle 3 Day 28 (each cycle is 28 days) and to compare against standard therapy using a matched-pairs analysis of historical data.

Secondary Objective The secondary objective is to further evaluate safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of azacitidine in this subject population.

Study Design This is a prospective, open-label, Phase 2 study consisting of 2 parallel experimental arms, one for each disease group: MDS and JMML. Each arm is designed based on Simon's Optimal 2 stage study design. The sample size has been calculated to allow evaluation of the response rate at 28 day-Cycle 3 Day 28 in each of the 2 disease groups. Each of the experimental arms will also individually be compared against a historical control arm using data retrospectively collected from the European Working Group of MDS in childhood (EWOG-MDS) registry by means of a matched-pairs analysis; matched for predefined subject baseline characteristics defined before any results from this study are known post Stage 1. If matched pair is not viable then other methodologies will be explored to evaluate and compare response rates reported in literature and also in registry database Twenty subjects with MDS and 35 JMML subjects evaluable for the primary endpoint (ie, subjects that receive at least 1 dose of investigational product \[IP\]) will be enrolled at approximately 45 centers in Europe. Each experimental arm has 1 interim analysis planned (at the end of Stage 1). If, during Stage 1 evaluation, less than 2 subjects are observed with a CR, PR, or marrow CR after 3 months of azacitidine in the first 9 subjects with MDS, then enrollment will be stopped. Similarly, if less than 3 subjects are observed with a cPR or cCR after 3 months of azacitidine in the first 18 subjects with JMML, then enrollment will be stopped.

Study Population Pediatric subjects aged 1 month to less than 18 years of age with newly diagnosed conditions of advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML).

Length of Study The enrollment period will last for up to 22 months with subjects being treated for a minimum of 3 months and a maximum of 6 months, until transplantation or disease progression (based on an independent central review of responses). Once investigational product (IP) has been discontinued, subjects will then be followed for 1 year after the last dose of investigational product (IP). The follow-up may not be terminated because of new anticancer treatment or hematopoietic stem cell transplantation (HSCT).

The End of Trial is defined as either the date of the last visit of the last subject to complete the study, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as pre-specified in the protocol and/or the Statistical Analysis Plan (SAP), whichever is the later date.

Study With Azacitidine in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML)

A Phase 2, Multicenter, Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Azacitidine and to Compare Azacitidine to Historical Controls in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia Before Hematopoietic Stem Cell Transplantation

Defined as proportion of subjects with complete remission \[CR\], partial remission \[PR\] or marrow CR according to modified criteria described by Cheson 2006, adapted to pediatric reference values at 3 months (28 days cycle). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4th months).

Defined as proportion of subjects with sustained clinical complete remission \[cCR\] or clinical partial remission \[cPR\] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 monthsto end of 4 months).

Cytogenetic response is defined as the number of subjects with complete disappearance of the chromosomal abnormality without appearance of new ones divided by the number of subjects within the analysis population.

Molecular response is defined as the number of subjects with absence of somatic mutations related to JMML divided by the number of subjects within the analysis population.

Duration of response will consist of only the subjects achieving a response (CR, PR or marrow CR) and is defined as the time from first observed response until either disease progression or any cause of death.

Duration of response will consist of only the subjects achieving a response (cCR or cPR) and is defined as the time from first observed response until either disease progression or any cause of death.

TTR is defined as the time from first study dose day until a response of CR, PR or marrow CR, whichever occurs first. Only subjects observed with a response will be included in the analysis with the median TTR across the subjects presented.

Time to Response (TTR) is defined as the time from first study dose day until a response cCR or cPR, whichever occurs first. Only subjects observed with a response will be included in the analysis with the median TTR across the subjects presented.

Time to Progression (TTP) is defined as the time from first study dose day until either disease progression or death due to progression.

Leukemia free survival (LFS) is defined as the time from HEMATOPOIETIC STEM CELL transplant (HSCT) date until leukemia progression or death for subjects receiving a HSCT only. Subjects alive and leukemia-free at the time of the statistical analysis will be censored at the time of their last disease assessment. Subjects will also be censored at the time of starting a new anticancer therapy if having not previously had a leukemia progression.

OS is defined as the time from first study dose day until death from any cause. Subjects alive at the time of analysis will be censored at the time they were last known to be alive.

DNA methylation levels will be assessed across time points by means of a repeated measures analysis of variance (ANOVA) test thereby allowing identification of possible changes in methylation levels over time by treatment arm as well as comparing DNA methylation levels between treatment arms per disease indication

Defined as the proportion of subjects undergoing HSCT during the conduct of this study over the total number of subjects enrolled into this study.

The time from first study dose day until HSCT date. Subjects not receiving a HSCT will be censored at the time of the analysis.

All reported adverse events during the duration of the study conduct.

Cmax is defined as the observed maximum plasma concentration

Tmax is defined as the observed time to maximum plasma concentration

Area under the plasma concentration-time curve from time zero to the last quantifiable time point

The AUC ∞ is defined as area under the plasma concentration-time curve from time zero to infinity

Terminal phase rate constant is determined by linear regression of the terminal points of the log-linear plasma concentration-time curve

The Terminal phase half-life will be calculated according to the following equation: t½ = 0.693/λz

The total clearance is calculated as Dose/AUC∞

The volume of distribution will be calculated according to the equation: Vz = (CL)/λz

Celgene

decitabine injection decitabine intravenous injection 20mg/m2 once a day for 5 consecutive days every 4 weeks

decitabine intravenous injection 20mg/m2 once a day for 5 consecutive days every 4 weeks

decitabine injection

Janssen Korea, Ltd. Clinical Trial

The purpose of this study is to investigate the overall response rate (ORR) and safety when treating patients with myelodysplastic syndrome with decitabine. Decitabine is to be administered as long as there is evidence of clinical benefit.

This study is a prospective (the study follows a group of individuals over time), open label (all people involved know the identity of the intervention), multicenter, single arm (one group of patients receiving the same treatment), observational study to observe the response rate and safety of decitabine when treating patients with myelodysplastic syndrome with decitabine. Decitabine is to be administered as long as there is evidence of clinical benefit. It will be assessed if the treatment duration could be prolonged by appropriate toxicity management such as an active antibiotic prophylaxis or dose and schedule modification, and whether this leads to potentially increased clinical benefits such as higher response rate, longer time to Acute Myeloid Leukemia, and prolonged overall survival. For safety evaluation, adverse events and peripheral blood findings will be collected. The patient will receive decitabine intravenous injection 20mg/m2 once a day for 5 consecutive days every 4 weeks.

An Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome

A Prospective Multicenter Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome

Janssen Korea, Ltd., Korea

Leukemia, Myeloid, Acute

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Micronutrients

Vitamins

Ascorbic Acid

Arsenic Trioxide

Vitamin C

Arsenic trioxide loading dose of 0.1 mg/kg/day IV x 5 days followed by weekly doses of 0.1 mg/kg IV for 15 additional weeks.

Decitabine 20 mg/m2 IV every day on days 1-5 of a 4 weeks cycle for 4 cycles.

Arsenic trioxide loading dose of 0.2 mg/kg/day IV x 5 days followed by weekly doses of 0.2 mg/kg IV for 15 additional weeks.

Decitabine 20 mg/m2 IV every day on days 1-5 of a 4 weeks cycle for 4 cycles.

Arsenic trioxide loading dose of 0.3 mg/kg/day IV x 5 days followed by weekly doses of 0.3 mg/kg IV for 15 additional weeks.

Decitabine 20 mg/m2 IV every day on days 1-5 of a 4 weeks cycle for 4 cycles.

Ravi Vij, M.D.

This study is designed to test the combination of decitabine, arsenic trioxide and ascorbic acid in patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia

Myelodysplastic syndromes (MDS) are hematological disorders characterized by ineffective hematopoiesis. DNA hypomethylating agents such as decitabine have been shown to have activity in this disorder by reversing the epigenetic mechanism of gene silencing.

Acute Myeloid Leukemia (AML) is a hematological disorder characterized by ineffective hematopoiesis and malignant expansion of clonal myeloid cells. In elderly patients (≥ 60 years old), MDS commonly precedes the diagnosis of AML. Standard therapy for AML consists of cytotoxic chemotherapy and is often followed with allogeneic stem cell transplantation. Unfortunately, elderly patients are often unable to tolerate such aggressive therapy.

Arsenic has also shown activity in patients with MDS and AML though modulation of apoptosis via increased oxidative stress. In preclinical modes, arsenic activity is related to the production of radical oxygen species that damage mitochondria. Cellular glutathione acts as a cellular antioxidant and can be depleted with the vitamin ascorbic acid which increases intracellular oxidative stress and sensitivity to arsenic trioxide induced apoptosis.

We are studying the combination of decitabine, arsenic trioxide and ascorbic acid, two primary agents and one vitamin all with different mechanisms of action in order to improve the response rate in patients with MDS and AML. This is an open-label, single-arm, single-center, dose escalation Phase I trial of decitabine, arsenic trioxide and ascorbic acid in patients with MDS, either de novo or secondary, fitting any of the FAB classifications and AML.

Decitabine, Arsenic Trioxide and Ascorbic Acid for Myelodysplastic Syndromes and Acute Myeloid Leukemia

A Phase I Study of Intravenous Decitabine in Combination With Arsenic Trioxide and Ascorbic Acid in Patients With Myelodysplastic Syndromes and Acute Myeloid Leukemia

Cephalon

Washington University School of Medicine

Anti-Infective Agents

Venetoclax

Voriconazole

Posaconazole

hypomethylating agents (azacytidine 32mg/m2 or decitabine 5mg/m2) for 5 days and venetoclax 400mg/d for 7 days, repeated every 28 days until up to 1-year posttransplant.

Participants will receive maintenance therapy with venetoclax and azacitidine or decitabine after allogeneic stem cell transplantation.

Azacitidine will be administered once daily subcutaneously (32mg/m2/d) on days 1-5, and venetoclax will be administered once daily orally (400mg/day) on days 1-7. If the patient is refractory or allergic to azacitidine, they will receive decitabine. Decitabine will be administered intravenously (5mg/m2/d) on days 1-5. If the patient is treated with CYP450 inhibitors(such as posaconazole or voriconazole), the dose of venetoclax will reduce to 100 mg once daily on days 1-7. Maintenance therapy will start from the 60th to 120th days after allogeneic hematopoietic stem cell transplantation and repeat every 28 days for up to 10 cycles within the first year after transplantation.

Azacitidine or decitabine

Xueying Ding, Ph.D.

Xueying Ding, Ph.D

Yanhong Zhu, M.S

Liping Wan, M.D., Ph.D.

Liping Wan, M.D.

The main objective of the study is to evaluate the efficacy and safety of maintenance therapy with hypomethylating agent and Venetoclax to improve leukemia free survival for high-risk myeloid malignancies after allogeneic hematopoietic stem cell transplantation .

This is a prospective single-arm study. Patients with high-risk AML or MDS aged between 18-70 years old will enroll in the study. They will be given hypomethylating agents (azacytidine 32mg/m2 or decitabine 5mg/m2) for 5 days and venetoclax 400mg/d for 7 days after allogeneic hematopoietic stem cell transplantation. The maintenance therapy will start from 60th days posttransplant, repeated every 28 days until up to 1-year posttransplant. The 1-year leukemia-free survival rate，1-year cumulative recurrence rate, and 1-year overall survival will be analyzed.

Hypomethylating Agent and Venetoclax After Allo-HSCT in Patients With High-risk Myeloid Malignancies.

A Single Arm, Phase 2 Study Evaluating Safety and Efficacy of Maintenance Therapy With Hypomethylating Agent and Venetoclax After Allogeneic Stem Cell Transplantation in Patients With f High-risk Myeloid Malignancies.

Summary statistics for LFS time will be computed for all patients.

Use the method of Gooley et al to estimate the cumulative incidence of relapse.

The method of Kaplan and Meier will be used to estimate the distribution of overall survival. Cox proportional hazards regression analysis will be used to model the association between overall survival and covariates of interest.

Descriptive statistics will be used to summarize adverse events.

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Associate Director of Department of Hematology

This is an observational study. Patients receiving 20 mg/m2 of decitabine injection intravenously (into a vein) once daily for 5 days every 4 weeks will be observed.

No intervention

Janssen Korea, Ltd., Korea Clinical Trial

The purpose of this study is to evaluate the effectiveness and safety of decitabine (Dacogen) intravenous injection in patients with Myelodysplastic Syndrome.

This is a prospective (in which the patients are first identified and then followed forward as time passes), multi-center (study conducted at multiple sites), observational study (a scientific study to make a clear and easy understanding of the cause and effect relationship) to evaluate the effectiveness and safety information of a 5 day decitabine (Dacogen) regimen in patients with Myelodysplastic Syndrome. This study consist of 3 phases; pre-treatment phase, treatment phase and end of treatment (Day 28\~61 after last administration of Dacogen). The patients will receive decitabine intravenous injection 20 mg/m2 one hour once daily for 5 consecutive days for every 4 weeks. Safety evaluations including adverse events and clinical laboratory tests and will be evaluated with adverse events reported for the period ranging from informed consent and during the study to the end of treatment visit including 56 days (8 weeks) after the last administration of the clinical study treatment.

A Study for Dacogen Treatment in Patients With Myelodysplastic Syndrome

A Prospective Multicenter Observational Study for Dacogen Treatment in Patients With Myelodysplastic Syndrome

The complete response includes the evaluations of Bone marrow aspiration and biopsy (less than or equal to 5 percents myeloblast), persistent dysplasia and peripheral blood.

The partial response includes all complete remission evaluating parameters with the exception of bone marrow blasts are decreased by more than or equal to 50 percents over pretreatment but still more than 5 percents and cellularity (the state of a tissue or other mass as regards the number of its constituent cells) and morphology (examination of structure)

Response rate is the combination of complete remission, partial remission and hematological improvement and performed according to the response criteria of 'International Working Group 2006' which is standardized criteria for assessing response are essential to ensure comparability among clinical trials for patients with MDS.

Overall survival will be evaluated from the registration day to death.

The time of progression from myelodysplastic syndromes to acute myeloid leukemia.

Hospitalization or undergoes surgical procedure due to disease progression.

Leukemia, Myelomonocytic, Chronic

Neoplasm Metastasis

Chronic Disease

Disease Attributes

Chronic Graft Versus Host Disease

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.

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.

Azacitidine (AZC)

Quality-of-Life Assessment

Bart L. Scott

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.

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.

Chemotherapy in Treating Patients With Myelodysplastic Syndrome Before Donor Stem Cell Transplant

Initial Cytoreductive Therapy for Myelodysplastic Syndrome Prior to Allogeneic Hematopoietic Cell Transplantation (the ICT-HCT Study)

18-month failure-free survival (failure defined as death or relapse).

The European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ-C30) (version 3) is a 30-item cancer-specific questionnaire for measuring the health-related quality of life (QOL) in cancer patients. It includes five functioning scales (physical, PF; role, RF; cognitive, CF; emotional, EF; and social, SF), three symptom scales (fatigue, FA; pain, PA; and nausea and vomiting, NV), a global health status/QOL scale (GL), and six single items (dyspnea, appetite loss, sleep disturbance, constipation, diarrhea, and financial impact of the disease and treatment). All items employ a 4-point Likert scale, ranging from 1 (not at all) to 4 (very much) with lower scores representing a better outcome and higher scores a worse outcome; with the exception of two items in the GL scale, which use 7-point scales (1=very poor to 7=excellent) lower scores representing a worse outcome and higher score representing a better outcome.

European Organization for Research and Treatment of Cancer Quality of Life Questionnaire High Dose Chemotherapy (EORTC QLQ-HDC29) is a treatment-specific quality of life questionnaire that addresses treatment specific side effects as well as emotional, social, and family issues for patients treated with high dose regimens and HCT. The QLQ-HDC29 module includes 29 items, consisting of 6 multi-item scales and 8 single-items; all items employ a 4-point Likert scale, ranging from 1 (not at all) to 4 (very much) with lower scores representing a better outcome and higher scores a worse outcome with the exception of question 47 and 52 being the a higher score resulting in a better outcome and lower score a worse outcome.

The total length of follow-up will be 18 months from the start of treatment (day 1). Four categories were added for participants alive 18 months from start of treatment (day 1) 1) Participants alive after 18 months from start of treatment who received hematopoietic cell transplantation (HCT); 2) Participants alive after 18months from start of treatment who did not receive HCT.; 3) Participants deceased after 18 months from start of treatment who received HCT; 4) Participants deceased after 18 months from start of treatment who did not receive HCT.

To compare which of the two, intensive chemotherapy versus hypomethylating agent-based therapy, have a factor of relapse post hematopoietic cell transplantation (HCT).

Frequency at which the participants received a hematopoietic cell transplantation (HCT)

Fred Hutchinson Cancer Center

Professor

Serious AEs and Other AEs were not collected for this study. Only All-Cause Mortality was monitored.

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.

Azacitidine (AZC): Given IV or SC

Decitabine: Given IV or SC

Quality-of-Life Assessment: Ancillary studies

Arm A (Decitabine or Azacitidine)

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.

Quality-of-Life Assessment: Ancillary studies

Arm B (Induction-like Chemotherapy Regimen)

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Eligible patients will be randomized in a 1:1 fashion. The primary outcome will be analyzed on an intention-to-treat basis.

Bart Lee Scott M.D.

Number of subjects enrolled and randomized in each arm.

Failure-free Survival (Failure Defined as Death or Relapse)

EORTCQLQ-C30 at Screening 1. Do you have any trouble doing strenuous activities?

EORTCQLQ-C30 at Pre-Transplant 1. Do you have any trouble doing strenuous activities?

EORTCQLQ-C30 at post-transplant 1. Do you have any trouble doing strenuous activities?

EORTCQLQ-C30 at Screening 2. Do you have any trouble taking a long walk?

EORTCQLQ-C30 at Pre-transplant 2. Do you have any trouble taking a long walk?

EORTCQLQ-C30 at Post-transplant 2. Do you have any trouble taking a long walk?

EORTCQLQ-C30 at Screening 3. Do you have any trouble taking a short walk outside of the house?

EORTCQLQ-C30 at Pre-transplant 3. Do you have any trouble taking a short walk outside of the house?

EORTCQLQ-C30 at Post-transplant 3.Do you have any trouble taking a short walk outside of the house?

EORTCQLQ-C30 at Screening 4. Do you need to stay in bed or a chair during the day?

EORTCQLQ-C30 at Pre transplant 4. Do you need to stay in bed or a chair during the day?

EORTCQLQ-C30 at Post-transplant 4. Do you need to stay in bed or a chair during the day?

EORTCQLQ-C30 at Screening 5. Do you need help with eating, dressing, washing yourself?

EORTCQLQ-C30 at Pre-transplant 5. Do you need help with eating, dressing, washing yourself?

EORTCQLQ-C30 at Post-transplant 5. Do you need help with eating, dressing, washing yourself?

EORTCQLQ-C30 at Screening 6. Were you limited in doing either your work or other daily activities?

EORTCQLQ-C30 Pre-transplant 6.Were you limited in doing either your work or other daily activities?

EORTCQLQ-C30 Post-Transplant 6.Were you limited in doing either your work/ other daily activities?

EORTCQLQ-C30 Screening 7.Were you limited in pursing your hobbies or other leisure time activities?

EORTCQLQ-C30 Pre-transplant 7.Were you limited in pursing your hobbies/other leisure activities?

EORTCQLQ-C30 Post-transplant 7.Were you limited in pursing your hobbies/other leisure activities?

EORTCQLQ-C30 Screening 8. Were you short of breath?

The QLQ-C30 is a validated and widely used questionnaire to assess QOL in cancer patients. For Arm A (Azacitidine or Decitabine) only 22 subjects submitted EORTC QLQ-C30 at screening; 14 subjects completed EORTC QLQ-C30 at pre-transplant and 10 subjects post-transplant. For Arm B (induction chemo) 25 subjects submitted EORTC QLQ-C30 at screening; 13 subjects completed QLQ-C30 at pre-transplant and 10 subjects post-transplant. Scores for each question and timepoints were averaged to obtain mean.

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.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Study of Low-Dose Decitabine in Relapsed or Refractory Acute Lymphocytic Leukemia (ALL)	NCT00349596	Acute Lymphocyt...	Decitabine	-	M.D. Anderson Cancer Center
Study With Azacitidine in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML)	NCT02447666	Myelodysplastic... Leukemia, Myelo...	Azacitidine	1 Month - 18 Years	Celgene
An Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome	NCT01400633	Myelodysplastic...	decitabine inje...	20 Years -	Janssen Korea, Ltd., Korea
Decitabine, Arsenic Trioxide and Ascorbic Acid for Myelodysplastic Syndromes and Acute Myeloid Leukemia	NCT00671697	Myelodysplastic...	Arsenic Trioxid... Decitabine	18 Years -	Washington University School of Medicine
Hypomethylating Agent and Venetoclax After Allo-HSCT in Patients With High-risk Myeloid Malignancies.	NCT05841771	Hypomethylating... Venetoclax Myeloid Maligna...	Venetoclax Azacitidine or ...	18 Years - 70 Years	Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
A Study of Low-Dose Decitabine in Relapsed or Refractory Acute Lymphocytic Leukemia (ALL)	NCT00349596	Acute Lymphocyt...	Decitabine	-	M.D. Anderson Cancer Center
A Study for Dacogen Treatment in Patients With Myelodysplastic Syndrome	NCT01041846	Myelodysplastic...	No intervention	18 Years -	Janssen Korea, Ltd., Korea
Chemotherapy in Treating Patients With Myelodysplastic Syndrome Before Donor Stem Cell Transplant	NCT01812252	Chronic Myelomo... de Novo Myelody... Myelodysplastic... Secondary Myelo...	Azacitidine (AZ... Decitabine Quality-of-Life...	18 Years -	Fred Hutchinson Cancer Center
An Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome	NCT01400633	Myelodysplastic...	decitabine inje...	20 Years -	Janssen Korea, Ltd., Korea
Hypomethylating Agent and Venetoclax After Allo-HSCT in Patients With High-risk Myeloid Malignancies.	NCT05841771	Hypomethylating... Venetoclax Myeloid Maligna...	Venetoclax Azacitidine or ...	18 Years - 70 Years	Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Phase 2 Study Adding Pracinostat to a Hypomethylating Agent (HMA) in Patients With MDS Who Failed to Respond to Single Agent HMA	NCT01993641	Myelodysplastic... MDS	pracinostat Azacitidine Decitabine	18 Years -	Helsinn Healthcare SA

Spots Global Cancer Trial Database for hypomethylating agent