Back to resultsAn Effectiveness and Safety Study of Decitabine in Patients With Myelodysplastic Syndrome
Primary Purpose
Myelodysplastic Syndrome
Status
Completed
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Decitabine at 15 mg/m2
Decitabine at 20 mg/m2
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Myelodysplastic Syndrome, Dacogen, Oncology, Decitabine, Bone marrow disorders
Eligibility Criteria
Inclusion Criteria:
- Must have diagnosed with Myelodysplastic Syndrome (MDS) denovo (previously not present) or secondary as per the classification of French-American-British (FAB) and International Prognostic Scoring System (IPSS) greater than or eaul to 0.5 as determined by complete blood count (CBC), bone marrow assessment and bone marrow cytogenetics
- Must have an Eastern Oncology Cooperative Group (ECOG) performance status of 0-2
- Must have adequate hepatic and renal function as measured by the aspartate transaminase (AST), alanine transaminase (ALT), total bilirubin and serum creatinine, respectively
- Must have recovered from all toxic effects of prior therapy and not received any chemotherapy for a minimum of 4 weeks (6 weeks if the patient has been treated with a nitrosoureas) prior to the first dose of study drug - Woman must be postmenopausal, or surgically sterile, or abstinent, or, if sexually active, be practicing an effective method of birth control (eg, oral contraceptives, contraceptive injections, intrauterine device, double-barrier method, contraceptive patch, male partner sterilization)
Exclusion Criteria:
- Must not have a diagnosis of acute myeloid leukemia (greater than 30% bone marrow blasts) - Must not have received radiotherapy within 14 days before the first dose of study drug - Must not have any other prior cancer, other than superficial bladder cancer, basal cell skin and cervical cancer - Must not have associated autoimmune hemolytic anemia or immune thrombocytopenia and inaspirable bone marrow - Must not have a mental illness or any other condition (eg, uncontrolled cardiac or pulmonary disease, diabetes), that could prevent full cooperation with the study requirements.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
3-Day Dose schedule
5-Day Dose schedule
Arm Description
Decitabine will be administered at a dose of 15 mg/m2 as a continuous intravenous infusion within a 3 hour period, repeated every 8 hours for 3 consecutive days. Cycles will be repeated every 6 weeks.
Decitabine will be administered at a dose of 20 mg/m2 as a intravenous infusion within 1 hour, once daily for 5 consecutive days. Cycles will be repeated every 4 weeks.
Outcomes
Primary Outcome Measures
Overall Response Rate (ORR): Number of Participants Who Achieved Either Complete Remission (CR), Partial Remission (PR), or Marrow Complete Remission (mCR) - International Working Group (IWG) 2006 Response Criteria
IWG 2006 response criteria - CR: bone marrow evaluation shows less than or equal to (<=) 5% blasts; normal maturation of all cells lines (mCR), peripheral blood evaluation shows hemoglobin >= 11 gram per deciliter (g/dL), neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts; PR: Same as CR, except blasts decrease by >=50%, still greater than 5% in bone marrow.
Secondary Outcome Measures
Hematological Improvement Rate: Number of Participants Who Achieved Complete Remission (CR), Partial Remission (PR) and Hematologic Improvement (HI) - International Working Group (IWG) 2006 Response Criteria
IWG 2006 response criteria - CR: bone marrow evaluation shows <= 5% blasts; normal maturation of all cells lines (mCR), peripheral blood evaluation shows hemoglobin >= 11 g/dL, neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts; PR: Same as CR, except blasts decrease by >= 50%, still greater than 5% in bone marrow; HI: hemoglobin increase of >= 1.5 g/dL, platelet increase of >= 30,000/mL (starting with > 20,000/mL), neutrophils increase of >= 100% and > 500/μL.
Cytogenetic Response Rate: Percentage of Participants Who Achieved Cytogenetic Response (Complete+Partial) by Status of Clinical Overall Response - International Working Group (IWG) 2006 Response Criteria
As per IWG 2006 response criteria - Complete cytogenetic response: disappearance of the chromosomal abnormality without appearance of new ones; Partial cytogenetic response: At least 50% reduction of the chromosomal abnormality. Status of Clinical response - complete remission (CR); marrow CR (mCR); partial remission (PR).
Transfusion Independence: Number of Participants Who Were Transfusion Independent
A participant was considered to be transfusion independent, if the participant had no transfusions of Red Blood Cells (RBCs) or platelets for 8 consecutive weeks or more.
Mean Percentage of Duration of Hospitalization (Relative to Days on Study Treatment)
Duration of hospitalization was calculated as, total number of days a participant stayed in hospital during study treatment divided by the study treatment duration
Overall Survival Rate: Percentage of Participants Who Survived During 6 Months and 12 Months of Treatment.
Mean Change From Baseline to End of Treatment in Scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ C-30) Physical Functioning Scale
EORTC QLQ-C30 is a questionnaire to assess quality of life of cancer patients. It is composed of 30 items, multi-item measure (28 items) and 2 single-item measures. For the multiple item measure, 4-point scale is used and the score for each item range from "1 = not at all" to "4 = very much". Higher scores indicate worsening. The 2 single-item measure involves question about the overall health and overall quality of life which will be rated on a 7-point scale ranging from "1 = very poor" to "7 = excellent". Lower scores indicate worsening. Scores are averaged, and transformed to 0-100 scale; higher score=better level of physical functioning.
Full Information
NCT ID
NCT01751867
First Posted
July 25, 2012
Last Updated
April 1, 2016
Sponsor
Xian-Janssen Pharmaceutical Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT01751867
Brief Title
An Effectiveness and Safety Study of Decitabine in Patients With Myelodysplastic Syndrome
Official Title
An Open-label, Multi-center, Phase IIIb Study for Decitabine in Patients With Myelodysplastic Syndrome (MDS)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2016
Overall Recruitment Status
Completed
Study Start Date
August 2009 (undefined)
Primary Completion Date
May 2011 (Actual)
Study Completion Date
April 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Xian-Janssen Pharmaceutical Ltd.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the effectiveness and safety of decitabine in the treatment of myelodysplastic syndrome (name of a group of conditions that occur when the blood-forming cells in the bone marrow are damaged) in Chinese patients.
Detailed Description
This is a prospective (look forward using periodic observations collected predominantly following patient enrollment), open-label (all people involved in the study know the identity of the assigned drug), Phase IIIb study to evaluate the efficacy and safety of decitabine in the treatment of myelodysplastic syndrome (MDS). Patients are randomized (study drug assigned by chance) in 1:1 ratio to receive treatment with decitabine either 3-day or 5-day course of therapy. When a minimum of 30 patients are reached for 3-day course of therapy, the rest of the patients will all be enrolled into 5-day course of therapy. Each patient in the study treated for a minimum of 4 cycles; however, a complete or partial response may take longer than 4 cycles. The entire study duration for each patient will be approximately two years. Safety will be evaluated for each patient by monitoring of adverse events, physical examinations, vital signs measurements, electrocardiogram, hematology and clinical chemistry testing.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome
Keywords
Myelodysplastic Syndrome, Dacogen, Oncology, Decitabine, Bone marrow disorders
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
135 (Actual)
8. Arms, Groups, and Interventions
Arm Title
3-Day Dose schedule
Arm Type
Experimental
Arm Description
Decitabine will be administered at a dose of 15 mg/m2 as a continuous intravenous infusion within a 3 hour period, repeated every 8 hours for 3 consecutive days. Cycles will be repeated every 6 weeks.
Arm Title
5-Day Dose schedule
Arm Type
Experimental
Arm Description
Decitabine will be administered at a dose of 20 mg/m2 as a intravenous infusion within 1 hour, once daily for 5 consecutive days. Cycles will be repeated every 4 weeks.
Intervention Type
Drug
Intervention Name(s)
Decitabine at 15 mg/m2
Intervention Description
Decitabine will be given at a dose of 15 mg/m2 as a continuous intravenous infusion within a 3-hour intravenous infusion, repeated every 8 hours for 3 consecutive days.The total dose per day is 45 mg/m2; The total dose per course is 135 mg/m2. Cycles will be repeated every 6 weeks.
Intervention Type
Drug
Intervention Name(s)
Decitabine at 20 mg/m2
Intervention Description
Decitabine will be given at a dose of 20 mg/m2 as 1-hour IV infusion once daily on Days 1 through 5, of a 4-week treatment cycle.
Primary Outcome Measure Information:
Title
Overall Response Rate (ORR): Number of Participants Who Achieved Either Complete Remission (CR), Partial Remission (PR), or Marrow Complete Remission (mCR) - International Working Group (IWG) 2006 Response Criteria
Description
IWG 2006 response criteria - CR: bone marrow evaluation shows less than or equal to (<=) 5% blasts; normal maturation of all cells lines (mCR), peripheral blood evaluation shows hemoglobin >= 11 gram per deciliter (g/dL), neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts; PR: Same as CR, except blasts decrease by >=50%, still greater than 5% in bone marrow.
Time Frame
From the date of first dose until 30 to 42 days after the last dose of the 2 years treatment period, or at time of discontinuation
Secondary Outcome Measure Information:
Title
Hematological Improvement Rate: Number of Participants Who Achieved Complete Remission (CR), Partial Remission (PR) and Hematologic Improvement (HI) - International Working Group (IWG) 2006 Response Criteria
Description
IWG 2006 response criteria - CR: bone marrow evaluation shows <= 5% blasts; normal maturation of all cells lines (mCR), peripheral blood evaluation shows hemoglobin >= 11 g/dL, neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts; PR: Same as CR, except blasts decrease by >= 50%, still greater than 5% in bone marrow; HI: hemoglobin increase of >= 1.5 g/dL, platelet increase of >= 30,000/mL (starting with > 20,000/mL), neutrophils increase of >= 100% and > 500/μL.
Time Frame
From the date of first dose until 30 to 42 days after the last dose of the 2 years treatment period, or at time of discontinuation
Title
Cytogenetic Response Rate: Percentage of Participants Who Achieved Cytogenetic Response (Complete+Partial) by Status of Clinical Overall Response - International Working Group (IWG) 2006 Response Criteria
Description
As per IWG 2006 response criteria - Complete cytogenetic response: disappearance of the chromosomal abnormality without appearance of new ones; Partial cytogenetic response: At least 50% reduction of the chromosomal abnormality. Status of Clinical response - complete remission (CR); marrow CR (mCR); partial remission (PR).
Time Frame
From the date of first dose until 30 to 42 days after the last dose of the 2 years treatment period, or at time of discontinuation
Title
Transfusion Independence: Number of Participants Who Were Transfusion Independent
Description
A participant was considered to be transfusion independent, if the participant had no transfusions of Red Blood Cells (RBCs) or platelets for 8 consecutive weeks or more.
Time Frame
Baseline; up to 2 years
Title
Mean Percentage of Duration of Hospitalization (Relative to Days on Study Treatment)
Description
Duration of hospitalization was calculated as, total number of days a participant stayed in hospital during study treatment divided by the study treatment duration
Time Frame
Up to 2 years
Title
Overall Survival Rate: Percentage of Participants Who Survived During 6 Months and 12 Months of Treatment.
Time Frame
From the date of dosing until death or lost to follow-up for up to 2.5 years after last patient was enrolled
Title
Mean Change From Baseline to End of Treatment in Scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ C-30) Physical Functioning Scale
Description
EORTC QLQ-C30 is a questionnaire to assess quality of life of cancer patients. It is composed of 30 items, multi-item measure (28 items) and 2 single-item measures. For the multiple item measure, 4-point scale is used and the score for each item range from "1 = not at all" to "4 = very much". Higher scores indicate worsening. The 2 single-item measure involves question about the overall health and overall quality of life which will be rated on a 7-point scale ranging from "1 = very poor" to "7 = excellent". Lower scores indicate worsening. Scores are averaged, and transformed to 0-100 scale; higher score=better level of physical functioning.
Time Frame
Baseline to end of treatment (approximately up to 2 years)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Must have diagnosed with Myelodysplastic Syndrome (MDS) denovo (previously not present) or secondary as per the classification of French-American-British (FAB) and International Prognostic Scoring System (IPSS) greater than or eaul to 0.5 as determined by complete blood count (CBC), bone marrow assessment and bone marrow cytogenetics
Must have an Eastern Oncology Cooperative Group (ECOG) performance status of 0-2
Must have adequate hepatic and renal function as measured by the aspartate transaminase (AST), alanine transaminase (ALT), total bilirubin and serum creatinine, respectively
Must have recovered from all toxic effects of prior therapy and not received any chemotherapy for a minimum of 4 weeks (6 weeks if the patient has been treated with a nitrosoureas) prior to the first dose of study drug - Woman must be postmenopausal, or surgically sterile, or abstinent, or, if sexually active, be practicing an effective method of birth control (eg, oral contraceptives, contraceptive injections, intrauterine device, double-barrier method, contraceptive patch, male partner sterilization)
Exclusion Criteria:
Must not have a diagnosis of acute myeloid leukemia (greater than 30% bone marrow blasts) - Must not have received radiotherapy within 14 days before the first dose of study drug - Must not have any other prior cancer, other than superficial bladder cancer, basal cell skin and cervical cancer - Must not have associated autoimmune hemolytic anemia or immune thrombocytopenia and inaspirable bone marrow - Must not have a mental illness or any other condition (eg, uncontrolled cardiac or pulmonary disease, diabetes), that could prevent full cooperation with the study requirements.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xian-Janssen Pharmaceutical Ltd Clinical Trial
Organizational Affiliation
Xian-Janssen Pharmaceutical Ltd.
Official's Role
Study Director
Facility Information:
City
Beijing
Country
China
City
Chengdu
Country
China
City
Guangdong
Country
China
City
Hangzhou
Country
China
City
Nanjing
Country
China
City
Shanghai
Country
China
City
Suzhou
Country
China
City
Tianjin
Country
China
12. IPD Sharing Statement
Citations:
PubMed Identifier
26568466
Citation
Wu D, Du X, Jin J, Xiao Z, Shen Z, Shao Z, Li X, Huang X, Liu T, Yu L, Li J, Chen B, He G, Cai Z, Liang H, Li J, Ruan C. Decitabine for Treatment of Myelodysplastic Syndromes in Chinese Patients: An Open-Label, Phase-3b Study. Adv Ther. 2015 Nov;32(11):1140-59. doi: 10.1007/s12325-015-0263-8. Epub 2015 Nov 14.
Results Reference
derived
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An Effectiveness and Safety Study of Decitabine in Patients With Myelodysplastic Syndrome
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