Clinical trial NCT01751867: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Myelodysplastic Syndrome
Keywords: Myelodysplastic Syndrome
Dacogen
Oncology
Decitabine
Bone marrow disorders
Lead sponsor: Xian-Janssen Pharmaceutical Ltd.
Sponsor class: INDUSTRY
First posted: 2012-12-18
Enrollment: 135
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: 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.

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.

Primary outcomes:
measure: 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
timeFrame: 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 outcomes:
measure: 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
timeFrame: 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
measure: 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
timeFrame: 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
measure: Transfusion Independence: Number of Participants Who Were Transfusion Independent
timeFrame: Baseline; up to 2 years
measure: Mean Percentage of Duration of Hospitalization (Relative to Days on Study Treatment)
timeFrame: Up to 2 years
measure: Overall Survival Rate: Percentage of Participants Who Survived During 6 Months and 12 Months of Treatment.
timeFrame: From the date of dosing until death or lost to follow-up for up to 2.5 years after last patient was enrolled
measure: 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
timeFrame: Baseline to end of treatment (approximately up to 2 years)
Other outcomes:
Locations:
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
Study officials:
affiliation: Xian-Janssen Pharmaceutical Ltd.
name: Xian-Janssen Pharmaceutical Ltd Clinical Trial
role: STUDY_DIRECTOR
References:
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.