Clinical trial NCT01400633: An Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome
Official title: A Prospective Multicenter Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myelodysplastic Syndromes
Keywords: Myelodysplastic Syndrome
Dacogen
decitabine
epigenetic therapy
hypomethylating agent
Lead sponsor: Janssen Korea, Ltd., Korea
Sponsor class: INDUSTRY
First posted: 2011-07-22
Enrollment: 156
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Secondary or tertiary hospitals in South Korea

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Patients diagnosed with (primary or secondary) Myelodysplastic Syndrome including Chronic Myelomonocytic Leukemia (CMML)
* Patients with an International Prognostic Scoring System \>= Int-1
* Patients who were never treated with hypomethylating agent (azacitidine and decitabine)
* Female patients who are postmenopausal or received contraceptive operation or refrain from sexual relations. Women of childbearing potential should conduct an effective method of birth control (oral contraceptives, injections, intrauterine device, double barrier method, contraceptive patch and male partner's sterilization), in case of male patients who will not have a baby within 2 months after the completion of decitabine therapy
* Patients who signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study.

Exclusion Criteria:

* Patients diagnosed with Acute Myelogenous Leukemia (AML, bone marrow stem cell counts exceeding 20%) or other progressive malignant diseases
* Patients with active infection of virus or bacteria
* Patients who used to be treated with azacitidine or decitabine
* Patients who are hypersensitive to excipients of decitabine
* Patients who are pregnant or breast-feeding.

Primary outcomes:
measure: overall response rate
timeFrame: every 28 days up to approximately 5 years
Secondary outcomes:
measure: The change of transfusion requirements
timeFrame: every 4 weeks up to approximately 5 years
measure: Overall survival
timeFrame: every 3 months up to approximately 5 years
measure: Adverse events
timeFrame: every 4 weeks up to approximately 5 years
Other outcomes:
Locations:
city: Ansan
country: Korea, Republic of
city: Anyang
country: Korea, Republic of
city: Busan
country: Korea, Republic of
city: Cheonan
country: Korea, Republic of
city: Chungcheongbuk-Do
country: Korea, Republic of
city: Chungnam
country: Korea, Republic of
city: Daegu
country: Korea, Republic of
city: Daejeon
country: Korea, Republic of
city: Gyeonggi-Do
country: Korea, Republic of
city: Hwasun Gun
country: Korea, Republic of
city: Incheon
country: Korea, Republic of
city: Jinju-Si
country: Korea, Republic of
city: Kwanju
country: Korea, Republic of
city: Pusan
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Suwon
country: Korea, Republic of
Study officials:
affiliation: Janssen Korea, Ltd.
name: Janssen Korea, Ltd. Clinical Trial
role: STUDY_DIRECTOR
References:
citation: Jung KS, Kim YJ, Kim YK, Park SK, Kim HG, Kim SJ, Park J, Choi CW, Do YR, Kim I, Park S, Mun YC, Jeong SH, Kim MK, Yi HG, Chang MH, Kim SY, Lee JH, Jang JH. Clinical Outcomes of Decitabine Treatment for Patients With Lower-Risk Myelodysplastic Syndrome on the Basis of the International Prognostic Scoring System. Clin Lymphoma Myeloma Leuk. 2019 Oct;19(10):656-664. doi: 10.1016/j.clml.2019.06.003. Epub 2019 Jun 27.