Efficacy and Safety of Ultra Small Dose Decitabine for the Lower Risk MDS Patients With Transfusion Dependent

NCT ID: NCT03045510

Last Updated: 2017-02-07

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-12-01
• Study Completion Date: 2020-07-30

Brief Summary

Myelodysplastic syndrome (MDS) is widely recognized as a clonal hematopoietic stem cell disorder. Decitabine has been approved for the treatment of all subtypes of myelodysplastic syndrome (MDS). However, the use of decitabine is often limited by its severe toxicity represented by myelosuppression even at relatively low doses. In lower-risk patients (including IPSS low and int-1 risk groups), treatment mainly aims at improving cytopenias, especially anemia. However, although several drugs may improve anemia, sometimes durably, most of lower risk MDS eventually require red blood cell (RBC) transfusions during their disease course. Long term RBC transfusions lead to iron overload mainly due to an increase in reticulo-endothelial iron recycling.Cardiac, liver and endocrine (diabetes mellitus) dysfunction due to iron overload and often leading to fatal outcome has been reported in heavily transfused lower risk MDS patients.

To date, the optimal regimen for decitabine treatment is not well established. In this study, we perform a prospective analysis to explore the decitabine schedule for the treatment of lower risk myelodysplastic syndrome patients with transfusion dependent.

Detailed Description

The investigators are undertaking a single-center, single-arm study of 50 lower risk myelodysplastic syndrome patients with transfusion dependent from Shandong University Qilu Hospital . All the participants are selected to receive ultra small dose decitabine treatment (given intravenously at a dose of 3.5mg/m2, qd x 5d, every four weeks for one cycle). A routine blood examination is performed twice every week. Bone marrow (BM) is examined with routine aspirate smear and G-banding analysis every 1-2 treatment courses to evaluate responses.Adverse events are also recorded throughout the study.

Conditions

Myelodysplastic Syndromes

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ultra small dose decitabine

Decitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles.

Group Type: EXPERIMENTAL

decitabine

Intervention Type: DRUG

Decitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles.

Interventions

decitabine

Decitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles.

Intervention Type: DRUG

Other Intervention Names

ultra small dose decitabine

Eligibility Criteria

Inclusion Criteria

• diagnosis of MDS
• The IPSS [17] score ≤ 1
• patients with transfusion dependent
• Adequate hepatic and renal function (aspartate aminotransferase [AST] ≤ 2.5 x upper normal limit, alanine aminotransferase [ALT] ≤ 2.5 x upper normal limit, bilirubin ≤ 1.5 x upper normal limit and creatinine < 2 x upper normal limit, Ccr > 60ml/min ).

Exclusion Criteria

• Decitabine and Arsenic trioxide allergy
• Pregnancy and lactation
• Cardiovascular disease
• ECOG score > 2
• HCV, HIV, HBsAg seropositive status

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shandong University

OTHER

Sponsor Role: lead

Responsible Party

Ming Hou

Professor and Director

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Ming Lv, Doctor

Role: PRINCIPAL_INVESTIGATOR

Shandong University

Locations

Qilu hospital, Shandong University

Jinan, Shandong, China

Site Status: RECRUITING

Shandong University Qilu Hospital

Jinan, Shandong, China

Site Status: RECRUITING

Countries

China

Central Contacts

Hou Ming

Role: CONTACT

houming@medmail.com.cn

Facility Contacts

Ming Hou

Role: primary

houming@medmail.com.cn

Ming Lv

Role: primary

Other Identifiers

lower risk MDS-decitabine

Identifier Type: -

Identifier Source: org_study_id