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

Myelodysplastic Syndromes Clinical Trial

Official title:

A Single-center Prospective Clinical Trial of the Efficacy and Safety of Ultra Small Dose Decitabine for the Lower Risk Myelodysplastic Syndrome Patients With Transfusion Dependent

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03045510
• Other study ID #: lower risk MDS-decitabine
• Status: Recruiting
• Phase: Phase 2
• First received: February 3, 2017
• Last updated: February 6, 2017
• Start date: December 1, 2016
• Est. completion date: July 30, 2020

Study information

• Verified date: January 2017
• Source: Shandong University
• Contact: Hou Ming
• Email: houming[@]medmail.com.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: July 30, 2020
• Est. primary completion date: December 30, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

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

Locations

Country	Name	City	State
China	Qilu hospital, Shandong University	Jinan	Shandong
China	Shandong University Qilu Hospital	Jinan	Shandong

Sponsors (1)

Lead Sponsor	Collaborator
Shandong University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	complete response	Bone marrow blasts not more than 5%, absolute neutrophil count more than 1*10^9/L, HgB more than 100g/L, and platelet count more than 100*10^9/L.	30 days from the emrollment