NCT03045510

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

Status unknown Phase 2 Last updated 7 February 2017

What this trial tests

Phase 2 trial testing decitabine in Myelodysplastic Syndromes in 50 participants. Status unknown.

Timeline

1 December 2016

Primary endpoint
30 December 2018

30 July 2020

Quick facts

Lead sponsor	Shandong University
Phase	Phase 2
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	50
Start date	1 December 2016
Primary completion	30 December 2018
Estimated completion	30 July 2020
Sites	2 locations across China

Drugs / interventions tested

• decitabine (decitabine) — full drug profile →

Conditions studied

• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →

Sponsor

Shandong University

Who can join

Adults 18 to 80, any sex, with Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Historical expectations with DNA methyltransferase inhibitor monotherapy in MDS: when is combination therapy truly "promising"?
   Brunner AM, Fell G, Steensma DP. · · 2022 · cited 3× · PMID 35143613 · DOI 10.1182/bloodadvances.2021006357