Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia

Phase 2 Completed 6 enrolled Carelon Research National Heart, Lung, and Blood Institute (NHLBI) · collab

NaSingle-groupOpen-labelTreatment

Summary

Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels. This study will evaluate the safety and effectiveness of decitabine at increasing hemoglobin levels in people with TI.

Timeline

Start: 2008-01
Primary completion: 2010-05
Completion: 2010-09

Outcome

Met primary endpoint

paper increased total hemoglobin from 7.88 ± 0.88 g/dL to 9.04 ± 0.77 g/dL (P = .004) PMID 21700776 ↗

registry Decitabine; p <0.01; Regression, Linear NCT00661726 ↗

Publications

Results Olivieri NF, Saunthararajah Y, Thayalasuthan V, Kwiatkowski J, Ware RE, Kuypers FA, Kim HY, Trachtenberg FL, Vichinsky EP; Thalassemia Clinical Research Network. A pilot study of subcutaneous decitabine in beta-thalassemia intermedia. Blood. 2011 Sep 8;118(10):2708-11. doi: 10.1182/blood-2011-03-341909. Epub 2011 Jun 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Decitabine	Small molecule	0.2 mg/kg	Subcutaneous