Study of Decitabine and Tetrahydrouridine (THU) in Patients With Sickle Cell Disease

Phase 1 Completed 25 enrolled Yogen Saunthararajah University of Illinois at Chicago · collab

RandomizedParallel-groupSingle-blindTreatment

Summary

The purposes of this study are to observe if oral tetrahydrouridine and decitabine can increase fetal hemoglobin levels and improve the symptoms of sickle cell disease, and to monitor how patient's bodies react to oral tetrahydrouridine and decitabine.

Timeline

Start: 2012-08
Primary completion: 2018-06
Completion: 2018-06

Publications

Molokie R, Lavelle D, Gowhari M, Pacini M, Krauz L, Hassan J, Ibanez V, Ruiz MA, Ng KP, Woost P, Radivoyevitch T, Pacelli D, Fada S, Rump M, Hsieh M, Tisdale JF, Jacobberger J, Phelps M, Engel JD, Saraf S, Hsu LL, Gordeuk V, DeSimone J, Saunthararajah Y. Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study. PLoS Med. 2017 Sep 7;14(9):e1002382. doi: 10.1371/journal.pmed.1002382. eCollection 2017 Sep.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Decitabine	Small molecule	0.01 mg/kg	Oral
Subject	Decitabine	Small molecule	0.02 mg/kg	Oral
Subject	Decitabine	Small molecule	0.04 mg/kg	Oral
Subject	Decitabine	Small molecule	0.08 mg/kg	Oral
Subject	Decitabine	Small molecule	0.16 mg/kg	Oral
Subject	Tetrahydrouridine	Small molecule	10 mg/kg	Oral

Study of Decitabine and Tetrahydrouridine (THU) in Patients With Sickle Cell Disease

Summary

Timeline

Publications

Drugs

Indications