A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease

Phase 1 Completed 133 enrolled Global Blood Therapeutics

RandomizedParallel-groupTriple-blindTreatment

Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).

Timeline

Start: 2014-12
Primary completion: 2017-03
Completion: 2017-05

Publications

Howard J, Hemmaway CJ, Telfer P, Layton DM, Porter J, Awogbade M, Mant T, Gretler DD, Dufu K, Hutchaleelaha A, Patel M, Siu V, Dixon S, Landsman N, Tonda M, Lehrer-Graiwer J. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Voxelotor	Small molecule	500 mg	Oral
Subject	Voxelotor	Small molecule	700 mg	Oral
Subject	Voxelotor	Small molecule	900 mg	Oral
Subject	Voxelotor	Small molecule	1000 mg	Oral

A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease

Summary

Timeline

Publications

Drugs

Indications