Voxelotor Sickle Cell Exercise Study

Phase 4 Completed 14 enrolled Elizabeth Yang, MD, PhD Global Blood Therapeutics · collab Pediatrix · collab University of California, San Francisco · collab

NaSingle-groupOpen-labelTreatment

Summary

This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA).

Timeline

Start: 2020-09-21
Primary completion: 2021-10-25
Completion: 2021-12-29

Publications

Phan V, Hershenson J, Caldarera L, Larkin SK, Wheeler K, Cortez AL, Dulman R, Briere N, Lewis A, Kuypers FA, Yang E. Effect of voxelotor on cardiopulmonary testing in youths with sickle cell anemia in a pilot study. Pediatr Blood Cancer. 2023 Aug;70(8):e30423. doi: 10.1002/pbc.30423. Epub 2023 May 29.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Voxelotor	Small molecule	1500 mg	Oral

Voxelotor Sickle Cell Exercise Study

Summary

Timeline

Publications

Drugs

Indications