Voxelotor Sickle Cell Exercise Study

Status Completed

Clinical Trial 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).

Clinical Trial Description

This study will assess exercise capacity by cardiopulmonary exercise testing (CPET) before and after 8 weeks of voxelotor therapy. Patients with genetically severe forms of sickle cell disease, including Hgb SS, Hgb S beta 0 thalassemia, Hgb SC Harlem, etc., age 12 or older, with stable Hgb and Hgb F will be recruited. Enrolled subjects will have study labs drawn, undergo baseline CPET in the exercise lab, then take voxelotor 1500mg daily for 2 months, followed by repeat study labs and a second CPET. Each subject's CPET results before and after voxelotor will be compared, and the study labs before and after voxelotor will be compared. Each subject will be compared to him/herself. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04581356
Study type	Interventional
Source	Pediatric Specialists of Virginia
Contact
Status	Completed
Phase	Phase 4
Start date	September 21, 2020
Completion date	December 29, 2021

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04134299` - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease	N/A
Completed	`NCT02712346` - The Role of Endothelin-1 in Sickle Cell Disease	Phase 1
Completed	`NCT01976416` - Novel Use Of Hydroxyurea in an African Region With Malaria	Phase 3
Withdrawn	`NCT02162225` - Study of Beet Juice for Patients With Sickle Cell Anemia	Phase 2
Completed	`NCT01137721` - State Of The Art Functional Imaging In Sickle Cell Disease
Terminated	`NCT01350232` - Treatment of Sickle Cell Anemia With Stem Cell Transplant	N/A
Withdrawn	`NCT00937144` - Endothelial Function in Patients With Sickle Cell Anemia Before and After Sildenafil	Phase 4
Completed	`NCT00512564` - Clinical and Laboratory Assessment of Iron Overload in Sickle Cell Anemia and Sickle Cell Thalassemia	N/A
Completed	`NCT00512226` - Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia	N/A
Completed	`NCT00004143` - Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes	Phase 2
Completed	`NCT00004412` - Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers	Phase 2
Withdrawn	`NCT01925001` - Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis	Phase 2
Completed	`NCT01783990` - Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
Completed	`NCT01848691` - Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis	N/A
Completed	`NCT00874172` - Effectiveness of New Analgesic Strategy Compared to the Usal Antalgic Strategy	N/A
Completed	`NCT01000155` - Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease	Phase 2
Completed	`NCT00236093` - Extension Study of ACTIQ Treatment for Children and Adolescents With Breakthrough Pain	Phase 2
Completed	`NCT00399074` - Sulfadoxine- Pyrimethamine Versus Weekly Chloroquine for Malaria Prevention in Children With Sickle Cell Anemia	Phase 3
Completed	`NCT00004492` - Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia	Phase 1/Phase 2
Completed	`NCT02065596` - Hematopoietic Stem Cell Transplant for Sickle Cell Disease	Phase 1/Phase 2