Expanded Access Protocol for Adults & Pediatric Patients With Sickle Cell

Status Available

Clinical Trial Summary

The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for the treatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatric patients aged 6 months to 11 years who have no alternative treatment options and are ineligible to participate in a clinical trial of voxelotor and the study C5341057 is the EAP for adults/adolescents 12 years of age and older for patients who cannot satisfactorily be treated with an authorized medicinal product

Clinical Trial Description

This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP. Please note that for US sites, enrollment is open only for 6months to < 4 years of age. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04724421
Study type	Expanded Access
Source	Pfizer
Contact	Pfizer CT.gov Call Center
Phone	1-800-718-1021
Email	ClinicalTrials.gov_Inquiries@pfizer.com
Status	Available
Phase

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See also
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Recruiting	`NCT05431088` - A Phase 2/3 Study in Adult and Pediatric Participants With SCD	Phase 2/Phase 3
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Withdrawn	`NCT02960503` - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease	Phase 1/Phase 2
Completed	`NCT02620488` - A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease	N/A
Completed	`NCT02567682` - Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects	Phase 1
Completed	`NCT02565082` - Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients	N/A

Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms