drugset / Trial / NCT04724421

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

NCT04724421 ↗

Expanded access No longer available Pfizer

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

Timeline

Start
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Primary completion
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Completion
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Drugs

EvaluationDrugModalityDoseRoute
Subject Voxelotor Small molecule — Oral

Indications