Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

Phase 3

Completed

• Conditions: Sickle Cell Disease; Anemia, Sickle Cell, Hemolytic, Congenital, Hemoglobinopathies, Genetic Diseases, Inborn

• Registration Number: LBCTR2019080217
• Lead Sponsor: Global Blood Therapeutics,Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-07-17
• Last Update Posted: 2024-08-19

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 7

Inclusion Criteria

Male or female study participants with sickle cell disease
- Participant has had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months.
- Age 12 to 65 years
- Hemoglobin (Hb) =5.5 and =10.5 g/dL during screening
- For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF

Exclusion Criteria

- More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit
- Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 60 days of signing the ICF
- Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to signing the ICF)
- Hepatic dysfunction characterized by alanine aminotransferase (ALT)>4 x ULN
- Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30mL/min/1.73m2 or on chronic dialysis

Study & Design

• Study Type: Interventional
• Study Design: Not specified