Phase 3
N=274
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Sickle Cell Disease
Bottom Line
View on ClinicalTrials.gov: NCT03036813 ↗Enrolled (actual)
274
Serious AEs
25.1%
Results posted
Jan 2021
Primary outcome: Primary: Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 — 30; 46; 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- voxelotor (Drug); Placebo (Other)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Oct 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 |
30; 46; 6 | — |
| SECONDARY Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate |
2.4; 2.4; 2.8 | — |
| SECONDARY Percentage Change From Baseline in Hemolysis Measures |
1.6; -4.6; 3.0 | — |
| SECONDARY Percentage Change From Baseline in Hemolysis Measures |
1.6; -4.6; 3.0 | — |
| SECONDARY Percentage Change From Baseline in Hemolysis Measures |
1.6; -4.6; 3.0 | — |
| SECONDARY Percentage Change From Baseline in Hemolysis Measures |
1.6; -4.6; 3.0 | — |
Summary
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria
- Male or female study participants with sickle cell disease
- Participants have 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 × upper limit of normal
- Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30 mL/min/1.73 m^2 or on chronic dialysis
Data sourced from ClinicalTrials.gov (NCT03036813). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.