Name of Study: GBT-HOPE – Study to Evaluate the Effect of GBT440 Administered Orally to Patients with Sickle Cell Disease
Brief Summary: A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease
Intervention- Drug: GBT440 vs. Other: Placebo
Age range: 12 – 65yr
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 the 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 an RBC transfusion for any reason within 60 days of signing the ICF
- Hospitalized for sickle cell crisis or other vaso-occlusive events 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
Sites in Ontario:
1. The Hospital for Sick Children, Toronto, ON
Contact: Manuela Merelles-Pulcini Email: manuela.merelles-pulcini@sickkids.ca
2. University Health Network, Toronto, ON
Contact: Emily Nelson Email: EmilySusan.Nelson@uhn.ca
Status: Recruiting
Click here for more information about this study
Source: clinical trials.gov Identifier #: NCT03036813
Last updated: May 31st, 2018