Sickle Cell Disease MedDRA version: 20.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850
Conditions
Interventions
Product Name: voxelotor 300 mg dispersible tablets
Product Code: GBT440
Pharmaceutical Form: Dispersible tablet
Current Sponsor code: GBT440
Sponsors
Global Blood Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: PART A: 1. Age 2 to =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: PART A and PART B: 1. Body weight 4x ULN for age. 5. Clinically relevant cardiac abnormality, in the opinion of the Investigator, such as: · • Hemodynamically significant heart disease, eg congenital heart disease with left to right shunt or cyanosis, congestive heart failure, or an unstable cardiac condition. · • An arrhythmic heart condition requiring medical therapy. · • History of clinically significant abnormal electrocardiogram (ECG), congenital long QT syndrome, second or third-degree heart block at rest (except for asymptomatic Mobitz type I second degree heart block). 6. Participants with clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy: • Participants with acute bacterial infection requiring antibiotic use should delay screening until the course of antibiotic therapy has been completed. • Participants with known active hepatitis A, B, or C or who are known to be human immunodeficiency virus (HIV)-positive. • Participants who have tested positive for malaria at screening. • Participants with acute malaria infection should delay screening until infection has been resolved and their hemoglobin level has, in the opinion of the Investigator, returned to the participant’s pre-infection level. 7. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable). 8. Received an investigational drug within 30 days or 5 half-lives, whichever is longer, of the parent/legal guardian signing the ICF. 9. Parent or legal guardian/participants are unlikely to comply with the study procedures. 10. Other medical, psychological or addictive condition that, in the opinion of the Investigator, would confound or interfere with evaluation of safety, efficacy and/or PK of the investigational drug, prevent compliance with the study protocol, or preclude informed consent. Additionally, for PART A only: 1. Meets the criteria for primary stroke prophylaxis. 2. Has been treated with erythropoietin or other hematopoietic growth factors within 28 days of signing informed consent/assent or if, in the opinion of the Investigator, there is an anticipated need for such agents during the study. 3. RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion) or has received a RBC transfusion or exch
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: PART A: Hb >1 g/dL from Baseline (the average of Hb results from screening and Day 1) to Week 24. PART B: From screening through to EOS visit. ; Main Objective: PART A: (placebo-controlled, double-blind, participants 2 to 1 g/dL from Baseline (the average of Hb results from screening and Day 1) to Week 24. A participant is a responder if the Hb is >1 g/dL compared to Baseline. PART B: The safety endpoints are: AEs, clinical laboratory assessments (including assessing blood chemistry, liver function test, and hematology), SCD related AEs (including dactylitis, splenic sequestration, hepatic sequestration, acute painful crisis, priapism, ACS, transient ischemic attack, stroke, sepsis), vital signs, and ECGs. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): PART A: • Rate of VOC (combined secondary endpoint analysis with main population from Phase 3 Study GBT440-031). • Change from Baseline to Week 24 in clinical measures of hemolysis (Hb, % reticulocytes, LDH, and unconjugated bilirubin). • Time to first VOC. • Rate of analgesic use, defined as proportion of days participants are on analgesics during the treatment period. • Time to first sickle cell related clinical event (eg, stroke, priapism, gall stones, acute chest syndrome, splenic sequestration, leg ulcers, and dactylitis) and rate of SCD related clinical events. • Time to first RBC transfusion and rate of RBC transfusion. • Change from Baseline to Week 48 using quality of life assessments (EQ-5DY self-administered and 2 proxy versions and the PROMIS Pediatric and PROMIS Parent Proxy Scales) PART B: • The secondary endpoint is the mean change from Baseline to Week 12 and Week 24 in clinical measures of hemolysis (Hb, % reticulocytes, LDH, and unconjugated bilirubin). • Rate of analgesic use, defined as proportion of days participants are on analgesics during the treatment period. • Rate of RBC transfusion. ; Timepoint(s) of evaluation of this end point: Measured at visits: Baseline, Weeks 2, 4, 8, 12, 16, 20, 24, 36, 48 and EOS (last doe plus 4 weeks). | — |
Countries
Egypt, France, Ghana, Kenya, Lebanon, United Kingdom, United States
Contacts
Public ContactSenior Director Regulatory Affairs
Global Blood Therapeutics, Inc.
Outcome results
None listed