Sickle Cell Disease MedDRA version: 20.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850
Conditions
Interventions
Product Name: GBT440 300 mg Tablets
Product Code: GBT440
Pharmaceutical Form: Tablet
CAS Number: 1446321-46-5
Current Sponsor code: GBT4
Sponsors
Global Blood Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female study participants with SCD who participated and received study treatment in Study GBT440-031. Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with GBT440. 2. Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. 3. Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must continue to use barrier methods of contraception to 30 days after the last dose of study drug. 4. Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant’s legal representative or legal guardian, and the participant’s assent must be obtained). Are the trial subjects under 18? yes Number of subjects for this age range: 50 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 335 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 50
Exclusion criteria
Exclusion criteria: 1. Female who is breast-feeding or pregnant. 2. Participant withdrew consent from Study GBT440-031. 3. Participant was lost to follow-up from Study GBT440-031. 4. Participant requiring chronic dialysis. 5. Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of this open-label extension (OLE) study is to assess the long-term safety and treatment effect of GBT440 in participants who have completed treatment in study GBT440-031, using the following parameters: • Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures. • Frequency of sickle cell-related complications • Hemolytic anemia as measured by hematological laboratory parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin). ;Secondary Objective: not applicable; Primary end point(s): 1. Safety endpoints include AEs, clinical laboratory tests, physical examinations and other clinical measures. 2. Frequency of SCD-related complications of long-term dosing with GBT440. 3. Hemoglobin and measures of hemolysis (e.g. reticulocytes and unconjugated bilirubin). ;Timepoint(s) of evaluation of this end point: Day 1, Weeks 4, 8, 12, 24, 48, then Q24 weeks until EOS visit (last dose, 4 Wks ± 7 days) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable | — |
Countries
Canada, Egypt, France, Ghana, Italy, Jamaica, Kenya, Lebanon, Netherlands, Nigeria, Oman, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trial Manager
Global Blood Therapeutics, Inc.
Outcome results
None listed