Haematological Disorders
Conditions
Interventions
Placebo
Sponsors
Global Blood Therapeutics
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female study participants with SCD who participated and received study treatment in 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 voxelotor. 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 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
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. 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 |
|---|---|
| The primary objective is to assess the efficacy of GBT440 in adolescents and adults with SCD as measured by improvement in anemia. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objectives are to evaluate the effects of GBT440 compared to placebo on SCD symptom exacerbation and Total Symptom Score (TSS) from Patient Reported Outcome measurement (PRO), measures of anemia and hemolysis, and other clinical measures (e.g., vaso-occlusive crisis [VOC], Acute Chest Syndrome [ACS], hospitalization, red blood cell [RBC] transfusions, opioid use). | — |
Countries
Kenya
Contacts
Public ContactMichelle Botha
Ass Dir Regulatory and Start Up
Outcome results
None listed