Haematological Disorders Paediatrics
Conditions
Interventions
Placebo
Sponsors
Global Blood Therapeutics
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants who meet all of the following criteria will be eligible for study enrollment: 1. Age = 2 to <15 years at the time of informed caregiver/legal guardian consent 2. Male or female study participants with SCA (HbSS, HbSß0 thalassemia genotype) a. Documentation of SCA genotype is required and may be based on documented history of laboratory testing or must be confirmed by laboratory testing during screening 3. TCD TAMMV arterial cerebral blood flow = 170 to < 200cm/sec during the Screening Period 4. Hb = 5.5 and = 10.5 g/dL during screening 5. Participant is in stable clinical condition with stable Hb level near their usual baseline as determined by the investigator 6. For participants taking HU, the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the informed consent form (ICF) and/or assent form, and with no anticipated need for dose adjustments (other than weight based). For participants not taking HU, no anticipated need for initiation of HU in the opinion of the Investigator 7. Adequate venous access, in the opinion of the Investigator, to permit monitoring of blood variables including laboratory safety variables and collection of PK samples 8. Ability to undergo TCD assessment without sedation 9. If sexually active and female, must agree to abstain from sexual intercourse or to use a highly effective method of contraception throughout the study period and for 30 days after discontinuation of study drug. If sexually active and male, must agree to abstain from sexual intercourse or willing to use barrier methods of contraception throughout the study period and for 30 days after discontinuation of study drug. 10. Females of child-bearing potential are required to have a negative pregnancy test before the administration of study drug. 11. Written informed parental/guardian consent and participant assent (where applicable) has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines.
Exclusion criteria
Exclusion criteria: 1. Body weight 4× upper limit of normal (ULN) for age 12. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including severe or unstable pulmonary hypertension 13. Clinically significant bacterial, fungal, parasitic, or viral infection currently requiring or will require therapy. a. Participants with acute bacterial infection requiring antibiotic use should delay screening until the course of antibiotic therapy has been completed and the infection has resolved, in the opinion of the investigator. 14. Known active hepatitis A, B, or C or human immunodeficiency virus (HIV)-positive. 15. Positive test indicative of active malaria infection at screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective is to evaluate the effect of voxelotor compared to placebo on the TCD time-averaged mean of the maximum velocity (TAMMV) arterial cerebral blood flow at 24 weeks in SCD participants = 2 to < 15 years of age with conditional (170 to < 200 cm/sec) TCD flow velocity. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objectives are to evaluate the effects of voxelotor compared to placebo on: ? Change in TCD flow velocity at Week 48 and Week 96 ? Conversion of TCD category from conditional to abnormal ? Reversion of TCD category from conditional to normal ? Proportion of participants with TCD response ? Change in Hb over time ? Change in clinical measures of hemolysis | — |
Countries
Ghana, Kenya
Contacts
Public ContactIQVIA IQVIA
Regulatory contact
Outcome results
None listed